Latest news with #Norgine
Yahoo
13-05-2025
- Business
- Yahoo
Fennec Pharmaceuticals Reports First Quarter 2025 Financial Results and Provides Business Update
~ Achieved First Quarter 2025 Total Net Revenues of $8.8 Million, Up 18% Year Over Year ~ ~ Positive Momentum from Company's Refined, Targeted Sales Strategy and Enhanced Patient Support Services ~ ~ PEDMARQSI Now Commercially Available in Germany and the United Kingdom ~ ~ Management to Host Conference Call Today at 8:30 a.m. ET ~ RESEARCH TRIANGLE PARK, N.C., May 13, 2025 (GLOBE NEWSWIRE) -- Fennec Pharmaceuticals Inc. (NASDAQ:FENC; TSX: FRX), a specialty pharmaceutical company, today reported its financial results for the first quarter ended March 31, 2025 and provided a business update. 'In the first quarter, we sharpened our strategic focus and achieved sustainable growth across key PEDMARK® accounts. Importantly, our Fennec HEARS™ patient support program has been newly revamped to ensure growth with a seamless, supportive experience for both providers and adolescent and young adult, or AYA, cancer patients at every step of the PEDMARK® journey,' said Jeff Hackman, chief executive officer of Fennec Pharmaceuticals. 'Looking ahead, we are energized by the momentum that we are building and remain committed to executional excellence. We are deepening our relationships with key accounts and expanding adoption with new and existing customers. With this strong foundation and forward vision, we are confident in our ability to drive value for patients, providers, and shareholders.' Business Highlights: Growth Within Key PEDMARK® Accounts: In the first quarter, Fennec's segmentation model and data-driven target lists enhanced field execution yielding measurable impact, driving the addition of multiple new accounts across both academic and community providers. Significantly Strengthened Fennec HEARS™ and Specialty Pharmacy Offering: Our newly revamped patient support program offerings have launched to deliver improved experiences through strengthened HCP and patient services, expanded payer reimbursement support, and streamlined access to home nursing resources. PEDMARQSI Commercial Launch in Europe: In February 2025, Norgine commercially launched PEDMARQSI® in Germany, following commercial launches in England and Wales during the first quarter of 2025. In May 2025, Norgine announced the Scottish Medicines Consortium (SMC) acceptance of PEDMARQSI® for use in Scotland. Collectively, these launches mark important steps in achieving Fennec's mission of expanding access to PEDMARK® and PEDMARQSI® to cancer patients at risk of hearing loss in the European Union and U.K. Upcoming Events: American Society of Clinical Oncology (ASCO) 2025 Annual Meeting: Fennec will be exhibiting at the ASCO Annual Meeting at Booth #37119, from May 30 – June 3, 2025, in Chicago, IL. Annual Meeting of Shareholders: Fennec would like to invite shareholders to attend its Annual General Meeting on Tuesday, June 3, 2025 at 10:00 a.m. ET, which will be held in person in the Chairman Room at the NY Lotte Palace hotel located at 455 Madison Avenue, New York, NY 10022, or online by visiting Investor Conferences: Fennec will be participating in the upcoming HCW 3rd Annual BioConnect Conference, held in partnership with Nasdaq in NYC, on Tuesday, May 20, 2025, as well as the 22nd Annual Craig-Hallum Institutional Investor Conference being held in Minneapolis, MN on Wednesday, May 28, 2025. Financial Results for the First Quarter 2025 Fiscal Year Ended March 31, 2025 Net Product Sales – For the first quarter of 2025, the Company recorded net product sales of approximately $8.8 million compared to $7.4 million in the first quarter of 2024. The increase in sales is attributable to growth across PEDMARK accounts including new accounts in the AYA population. Selling and Marketing Expenses – The Company recorded $2.9 million in selling and marketing expenses in the first quarter of 2025 compared to $3.9 million in the fourth quarter of 2024 and $5.2 million in the first quarter of 2024. The decrease year over year is primarily attributable to the elimination of expenses associated with European pre commercialization which occurred in 2024 prior to the announcement of the Norgine partnership. General and Administrative (G&A) Expenses – The Company recorded $6.1 million in G&A expenses in the first quarter of 2025 compared to $4.1 million in the fourth quarter of 2024 and $5.9 million in the first quarter of 2024. For the first quarter of 2025, G&A expenses increased on a quarter over quarter basis primarily due to non-cash stock-based compensation. Cash Position – Cash and cash equivalents were $22.6 million as of March 31, 2025 compared to $26.6 million as of December 31, 2024. The decrease in cash in the first quarter is primarily due to seasonal spending patterns in the first quarter of the year. First Quarter 2025 Conference Call Information Date: Tuesday, May 13, 2025Time: 8:30 a.m. ETWebcast Link: Participant Link: To access the live webcast link, log onto and proceed to the News & Events/Event Calendar page under the Investors & Media heading. Please connect to the company's website at least 15 minutes prior to the conference call to ensure adequate time for any software download that may be required to listen to the webcast. A webcast replay of the conference call will also be archived on for thirty days. Financial Update The selected financial data presented below is derived from our unaudited condensed consolidated financial statements, which were prepared in accordance with U.S. generally accepted accounting principles. The complete unaudited condensed consolidated financial statements for the period ended March 31, 2025 and management's discussion and analysis of financial condition and results of operations will be available via and All values are presented in thousands unless otherwise noted. Three Months Ended March 31, March 31, 2025 2024 Revenue PEDMARK product sales, net $ 8,751 $ 7,419 Licensing revenue — 17,958 Total revenue 8,751 25,377 Operating expenses: Cost of products sold 373 550 Research and development 94 3 Selling and marketing 2,947 5,209 General and administrative 6,145 5,872 Total operating expenses 9,559 11,634 Income/(loss) from operations (808 ) 13,743 Other (expense)/income Unrealized foreign exchange loss 13 (38 ) Amortization expense (13 ) (20 ) Unrealized loss on securities (1 ) (11 ) Interest income 236 197 Interest expense (592 ) (1,034 ) Total other expense (357 ) (906 ) Net income/(loss) $ (1,165 ) $ 12,837 Basic net income/(loss) per common share $ (0.04 ) $ 0.47 Diluted net income/(loss) per common share $ (0.04 ) $ 0.41 Weighted-average number of common shares outstanding basic 27,578 27,045 Weighted-average number of common shares outstanding diluted 27,578 31,136 Unaudited Audited March 31, December 31, 2025 2024 Assets Current assets Cash and cash equivalents $ 22,675 $ 26,634 Accounts receivable, net 15,448 12,884 Prepaid expenses 2,444 3,080 Inventory 1,404 1,060 Other current assets 1,004 466 Total current assets 42,975 44,124 Non-current assets Other non-current assets, net amortization 3,428 822 Total non-current assets 3,428 822 Total assets $ 46,403 $ 44,946 Liabilities and stockholders' equity/(deficit) Current liabilities: Accounts payable $ 5,280 $ 3,241 Accrued liabilities 2,843 3,428 Operating lease liability - current 248 2 Contract liability - Norgine — 248 Total current liabilities 8,371 6,919 Long term liabilities Term loan 18,206 18,206 PIK interest 1,271 1,271 Debt discount (126 ) (139 ) Contract liabilty - Norgine 24,561 24,561 Total long term liabilities 43,912 43,899 Total liabilities 52,283 50,818 Stockholders' equity/(deficit): Common stock, no par value; unlimited shares authorized; 27,594 shares issued and outstanding (2024 ‑27,527) 145,979 145,608 Additional paid-in capital 67,744 66,958 Accumulated deficit (220,846 ) (219,681 ) Accumulated other comprehensive income 1,243 1,243 Total stockholders' equity/(deficit) (5,880 ) (5,872 ) Total liabilities and stockholders' equity/(deficit) $ 46,403 $ 44,946 About Cisplatin-Induced OtotoxicityCisplatin and other platinum compounds are essential chemotherapeutic agents for the treatment of many malignancies. Unfortunately, platinum-based therapies can cause ototoxicity, or hearing loss, which is permanent, irreversible, and particularly harmful to the survivors of pediatric cancer.i The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids or cochlear implants, which can be helpful for some, but do not reverse the hearing loss and can be costly over Infants and young children that are affected by ototoxicity at critical stages of development lack speech and language development and literacy, and older children and adolescents often lack social-emotional development and educational PEDMARK® (sodium thiosulfate injection)PEDMARK® is the first and only U.S. Food and Drug Administration (FDA) approved therapy indicated to reduce the risk of ototoxicity associated with cisplatin treatment in pediatric patients with localized, non-metastatic, solid tumors. It is a unique formulation of sodium thiosulfate in single-dose, ready-to-use vials for intravenous use in pediatric patients. PEDMARK is also the first and only therapeutic agent with proven efficacy and safety data with an established dosing regimen, across two open-label, randomized Phase 3 clinical studies, the Children's Oncology Group (COG) Protocol ACCL0431 and SIOPEL 6. As a reminder, PEDMARK is indicated to reduce the risk of ototoxicity associated with cisplatin in pediatric patients 1 month of age and older with localized, non-metastatic solid tumors. PEDMARK is recommended for the AYA population by the National Comprehensive Cancer Network, or NCCN, with a 2A endorsement. In the U.S. and Europe, it is estimated that, annually, more than 10,000 children may receive platinum-based chemotherapy. The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids. There is currently no established preventive agent for this hearing loss and only expensive, technically difficult, and sub-optimal cochlear (inner ear) implants have been shown to provide some benefit. Infants and young children that suffer ototoxicity at critical stages of development lack speech language development and literacy, and older children and adolescents lack social-emotional development and educational achievement. PEDMARK has been studied by co-operative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity, COG ACCL0431 and SIOPEL 6. Both studies have been completed. The COG ACCL0431 protocol enrolled childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, medulloblastoma, and other solid tumors. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors. Indications and UsagePEDMARK® (sodium thiosulfate injection) is indicated to reduce the risk of ototoxicity associated with cisplatin in pediatric patients 1 month of age and older with localized, non-metastatic solid tumors. Limitations of UseThe safety and efficacy of PEDMARK have not been established when administered following cisplatin infusions longer than 6 hours. PEDMARK may not reduce the risk of ototoxicity when administered following longer cisplatin infusions, because irreversible ototoxicity may have already occurred. Important Safety Information PEDMARK is contraindicated in patients with history of a severe hypersensitivity to sodium thiosulfate or any of its components. Hypersensitivity reactions occurred in 8% to 13% of patients in clinical trials. Monitor patients for hypersensitivity reactions. Immediately discontinue PEDMARK and institute appropriate care if a hypersensitivity reaction occurs. Administer antihistamines or glucocorticoids (if appropriate) before each subsequent administration of PEDMARK. PEDMARK may contain sodium sulfite; patients with sulfite sensitivity may have hypersensitivity reactions, including anaphylactic symptoms and life-threatening or severe asthma episodes. Sulfite sensitivity is seen more frequently in people with asthma. PEDMARK is not indicated for use in pediatric patients less than 1 month of age due to the increased risk of hypernatremia or in pediatric patients with metastatic cancers. Hypernatremia occurred in 12% to 26% of patients in clinical trials, including a single Grade 3 case. Hypokalemia occurred in 15% to 27% of patients in clinical trials, with Grade 3 or 4 occurring in 9% to 27% of patients. Monitor serum sodium and potassium levels at baseline and as clinically indicated. Withhold PEDMARK in patients with baseline serum sodium greater than 145 mmol/L. Monitor for signs and symptoms of hypernatremia and hypokalemia more closely if the glomerular filtration rate (GFR) falls below 60 mL/min/1.73m2. Administer antiemetics prior to each PEDMARK administration. Provide additional antiemetics and supportive care as appropriate. The most common adverse reactions (≥25% with difference between arms of >5% compared to cisplatin alone) in SIOPEL 6 were vomiting, nausea, decreased hemoglobin, and hypernatremia. The most common adverse reaction (≥25% with difference between arms of >5% compared to cisplatin alone) in COG ACCL0431 was hypokalemia. Please see full Prescribing Information for PEDMARK® at: About Fennec PharmaceuticalsFennec Pharmaceuticals Inc. is a specialty pharmaceutical company focused on the development and commercialization of PEDMARK® to reduce the risk of platinum-induced ototoxicity in pediatric patients. Further, PEDMARK received FDA approval in September 2022 and European Commission approval in June 2023 and U.K. approval in October 2023 under the brand name PEDMARQSI. PEDMARK has received Orphan Drug Exclusivity in the U.S. and PEDMARQSI has received Pediatric Use Marketing Authorization in Europe which includes eight years plus two years of data and market protection. For more information, please visit Except for historical information described in this press release, all other statements are forward-looking. Words such as 'believe,' 'anticipate,' 'plan,' 'expect,' 'estimate,' 'intend,' 'may,' 'will,' or the negative of those terms, and similar expressions, are intended to identify forward-looking statements. These forward-looking statements include statements about our business strategy, timeline and other goals, plans and prospects, including our commercialization plans respecting PEDMARK®/PEDMARQSI®, the market opportunity for and market impact of PEDMARK®/ PEDMARQSI®, its potential impact on patients and anticipated benefits associated with its use, future commercial and regulatory milestone and royalty payments from Norgine, and potential access to further funding after the date of this release. Forward-looking statements are subject to certain risks and uncertainties inherent in the Company's business that could cause actual results to vary, including the risks and uncertainties that regulatory and guideline developments may change, scientific data and/or manufacturing capabilities may not be sufficient to meet regulatory standards or receipt of required regulatory clearances or approvals, clinical results may not be replicated in actual patient settings, unforeseen global instability, including political instability, or instability from an outbreak of pandemic or contagious disease, such as the novel coronavirus (COVID-19), or surrounding the duration and severity of an outbreak, protection offered by the Company's patents and patent applications may be challenged, invalidated or circumvented by its competitors, the available market for the Company's products will not be as large as expected, the Company's products will not be able to penetrate one or more targeted markets, revenues will not be sufficient to fund further development and clinical studies, our ability to obtain necessary capital when needed on acceptable terms or at all, the Company may not meet its future capital requirements in different countries and municipalities, and other risks detailed from time to time in the Company's filings with the Securities and Exchange Commission including its Annual Report on Form 10-K for the year ended December 31, 2024. Fennec disclaims any obligation to update these forward-looking statements except as required by law. For a more detailed discussion of related risk factors, please refer to our public filings available at and PEDMARK® PEDMARQSI® and Fennec® are registered trademarks of Fennec Pharmaceuticals Inc. ©2025 Fennec Pharmaceuticals Inc. All rights reserved. FEN-1604-v1 For further information, please contact: Investors:Robert AndradeChief Financial OfficerFennec Pharmaceuticals Inc.+1 919-246-5299 Corporate and Media:Lindsay Rocco Elixir Health Public Relations+1 862-596-1304lrocco@ ____________________________ i Rybak L. Mechanisms of Cisplatin Ototoxicity and Progress in Otoprotection. Current Opinion in Otolaryngology & Head and Neck Surgery. 2007, Vol. 15: Landier W. Ototoxicity and Cancer Therapy. Cancer. June 2016 Vol. 122, No.11: Bass JK, Knight KR, Yock TI, et al. Evaluation and Management of Hearing Loss in Survivors of Childhood and Adolescent Cancers: A Report from the Children's Oncology Group. Pediatric Blood & Cancer. 2016 Jul;63(7): in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
![Norgine welcomes TGA registration in Australia of IFINWIL® (eflornithine) for adults and children diagnosed with high-risk neuroblastoma (HRNB)[1]](/_next/image?url=https%3A%2F%2Fall-logos-bucket.s3.amazonaws.com%2Fapnews.com.png&w=48&q=75){kind=small}
Associated Press
17-04-2025
- Business
- Associated Press
Norgine welcomes TGA registration in Australia of IFINWIL® (eflornithine) for adults and children diagnosed with high-risk neuroblastoma (HRNB)[1]
UXBRIDGE, England, April 17, 2025 /CNW/ -- Norgine is pleased to announce that the Australian Therapeutic Goods Administration (TGA) has approved the registration of IFINWIL® (eflornithine) for the treatment of adults and paediatric patients with high-risk neuroblastoma (HRNB), who have responded to prior multiagent, multimodality therapy.1 Neuroblastoma Australia welcomed the news today: 'On behalf of all families of children impacted by neuroblastoma, we welcome the TGA's decision to approve IFINWIL®. We urgently need treatments for children diagnosed with neuroblastoma and this milestone marks a step in the right direction towards a better future for children and their families. We thank the Federal Government for taking action to ensure access and we look forward to continued support for children with aggressive cancers.' said Lucy Jones, CEO Neuroblastoma Australia. High Risk Neuroblastoma is a rare but aggressive form of cancer, predominantly affecting children and most commonly presenting in the first 5 years of life2. Each year in Australia, approximately 50 children are diagnosed with neuroblastoma, with about half of these cases being classified as high risk neuroblastoma3,4. Neuroblastoma originates in the body's nerve cells (neuroblasts) and typically presents as a primary tumour in the adrenal glands5. It is considered an aggressive tumour because it often spreads to other parts of the body (metastasizes). In most cases, it has spread by the time it is diagnosed5. 'We are committed to improving the lives of children and their families living with high-risk neuroblastoma' said Gus Rudolph, General Manager, Norgine, Australia. 'This rare childhood cancer has devastating consequences for those impacted and while more needs to be done to improve treatment outcomes, we would like to recognise the TGA for their work to-date on this approval. Norgine will continue to engage with the relevant stakeholders to bring IFINWIL® to patients as quickly as possible.' 'This approval, as part of the Project Orbis initiative, represents a vital step forward in ensuring access to innovative cancer treatments for patients around the world,' said Dr. David Gillen, Chief Medical Officer, Norgine. 'By working collaboratively with international regulatory partners, we are able to help bring promising therapies to paediatric patients sooner – a goal that is especially important when time is critical. We're proud to support Project Orbis as we strive to expedite access to high-impact oncology medicines.' Receiving the TGA approval is an important milestone on the path to realising sustainable and equitable access. IFINWIL® is not currently included on the PBS. Please refer to the IFINWIL® Consumer Medicines Information (CMI) for full safety information on risks, side effects and precautions including the risk of low red blood cells (anaemia), low neutrophils (blood cells that fight infection), low platelets (clotting cells), increase in liver enzymes, and hearing loss or problems balancing.1 Notes to Editors: About IFINWIL® IFINWIL® has been investigated for use as a post maintenance treatment for high-risk neuroblastoma (HRNB) in paediatric patients with no active disease (NAD) / no evidence of disease (NED) after first line multiagent, multimodality therapy.6 IFINWIL® is a therapy that blocks an enzyme called ornithine decarboxylase (ODC) responsible for producing polyamines, which are important to tumour growth and development 7. For more information on IFINWIL®, find the CMI here: IFINWIL Consumer Medicine Information (CMI) or the Therapeutic Goods Administration at or speak to your healthcare practitioner. High-Risk Neuroblastoma (HRNB) Treatment Background Children diagnosed with HRNB undergo an intense treatment regimen that still leaves them vulnerable to relapse and death.8 Although there have been some improvements in survival, children with high risk neuroblastoma still face a 30% chance of recurrence (relapse) within the first 5 years post maintenance, and have an extremely poor prognosis and low likelihood of long term survival9 (e.g. estimates as low as 15% of patients will live for five years after relapsing).10 Avoiding relapse is key to long-term survival, and until now, there have been no approved therapies for the post maintenance treatment period in major markets outside of the United States11. About Project Orbis Project Orbis is an initiative (since May 2019) of the US FDA Oncology Center of Excellence (OCE) and provides a framework for concurrent submission and collaborative review of innovative oncology products among international regulatory authorities. It was created with the overarching goal to speed worldwide patient access to innovative cancer therapies. Project Orbis is coordinated by the FDA, and its partners include United Kingdom Medicines and Healthcare Products regulatory Agency (UK MHRA), Australia Therapeutic Goods Administration (TGA), Canada (Health Canada), Singapore (Health Sciences Authority (HSA), Switzerland (Swissmedic), Brazil (Agência Nacional de Vigilância Sanitária (ANVISA), Israel (Ministry of Health). In April 2024, Norgine submitted an application for approval of eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom. This milestone supports Norgine's efforts to deliver patient access to eflornithine and bring a further treatment option in the field of paediatric oncology. About Norgine Norgine is a leading European specialist pharmaceutical company that has been bringing transformative medicines to patients for over a century. Our commitment to transforming people's lives drives everything we do, and our European experience, fully integrated infrastructure and exceptional partnership approach enable us to quickly apply creative solutions to bring life-changing medicines to patients that they may not otherwise be able to access. Norgine is proud to have helped more than 25 million patients around the world in 2022 and generated €530 million in net product sales, a growth of nearly 5% over 2021. Norgine has a direct presence in 16 European countries, as well as Australia and New Zealand. We also have a strong global network of partnerships in non-Norgine markets. We are a flexible and fully integrated pharmaceutical business, with manufacturing (Hengoed, Wales and Dreux, France), third party supply networks and significant product development capabilities, in addition to our sales and marketing infrastructure. This enables us to acquire, develop and commercialise specialist and innovative products that make a real difference to the lives of patients around the world. NORGINE and the sail logo are trademarks of the Norgine group of companies. References Logo - View original content: SOURCE Norgine
Korea Herald
17-04-2025
- Business
- Korea Herald
Norgine strengthens rare disease portfolio with acquisition of Theravia
Acquisition of Theravia from Mérieux Equity Partners represents meaningful step forward in Norgine's strategy for sustainable growth, adding a complementary portfolio of rare disease medicines Transaction further establishes Norgine's position as a partner of choice for commercialising rare and specialty pharmaceuticals in Europe PARIS, April 17, 2025 /PRNewswire/ -- Norgine and Theravia announced today that they have entered into a definitive agreement under which Norgine will acquire Theravia, an international pharmaceutical company specialising in cutting-edge treatments for patients with rare and debilitating conditions. Recent deals with Fennec Pharma for PEDMARQSI ® and X4 Pharma for mavorixafor demonstrate Norgine's strong emphasis on driving growth in rare disease and specialty medicines through acquisitions and in-licensing. Theravia's innovations have resulted in successful launches of life-saving medications which have changed patient outcomes worldwide. Theravia has several products in the rare disease space, including SIKLOS ®, for adults and children with sickle cell disease, and ORPHACOL ®, a medicine for adults and children who have a genetic disorder that affects bile production by the liver. These products span both the rare haematology and rare hepatology therapeutic areas and are complementary to Norgine's existing rare and specialty portfolio. The development of Theravia in the past few years has been supported by its majority shareholder Mérieux Equity Partners, a healthcare dedicated investment firm based in France. " This acquisition is a unique opportunity for Norgine to bolster our growth trajectory, as well as our rare disease portfolio." said Janneke van der Kamp, Chief Executive Officer, Norgine. " With our strong legacy and proven track record of successfully bringing innovative treatments to patients, we believe we are well placed to ensure the Theravia medicines reach their full potential." Franck Hamalian, Chief Executive Officer, Theravia, added: "The joining of our companies brings together the skills, know-how and geographical presence that will create a European-based champion of rare and ultra rare disease. Our combined product portfolios and Norgine's legacy and experience will enable broader access to medicines for patients with high unmet medical needs." With the acquisition of Theravia, Norgine will now have six core products in its rare disease portfolio (PEDMARQSI ®, eflornithine, mavorixafor, AGILUS ®, SIKLOS ® and ORPHACOL ®) thereby comprising a franchise of critical scale with the ability to be a key growth driver in the medium-to-long-term. Norgine looks forward to building on this transaction as we continue to strengthen our platform for future acquisitions and in-licensing opportunities to drive growth. The transaction remains subject to obtaining customary regulatory approvals from the competent authorities. About Theravia Theravia is an international pharmaceutical laboratory specializing in rare or neglected diseases. Formed through the merger of Addmedica and CTRS, Theravia is dedicated to addressing the unmet medical needs of patients with these challenging conditions. About SIKLOS ® SIKLOS ® is used in adults, adolescents and children over two years of age who have sickle-cell syndrome, a genetic disease where the red blood cells become rigid and sticky and change from being disc-shaped to being crescent-shaped (like a sickle). It is used to prevent recurrent, painful vaso-occlusive crises that happen when blood vessels become blocked by the abnormal red blood cells, restricting the flow of blood to an organ. They can include acute chest syndrome, a life-threatening condition when the patient has sudden chest pain, fever, hard breathing or signs of fluid in the lungs on an X-ray. SIKLOS ® is marketed in 17 countries around the world. About ORPHACOL ® ORPHACOL ® is a medicine containing cholic acid, a substance found in the bile, which is used to digest fats. It is approved in the US and Europe used to treat adults and children from one month of age who have a genetic abnormality that makes them unable to produce bile. The therapy is used in patients who do not have enough of two specific liver enzymes which makes their liver unable to produce enough of the main components of bile, called primary bile acids, such as cholic acid. When these primary bile acids are lacking, the body produces abnormal bile acids instead which can damage the liver, potentially leading to life-threatening liver failure. ORPHACOL ® is marketed in 23 countries around the world. About PEDMARQSI ® PEDMARQSI ® is a novel formulation of anhydrous sodium thiosulfate, specifically developed and manufactured for the prevention of cisplatin-induced hearing loss in patients 1 month to <18 years of age. It is the first and only preventative treatment developed for cisplatin-induced ototoxicity to support children and adolescent patients with localised, non-metastatic solid tumour cancers. It was granted marketing authorisation by the EMA in May 2023 (under the paediatric-use marketing authorisation (PUMA) programme) and the MHRA in October 2023. About Mavorixafor Mavorixafor is a selective CXCR4 receptor antagonist approved in the U.S. and marketed by X4 as XOLREMDI ®, an oral, once-daily treatment for patients 12 years of age and older with WHIM syndrome, a rare primary immunodeficiency. In January 2025, X4 submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for mavorixafor for the treatment of WHIM syndrome, for which it has been granted Orphan Drug Designation by both the EMA and the U.S. Food and Drug Administration. X4 is also developing the medicine to treat chronic neutropenia (CN) and is currently conducting a global, pivotal Phase 3 clinical trial in certain CN disorders. Norgine has responsibility for commercialisation in Europe, Australia, and New Zealand following regulatory approvals. About Eflornithine Eflornithine has been investigated for use as a post maintenance treatment for high-risk neuroblastoma (HRNB) in paediatric patients with no active disease (NAD) / no evidence of disease (NED) after first line multiagent, multimodality therapy. It is a targeted therapy that blocks an enzyme called ornithine decarboxylase (ODC), responsible for producing polyamines, which are important to tumour growth and development. The medicine is approved by the FDA for a maintenance therapy for high-risk neuroblastoma in adult and pediatric patients. In April 2024, an application for approval was submitted by Norgine for the use of eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom. In January 2025, a marketing authorisation application was filed to the European Medicines Agency (EMA) for the medicine in high-risk neuroblastoma (HRNB). About AGILUS® AGILUS ® is a medicine used to treat malignant hyperthermia (rapid rise in body temperature caused by uncontrolled muscle contractions) in adults and children. Malignant hyperthermia is a serious reaction to certain medicines used for general anaesthesia during surgery or other medical procedures. AGILUS ® is approved for use by the EMA for for the treatment of malignant hyperthermia in adults and children in combination with adequate support measures. About Mérieux Equity Partners Founded in 2009, Mérieux Equity Partners ("MxEP") is a leading European healthcare-specialized investment firm, with two dedicated platforms, Venture Capital and Buyout, supporting companies ranging from start-ups to established leaders. Benefiting from a longstanding expertise and a large network, MxEP invests in companies with ambitious growth projects and transformative products or services in healthcare. MxEP is AMF-accredited and currently manages c.€1.5bn of AuM. About Norgine Norgine is a uniquely positioned, specialty pharmaceutical and consumer healthcare company, with over €550 million of annual revenues and a 120-year track record of bringing life-changing products to patients and consumers across our core markets of Western Europe, Australia and New Zealand. Norgine's integrated approach – strong commercial capabilities, as well as deep medical, regulatory and clinical expertise, in-house manufacturing, robust supply networks, and best-in-class enabling functions – ensures that Norgine can deliver high-quality, transformative medicines quickly and effectively to over 25 million patients annually. Norgine is a nimble, innovative, and high-performing company that has been transformed by a relentless focus on operational excellence. This focus will enable us to secure the legacy of more than a century of innovation and doing the right thing by our patients, as we push the boundaries and take strides into therapeutic innovation.
Korea Herald
17-04-2025
- Business
- Korea Herald
Norgine welcomes TGA registration in Australia of IFINWIL® (eflornithine) for adults and children diagnosed with high-risk neuroblastoma (HRNB)[1]
UXBRIDGE, England, April 17, 2025 /PRNewswire/ -- Norgine is pleased to announce that the Australian Therapeutic Goods Administration (TGA) has approved the registration of IFINWIL ® (eflornithine) for the treatment of adults and paediatric patients with high-risk neuroblastoma (HRNB), who have responded to prior multiagent, multimodality therapy. 1 Neuroblastoma Australia welcomed the news today: " On behalf of all families of children impacted by neuroblastoma, we welcome the TGA's decision to approve IFINWIL ®. We urgently need treatments for children diagnosed with neuroblastoma and this milestone marks a step in the right direction towards a better future for children and their families. We thank the Federal Government for taking action to ensure access and we look forward to continued support for children with aggressive cancers." said Lucy Jones, CEO Neuroblastoma Australia. High Risk Neuroblastoma is a rare but aggressive form of cancer, predominantly affecting children and most commonly presenting in the first 5 years of life 2. Each year in Australia, approximately 50 children are diagnosed with neuroblastoma, with about half of these cases being classified as high risk neuroblastoma 3,4. Neuroblastoma originates in the body's nerve cells (neuroblasts) and typically presents as a primary tumour in the adrenal glands 5. It is considered an aggressive tumour because it often spreads to other parts of the body (metastasizes). In most cases, it has spread by the time it is diagnosed 5. "We are committed to improving the lives of children and their families living with high-risk neuroblastoma" said Gus Rudolph, General Manager, Norgine, Australia. "This rare childhood cancer has devastating consequences for those impacted and while more needs to be done to improve treatment outcomes, we would like to recognise the TGA for their work to-date on this approval. Norgine will continue to engage with the relevant stakeholders to bring IFINWIL ® to patients as quickly as possible." "This approval, as part of the Project Orbis initiative, represents a vital step forward in ensuring access to innovative cancer treatments for patients around the world," said Dr. David Gillen, Chief Medical Officer, Norgine. "By working collaboratively with international regulatory partners, we are able to help bring promising therapies to paediatric patients sooner – a goal that is especially important when time is critical. We're proud to support Project Orbis as we strive to expedite access to high-impact oncology medicines." Receiving the TGA approval is an important milestone on the path to realising sustainable and equitable access. IFINWIL ® is not currently included on the PBS. Please refer to the IFINWIL ® Consumer Medicines Information (CMI) for full safety information on risks, side effects and precautions including the risk of low red blood cells (anaemia), low neutrophils (blood cells that fight infection), low platelets (clotting cells), increase in liver enzymes, and hearing loss or problems balancing. 1 Notes to Editors: About IFINWIL ® IFINWIL ® has been investigated for use as a post maintenance treatment for high-risk neuroblastoma (HRNB) in paediatric patients with no active disease (NAD) / no evidence of disease (NED) after first line multiagent, multimodality therapy. 6 IFINWIL ® is a therapy that blocks an enzyme called ornithine decarboxylase (ODC) responsible for producing polyamines, which are important to tumour growth and development 7. For more information on IFINWIL ®, find the CMI here: IFINWIL Consumer Medicine Information (CMI) or the Therapeutic Goods Administration at or speak to your healthcare practitioner. High-Risk Neuroblastoma (HRNB) Treatment Background Children diagnosed with HRNB undergo an intense treatment regimen that still leaves them vulnerable to relapse and death. 8 Although there have been some improvements in survival, children with high risk neuroblastoma still face a 30% chance of recurrence (relapse) within the first 5 years post maintenance, and have an extremely poor prognosis and low likelihood of long term survival 9 (e.g. estimates as low as 15% of patients will live for five years after relapsing). 10 Avoiding relapse is key to long-term survival, and until now, there have been no approved therapies for the post maintenance treatment period in major markets outside of the United States 11. About Project Orbis Project Orbis is an initiative (since May 2019) of the US FDA Oncology Center of Excellence (OCE) and provides a framework for concurrent submission and collaborative review of innovative oncology products among international regulatory authorities. It was created with the overarching goal to speed worldwide patient access to innovative cancer therapies. Project Orbis is coordinated by the FDA, and its partners include United Kingdom Medicines and Healthcare Products regulatory Agency (UK MHRA), Australia Therapeutic Goods Administration (TGA), Canada (Health Canada), Singapore (Health Sciences Authority (HSA), Switzerland (Swissmedic), Brazil (Agência Nacional de Vigilância Sanitária (ANVISA), Israel (Ministry of Health). In April 2024, Norgine submitted an application for approval of eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom. This milestone supports Norgine's efforts to deliver patient access to eflornithine and bring a further treatment option in the field of paediatric oncology. About Norgine Norgine is a leading European specialist pharmaceutical company that has been bringing transformative medicines to patients for over a century. Our commitment to transforming people's lives drives everything we do, and our European experience, fully integrated infrastructure and exceptional partnership approach enable us to quickly apply creative solutions to bring life-changing medicines to patients that they may not otherwise be able to access. Norgine is proud to have helped more than 25 million patients around the world in 2022 and generated €530 million in net product sales, a growth of nearly 5% over 2021. Norgine has a direct presence in 16 European countries, as well as Australia and New Zealand. We also have a strong global network of partnerships in non-Norgine markets. We are a flexible and fully integrated pharmaceutical business, with manufacturing (Hengoed, Wales and Dreux, France), third party supply networks and significant product development capabilities, in addition to our sales and marketing infrastructure. This enables us to acquire, develop and commercialise specialist and innovative products that make a real difference to the lives of patients around the world.
Yahoo
16-04-2025
- Business
- Yahoo
Norgine strengthens rare disease portfolio with acquisition of Theravia
Acquisition of Theravia from Mérieux Equity Partners represents meaningful step forward in Norgine's strategy for sustainable growth, adding a complementary portfolio of rare disease medicines Transaction further establishes Norgine's position as a partner of choice for commercialising rare and specialty pharmaceuticals in Europe PARIS, April 15, 2025 /CNW/ -- Norgine and Theravia announced today that they have entered into a definitive agreement under which Norgine will acquire Theravia, an international pharmaceutical company specialising in cutting-edge treatments for patients with rare and debilitating conditions. Recent deals with Fennec Pharma for PEDMARQSI® and X4 Pharma for mavorixafor demonstrate Norgine's strong emphasis on driving growth in rare disease and specialty medicines through acquisitions and in-licensing. Theravia's innovations have resulted in successful launches of life-saving medications which have changed patient outcomes worldwide. Theravia has several products in the rare disease space, including SIKLOS®, for adults and children with sickle cell disease, and ORPHACOL®, a medicine for adults and children who have a genetic disorder that affects bile production by the liver. These products span both the rare haematology and rare hepatology therapeutic areas and are complementary to Norgine's existing rare and specialty portfolio. The development of Theravia in the past few years has been supported by its majority shareholder Mérieux Equity Partners, a healthcare dedicated investment firm based in France. "This acquisition is a unique opportunity for Norgine to bolster our growth trajectory, as well as our rare disease portfolio." said Janneke van der Kamp, Chief Executive Officer, Norgine. "With our strong legacy and proven track record of successfully bringing innovative treatments to patients, we believe we are well placed to ensure the Theravia medicines reach their full potential." Franck Hamalian, Chief Executive Officer, Theravia, added: "The joining of our companies brings together the skills, know-how and geographical presence that will create a European-based champion of rare and ultra rare disease. Our combined product portfolios and Norgine's legacy and experience will enable broader access to medicines for patients with high unmet medical needs." With the acquisition of Theravia, Norgine will now have six core products in its rare disease portfolio (PEDMARQSI®, eflornithine, mavorixafor, AGILUS®, SIKLOS® and ORPHACOL®) thereby comprising a franchise of critical scale with the ability to be a key growth driver in the medium-to-long-term. Norgine looks forward to building on this transaction as we continue to strengthen our platform for future acquisitions and in-licensing opportunities to drive growth. The transaction remains subject to obtaining customary regulatory approvals from the competent authorities. Notes About TheraviaTheravia is an international pharmaceutical laboratory specializing in rare or neglected diseases. Formed through the merger of Addmedica and CTRS, Theravia is dedicated to addressing the unmet medical needs of patients with these challenging conditions. About SIKLOS®SIKLOS® is used in adults, adolescents and children over two years of age who have sickle-cell syndrome, a genetic disease where the red blood cells become rigid and sticky and change from being disc-shaped to being crescent-shaped (like a sickle). It is used to prevent recurrent, painful vaso-occlusive crises that happen when blood vessels become blocked by the abnormal red blood cells, restricting the flow of blood to an organ. They can include acute chest syndrome, a life-threatening condition when the patient has sudden chest pain, fever, hard breathing or signs of fluid in the lungs on an X-ray. SIKLOS® is marketed in 17 countries around the world. About ORPHACOL®ORPHACOL® is a medicine containing cholic acid, a substance found in the bile, which is used to digest fats. It is approved in the US and Europe used to treat adults and children from one month of age who have a genetic abnormality that makes them unable to produce bile. The therapy is used in patients who do not have enough of two specific liver enzymes which makes their liver unable to produce enough of the main components of bile, called primary bile acids, such as cholic acid. When these primary bile acids are lacking, the body produces abnormal bile acids instead which can damage the liver, potentially leading to life-threatening liver failure. ORPHACOL® is marketed in 23 countries around the world. About PEDMARQSI®PEDMARQSI® is a novel formulation of anhydrous sodium thiosulfate, specifically developed and manufactured for the prevention of cisplatin-induced hearing loss in patients 1 month to <18 years of age. It is the first and only preventative treatment developed for cisplatin-induced ototoxicity to support children and adolescent patients with localised, non-metastatic solid tumour cancers. It was granted marketing authorisation by the EMA in May 2023 (under the paediatric-use marketing authorisation (PUMA) programme) and the MHRA in October 2023. About MavorixaforMavorixafor is a selective CXCR4 receptor antagonist approved in the U.S. and marketed by X4 as XOLREMDI®, an oral, once-daily treatment for patients 12 years of age and older with WHIM syndrome, a rare primary immunodeficiency. In January 2025, X4 submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for mavorixafor for the treatment of WHIM syndrome, for which it has been granted Orphan Drug Designation by both the EMA and the U.S. Food and Drug Administration. X4 is also developing the medicine to treat chronic neutropenia (CN) and is currently conducting a global, pivotal Phase 3 clinical trial in certain CN disorders. Norgine has responsibility for commercialisation in Europe, Australia, and New Zealand following regulatory approvals. About EflornithineEflornithine has been investigated for use as a post maintenance treatment for high-risk neuroblastoma (HRNB) in paediatric patients with no active disease (NAD) / no evidence of disease (NED) after first line multiagent, multimodality therapy. It is a targeted therapy that blocks an enzyme called ornithine decarboxylase (ODC), responsible for producing polyamines, which are important to tumour growth and development. The medicine is approved by the FDA for a maintenance therapy for high-risk neuroblastoma in adult and pediatric patients. In April 2024, an application for approval was submitted by Norgine for the use of eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom. In January 2025, a marketing authorisation application was filed to the European Medicines Agency (EMA) for the medicine in high-risk neuroblastoma (HRNB). About AGILUS®AGILUS® is a medicine used to treat malignant hyperthermia (rapid rise in body temperature caused by uncontrolled muscle contractions) in adults and children. Malignant hyperthermia is a serious reaction to certain medicines used for general anaesthesia during surgery or other medical procedures. AGILUS® is approved for use by the EMA for for the treatment of malignant hyperthermia in adults and children in combination with adequate support measures. About Mérieux Equity PartnersFounded in 2009, Mérieux Equity Partners ("MxEP") is a leading European healthcare-specialized investment firm, with two dedicated platforms, Venture Capital and Buyout, supporting companies ranging from start-ups to established leaders. Benefiting from a longstanding expertise and a large network, MxEP invests in companies with ambitious growth projects and transformative products or services in healthcare. MxEP is AMF-accredited and currently manages c.€1.5bn of AuM. About NorgineNorgine is a uniquely positioned, specialty pharmaceutical and consumer healthcare company, with over €550 million of annual revenues and a 120-year track record of bringing life-changing products to patients and consumers across our core markets of Western Europe, Australia and New Zealand. Norgine's integrated approach – strong commercial capabilities, as well as deep medical, regulatory and clinical expertise, in-house manufacturing, robust supply networks, and best-in-class enabling functions – ensures that Norgine can deliver high-quality, transformative medicines quickly and effectively to over 25 million patients annually. Norgine is a nimble, innovative, and high-performing company that has been transformed by a relentless focus on operational excellence. This focus will enable us to secure the legacy of more than a century of innovation and doing the right thing by our patients, as we push the boundaries and take strides into therapeutic innovation. NORGINE and the sail logo are trademarks of the Norgine group of companies. Logo - View original content: SOURCE Norgine View original content: Sign in to access your portfolio
