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Business Wire
25-07-2025
- Business
- Business Wire
Prilenia and Ferrer Provide Update on European Regulatory Process for Pridopidine in Huntington's Disease
NAARDEN, Netherlands & WALTHAM, Mass. & BARCELONA, Spain--(BUSINESS WIRE)-- Prilenia Therapeutics B.V. and Ferrer today announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the refusal of the marketing authorization for pridopidine's marketing authorization application for Huntington's disease (HD). We are disappointed, but undeterred in our commitment to bring what we believe is an effective therapy to patients and will explore all options collaboratively with regulators. Prilenia and Ferrer are focused on bringing pridopidine to people living with HD and amyotrophic lateral sclerosis (ALS) worldwide as quickly as possible. Near-term plans are in place to initiate a potentially registrational global HD study, to provide confirmation of the previously observed pridopidine results, and a pivotal global Phase 3 ALS study, with recruitment for both expected to start as soon as possible. About pridopidine Pridopidine (45 mg twice daily) is a potent and selective, orally administered sigma-1 receptor (S1R) agonist which stimulates key neuroprotective mechanisms often impaired in neurodegenerative diseases such as HD and ALS i. Pridopidine's extensive development program involved approximately 1,600 people, demonstrating clinically meaningful and sustained benefits in disease progression, cognition, motor ability, and quality of life in patients, with a favorable safety and tolerability profile. In addition to HD, pridopidine is in late-stage clinical development for ALS, with Prilenia and Ferrer planning to initiate a single, pivotal Phase 3 trial in ALS as early as possible, building on the findings in the population with early and rapid progressing disease from the Phase 2 HEALEY ALS Platform Trial. Pridopidine has Orphan Drug designation in HD and ALS in the US and EU, and FDA Fast Track designation for the treatment of HD ii. Nurzigma ® (pridopidine) is a registered trademark of Prilenia. About Huntington's Disease Huntington's disease (HD) is a rare, inherited, autosomal dominant, neurodegenerative disease that results in functional, motor, cognitive and behavioral symptoms. HD is caused by a mutation in the huntingtin gene iii, and each child of a parent with HD has a 50 percent chance of developing the disease. iv HD affects approximately 4.88 out of 100,000 people around the world with an additional 300,000 people at risk of developing HD v,vi. It is usually diagnosed between the ages of 30 and 50, although HD can occur at any age, including in children and young adults (known as juvenile onset HD or JHD). The disease progresses slowly over 15 to 20 years, with patients slowly losing their ability to work, communicate, manage day-to-day life and take care of themselves. This increasing disability leads to full reliance on a caregiver and, ultimately, death. The only currently available treatments for HD focus on symptomatic relief and palliative care, with nothing impacting measures of overall progression. About Prilenia Prilenia is a private biopharmaceutical company driven by an unwavering commitment to scientific excellence and accelerating progress for people affected by Huntington's disease (HD), amyotrophic lateral sclerosis (ALS) and other children and adults with neurodegenerative disorders. Our mission is simple but urgent: to develop and provide sustainable access to transformative medicines for people affected by devastating neurodegenerative diseases. On April 28, 2025, Prilenia entered into a collaboration and license agreement with Ferrer for the commercialization and co-development of pridopidine in Europe and other select markets, retaining full commercialization and development rights to pridopidine in North America, Japan and Asia Pacific. The company is incorporated in the Netherlands and backed by leading life sciences investors. For more information, please visit and connect with us on LinkedIn or X (Twitter). About Ferrer At Ferrer we use business to fight for social justice. We have long been a company that wants to do things differently; instead of maximizing shareholder returns, we reinvest much of our profit in initiatives that give back to society. Back where it belongs. We go beyond compliance and are guided by the highest standards of sustainability, ethics and integrity. As such, since 2022, we are a B Corp. Founded in Barcelona in 1959, Ferrer offers transformative solutions for life-threatening diseases in more than one hundred countries. In line with our purpose, we have an increasing focus on pulmonary vascular and interstitial lung diseases and rare neurological disorders in adults and children. Our 1,800-strong team is driven by a clear conviction: our business is not an end in itself, but a way to change lives. We are Ferrer. Ferrer for good. ___________________________ i Naia, L., Ly, P., Mota, S.I. et al. The Sigma-1 Receptor Mediates Pridopidine Rescue of Mitochondrial Function in Huntington Disease Models. Neurotherapeutics 18, 1017–1038 (2021). ii Cudkowicz, M. AAN Annual Meeting, April 6-9, 2025, San Diego, CA iii Eddings CR, Arbez N, Akimov S, Geva M, Hayden MR, Ross CA. Pridopidine protects neurons from mutant-huntingtin toxicity via the sigma-1 receptor. Neurobiol Dis. 2019 Sep;129:118-129. doi: 10.1016/ Epub 2019 May 17. PMID: 31108174; PMCID: PMC6996243. iv Myers RH. Huntington's disease genetics. NeuroRx. 2004 Apr;1(2):255-62. doi: 10.1602/neurorx.1.2.255. PMID: 15717026; PMCID: PMC534940.) v Medina et al., Prevalence and Incidence of Huntington's Disease: An Updated Systematic Review and Meta-Analysis. Mov Disord. 2022 Dec;37(12):2327-2335. vi Jiang, A., Handley, R. R., Lehnert, K., & Snell, R. G. (2023). From Pathogenesis to Therapeutics: A Review of 150 Years of Huntington's Disease Research. International Journal of Molecular Sciences, 24(16), 13021.
Globe and Mail
23-07-2025
- Business
- Globe and Mail
Lantern Pharma (NASDAQ: LTRN) Secures EU Patent for AI-Designed Cancer Drug LP-284
Lantern Pharma (NASDAQ: LTRN) announced the European Patent Office has issued a notice of allowance for a composition of matter patent covering LP-284, a clinical-stage drug candidate for relapsed or refractory non-Hodgkin's lymphoma. The patent, expected to grant with exclusivity through 2039, adds to existing IP in the U.S. and Japan and supports global commercialization efforts. LP-284, developed using Lantern's RADR(R) AI platform, is in Phase 1 trials and holds Orphan Drug Designations for MCL and HGBL. The drug targets a $4 billion global market, and its rapid development timeline and cost efficiency bolster Lantern's strategic partnership potential. To view the full press release, visit About Lantern Pharma Lantern Pharma is an AI company transforming the cost, pace, and timeline of oncology drug discovery and development. The company's proprietary AI and machine learning platform, RADR(R), leverages over 200 billion oncology-focused data points and a library of 200+ advanced ML algorithms to help solve real-world problems in oncology drug development. By harnessing the power of AI and with input from world-class scientific advisors and collaborators, Lantern has accelerated the development of its growing pipeline of therapies that span multiple cancer indications, including both solid tumors and blood cancers. On average, Lantern's newly developed drug programs have been advanced from initial AI insights to first-in-human clinical trials in 2-3 years and at approximately $2.5 million per program. NOTE TO INVESTORS: The latest news and updates relating to LTRN are available in the company's newsroom at About BioMedWire BioMedWire (BMW) is an information service that provides (1) access to our news aggregation and syndication servers, (2) BioMedNewsBreaks that summarize corporate news and information, (3) enhanced press release services, (4) social media distribution and optimization services, and (5) a full array of corporate communication solutions. As a multifaceted financial news and content distribution company with an extensive team of contributing journalists and writers, BMW is uniquely positioned to best serve private and public companies that desire to reach a wide audience of investors, consumers, journalists and the general public. BMW has an ever-growing distribution network of more than 5,000 key syndication outlets across the country. By cutting through the overload of information in today's market, BMW brings its clients unparalleled visibility, recognition and brand awareness. BMW is where news, content and information converge. To receive SMS text alerts from BioMedWire, text 'Biotech' to 888-902-4192 (U.S. Mobile Phones Only) For more information please visit Please see full terms of use and disclaimers on the BioMedWire website applicable to all content provided by BMW, wherever published or re-published:
Yahoo
10-07-2025
- Business
- Yahoo
AbbVie to pay $700M for trispecific drug from Ichnos Glenmark
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. AbbVie agreed to pay Ichnos Glenmark $700 million upfront for rights to an early-stage experimental trispecific antibody that has shown promise in multiple myeloma. Under the terms of the deal announced Thursday, AbbVie will gain rights to ISB 2001 in North America, Europe, Japan and China. Ichnos Glenmark will be eligible for as much as $1.23 billion more in payments if the medicine reaches certain development, regulatory and commercial milestones and will also receive royalties on sales if the drug reaches the market. Glenmark Pharmaceuticals, which joined with Ichnos Sciences to create Ichnos Glenmark in 2024, will be responsible for development, manufacturing and lead commercialization of the drug in emerging markets. That includes the rest of Asia, Latin America and the Middle East. AbbVie is betting on a treatment that promises to knock out cancer cells by engaging three different targets, building on the success of bispecific drugs in oncology. ISB 2001 binds CD3 on T cells, while also hunting for two proteins, BCMA and CD38, that are expressed on cancerous cells. The company says the triple action can eliminate more myeloma cells more effectively, offering the potential of a cure for the disease. AbbVie also aims to test the approach on autoimmune diseases, a therapeutic area where multifaceted antibody drugs have recently attracted interest. The promise of ISB 2001 led the Food and Drug Administration to award Orphan Drug and Fast Track Designations to the drug, smoothing the path for faster development. So far, the experimental treatment has only reached Phase 1 testing. Preliminary results showed an overall response rate of 79% among patients with relapsed or refractory multiple myeloma, according to data presented at the recent American Society of Clinical Oncology's annual meeting. The drug was generally well tolerated, the company said. Sign in to access your portfolio
Yahoo
06-06-2025
- Business
- Yahoo
Hemispherian receives positive opinion for Orphan Medicinal Product Designation in the EU for GLIX1 in glioma
OSLO, Norway, June 6, 2025 /PRNewswire/ -- Hemispherian AS, a pioneering biotech company developing next-generation therapeutics for aggressive cancers, announced today that the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending Orphan Drug Designation (ODD) for GLIX1, the company's lead molecule, for the treatment of glioma, one of the most devastating and lethal brain cancers. Significant clinical benefit beyond current therapies. The designation marks a major regulatory milestone for Hemispherian, recognizing both the urgent unmet medical need in glioma and the potential of GLIX1 to offer significant clinical benefit beyond current therapies. "We are proud to have received this recommendation for an Orphan Drug Designation from the EMA Committee. This validates our scientific approach and supports our mission to transform treatment for patients with glioma, who currently face extremely limited and ineffective options," said Zeno Albisser, CEO of Hemispherian. About the Designation Following a detailed review, the COMP determined that GLIX1 meets the criteria for orphan designation under Regulation (EC) No 141/2000. In particular: Glioma is a life-threatening and chronically debilitating disease affecting approximately 2.6 in 10,000 people in the EU. Existing therapies for Glioblastoma (a form of Glioma) offer only limited survival benefits, with median overall survival typically less than 15 months. Non-clinical studies with GLIX1 demonstrated significant tumor reduction and extended survival in validated animal models, including cases of complete tumor eradication. These results suggest that GLIX1 offers a clinically relevant advantage over existing treatments, fulfilling the EMA's criteria for "significant benefit." Benefits of Orphan Drug Designation- Faster market Access- 10 Years of market Exclusivity Orphan Drug Designation by the EMA provides Hemispherian with a range of development and commercial incentives, including: 10 years of market exclusivity in the EU upon approval. Protocol assistance and regulatory guidance from EMA during clinical development. Eligibility for fee reductions for regulatory submissions, including marketing authorization. These incentives are designed to encourage the development of innovative treatments for rare diseases with high unmet need. About Hemispherian Hemispherian is an Oslo-based pharmaceutical company focused on the development of a novel class of small-molecule drugs targeting glioblastoma and other aggressive cancers. The company's proprietary GLIX platform is based on unique DNA-targeting technology aimed at improving patient survival and quality of life. So far, the treatment shows impressive effects, with limited to no side effects, and with no damage to healthy tissue. Contact: Zeno Albisser, CEO – zeno@ +47 40603455 View original content: SOURCE Hemispherian AS Error 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
Yahoo
06-06-2025
- Business
- Yahoo
Hemispherian receives positive opinion for Orphan Medicinal Product Designation in the EU for GLIX1 in glioma
OSLO, Norway, June 6, 2025 /PRNewswire/ -- Hemispherian AS, a pioneering biotech company developing next-generation therapeutics for aggressive cancers, announced today that the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending Orphan Drug Designation (ODD) for GLIX1, the company's lead molecule, for the treatment of glioma, one of the most devastating and lethal brain cancers. Significant clinical benefit beyond current therapies. The designation marks a major regulatory milestone for Hemispherian, recognizing both the urgent unmet medical need in glioma and the potential of GLIX1 to offer significant clinical benefit beyond current therapies. "We are proud to have received this recommendation for an Orphan Drug Designation from the EMA Committee. This validates our scientific approach and supports our mission to transform treatment for patients with glioma, who currently face extremely limited and ineffective options," said Zeno Albisser, CEO of Hemispherian. About the Designation Following a detailed review, the COMP determined that GLIX1 meets the criteria for orphan designation under Regulation (EC) No 141/2000. In particular: Glioma is a life-threatening and chronically debilitating disease affecting approximately 2.6 in 10,000 people in the EU. Existing therapies for Glioblastoma (a form of Glioma) offer only limited survival benefits, with median overall survival typically less than 15 months. Non-clinical studies with GLIX1 demonstrated significant tumor reduction and extended survival in validated animal models, including cases of complete tumor eradication. These results suggest that GLIX1 offers a clinically relevant advantage over existing treatments, fulfilling the EMA's criteria for "significant benefit." Benefits of Orphan Drug Designation- Faster market Access- 10 Years of market Exclusivity Orphan Drug Designation by the EMA provides Hemispherian with a range of development and commercial incentives, including: 10 years of market exclusivity in the EU upon approval. Protocol assistance and regulatory guidance from EMA during clinical development. Eligibility for fee reductions for regulatory submissions, including marketing authorization. These incentives are designed to encourage the development of innovative treatments for rare diseases with high unmet need. About Hemispherian Hemispherian is an Oslo-based pharmaceutical company focused on the development of a novel class of small-molecule drugs targeting glioblastoma and other aggressive cancers. The company's proprietary GLIX platform is based on unique DNA-targeting technology aimed at improving patient survival and quality of life. So far, the treatment shows impressive effects, with limited to no side effects, and with no damage to healthy tissue. Contact: Zeno Albisser, CEO – zeno@ +47 40603455 View original content: SOURCE Hemispherian AS
