Latest news with #LydiaAbad-Franch
Yahoo
25-07-2025
- Business
- Yahoo
TRYNGOLZA® (olezarsen) recommended for approval in the EU by CHMP for familial chylomicronemia syndrome (FCS)
– Recommendation based on Phase 3 Balance results, which showed a significant reduction of triglycerides and substantial reduction of acute pancreatitis events with TRYNGOLZA – – European Commission decision expected by Q4 2025 – CARLSBAD, Calif., July 25, 2025--(BUSINESS WIRE)--Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Sobi® today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion of TRYNGOLZA® (olezarsen) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS). The positive opinion is now referred to the European Commission (EC) for an approval decision, which is expected by Q4 2025. "Building on the strong early launch of TRYNGOLZA in the U.S., the positive CHMP opinion advances our commitment to expand access to TRYNGOLZA globally," said Brett P. Monia, Ph.D., chief executive officer, Ionis. "TRYNGOLZA has demonstrated significant reductions in triglycerides and substantial reductions in acute pancreatitis events with favorable safety and tolerability. With this robust clinical profile, combined with Sobi's deep commercial market expertise in FCS, TRYNGOLZA has the potential to make a meaningful difference for people living with FCS in the EU." The CHMP opinion is based on positive data from the Phase 3 Balance study, in which TRYNGOLZA demonstrated a statistically significant reduction in triglyceride levels at six months that was sustained through 12 months. Additionally, TRYNGOLZA demonstrated a substantial and clinically meaningful reduction in acute pancreatitis events over 12 months. TRYNGOLZA showed a favorable safety and tolerability profile. Study results were published in The New England Journal of Medicine (NEJM). FCS is a rare and genetic form of severe hypertriglyceridemia (sHTG) that prevents the body from breaking down fats and severely impairs the ability to remove triglycerides from the bloodstream. People with FCS often have triglyceride levels of more than 880 mg/dL (10 mmol/L), compared to normal levels of <150 mg/dL (1.7 mmol/L), and are at high risk of developing acute pancreatitis, which can be life-threatening. In the EU, FCS is estimated to impact up to 13 people per million. Sobi has exclusive rights to commercialize TRYNGOLZA in countries outside the U.S., Canada and China. As Ionis' European commercial partner for Waylivra (volanesorsen), the only medicine currently approved for FCS in the EU, Sobi will leverage existing market expertise and distribution channels to enable an effective TRYNGOLZA launch in FCS, if approved. "The approval recommendation brings us one step closer toward delivering TRYNGOLZA to people living with FCS in the EU and is a testament to our long-standing support for the FCS community," said Lydia Abad-Franch, M.D., MBA, head of research, development and medical affairs and chief medical officer, Sobi. "Patients with FCS suffer from complications such as acute pancreatitis. These are very severe events, often requiring intensive care and sometimes causing multiorgan failure as well as increasing morbidity and mortality. We believe TRYNGOLZA has the potential to be an important treatment for people living with this rare and serious disease, and we look forward to the final decision from the EC later this year." TRYNGOLZA was approved in the United States in December 2024 and granted orphan designation in the EU. Olezarsen is also being evaluated for sHTG, a serious condition defined by dangerously high triglycerides (≥500 mg/dL), and data from the Phase 3 CORE and CORE2 studies are expected in Q3 2025. About the Balance Study Balance is a global, multicenter, randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of olezarsen in patients with FCS at six and 12 months. The primary endpoint was the percent change from baseline in fasting triglyceride levels at six months compared to placebo. Secondary endpoints included percent changes in triglyceride levels at 12 months, percent changes in other lipid parameters and adjudicated acute pancreatitis event rates over the treatment period. Following treatment and the end-of-trial assessments, patients were eligible to enter an open-label extension study to continue receiving olezarsen once every four weeks. About Familial Chylomicronemia Syndrome (FCS) FCS is a rare, genetic disease characterized by extremely elevated triglyceride levels. It is caused by impaired function of the enzyme lipoprotein lipase (LPL). Because of limited LPL production or function, people with FCS cannot effectively break down chylomicrons, lipoprotein particles that are 90% triglycerides. People living with FCS are at high risk of acute pancreatitis in addition to other chronic health issues such as fatigue and severe, recurrent abdominal pain. People living with FCS are sometimes unable to work, adding to the burden of disease. About TRYNGOLZA® (olezarsen) TRYNGOLZA® is an RNA-targeted medicine designed to lower the body's production of apoC-III, a protein produced in the liver that is a key regulator of triglyceride metabolism. TRYNGOLZA® (olezarsen) is approved in the United States as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). For more information about TRYNGOLZA, visit TRYNGOLZA is not yet approved for any indication in Europe. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS TRYNGOLZA is contraindicated in patients with a history of serious hypersensitivity to TRYNGOLZA or any of the excipients in TRYNGOLZA. Hypersensitivity reactions requiring medical treatment have occurred. WARNINGS AND PRECAUTIONS Hypersensitivity Reactions Hypersensitivity reactions (including symptoms of bronchospasm, diffuse erythema, facial swelling, urticaria, chills and myalgias) have been reported in patients treated with TRYNGOLZA. Advise patients on the signs and symptoms of hypersensitivity reactions and instruct patients to promptly seek medical attention and discontinue use of TRYNGOLZA if hypersensitivity reactions occur. ADVERSE REACTIONS The most common adverse reactions (incidence >5% of TRYNGOLZA-treated patients and >3% higher frequency than placebo) were injection site reactions, decreased platelet count and arthralgia. Please see full Prescribing Information for TRYNGOLZA. About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has marketed medicines and a leading pipeline in neurology, cardiology and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit and follow us on X (Twitter), LinkedIn and Instagram. Ionis Forward-looking Statements This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of our commercial medicines, olezarsen, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2024, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company. In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals® and TRYNGOLZA® are trademarks of Ionis Pharmaceuticals, Inc. View source version on Contacts Ionis Investor Contact: D. Wade Walke, 760-603-2331Ionis Media Contact: Hayley Soffermedia@ 760-603-4679 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
Business Wire
25-07-2025
- Business
- Business Wire
TRYNGOLZA® (olezarsen) recommended for approval in the EU by CHMP for familial chylomicronemia syndrome (FCS)
CARLSBAD, Calif.--(BUSINESS WIRE)-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Sobi® today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion of TRYNGOLZA® (olezarsen) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS). The positive opinion is now referred to the European Commission (EC) for an approval decision, which is expected by Q4 2025. 'Building on the strong early launch of TRYNGOLZA in the U.S., the positive CHMP opinion advances our commitment to expand access to TRYNGOLZA globally,' said Brett P. Monia, Ph.D., chief executive officer, Ionis. 'TRYNGOLZA has demonstrated significant reductions in triglycerides and substantial reductions in acute pancreatitis events with favorable safety and tolerability. With this robust clinical profile, combined with Sobi's deep commercial market expertise in FCS, TRYNGOLZA has the potential to make a meaningful difference for people living with FCS in the EU.' The CHMP opinion is based on positive data from the Phase 3 Balance study, in which TRYNGOLZA demonstrated a statistically significant reduction in triglyceride levels at six months that was sustained through 12 months. Additionally, TRYNGOLZA demonstrated a substantial and clinically meaningful reduction in acute pancreatitis events over 12 months. TRYNGOLZA showed a favorable safety and tolerability profile. Study results were published in The New England Journal of Medicine (NEJM). FCS is a rare and genetic form of severe hypertriglyceridemia (sHTG) that prevents the body from breaking down fats and severely impairs the ability to remove triglycerides from the bloodstream. People with FCS often have triglyceride levels of more than 880 mg/dL (10 mmol/L), compared to normal levels of <150 mg/dL (1.7 mmol/L), and are at high risk of developing acute pancreatitis, which can be life-threatening. In the EU, FCS is estimated to impact up to 13 people per million. Sobi has exclusive rights to commercialize TRYNGOLZA in countries outside the U.S., Canada and China. As Ionis' European commercial partner for Waylivra (volanesorsen), the only medicine currently approved for FCS in the EU, Sobi will leverage existing market expertise and distribution channels to enable an effective TRYNGOLZA launch in FCS, if approved. 'The approval recommendation brings us one step closer toward delivering TRYNGOLZA to people living with FCS in the EU and is a testament to our long-standing support for the FCS community,' said Lydia Abad-Franch, M.D., MBA, head of research, development and medical affairs and chief medical officer, Sobi. 'Patients with FCS suffer from complications such as acute pancreatitis. These are very severe events, often requiring intensive care and sometimes causing multiorgan failure as well as increasing morbidity and mortality. We believe TRYNGOLZA has the potential to be an important treatment for people living with this rare and serious disease, and we look forward to the final decision from the EC later this year.' TRYNGOLZA was approved in the United States in December 2024 and granted orphan designation in the EU. Olezarsen is also being evaluated for sHTG, a serious condition defined by dangerously high triglycerides (≥500 mg/dL), and data from the Phase 3 CORE and CORE2 studies are expected in Q3 2025. About the Balance Study Balance is a global, multicenter, randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of olezarsen in patients with FCS at six and 12 months. The primary endpoint was the percent change from baseline in fasting triglyceride levels at six months compared to placebo. Secondary endpoints included percent changes in triglyceride levels at 12 months, percent changes in other lipid parameters and adjudicated acute pancreatitis event rates over the treatment period. Following treatment and the end-of-trial assessments, patients were eligible to enter an open-label extension study to continue receiving olezarsen once every four weeks. About Familial Chylomicronemia Syndrome (FCS) FCS is a rare, genetic disease characterized by extremely elevated triglyceride levels. It is caused by impaired function of the enzyme lipoprotein lipase (LPL). Because of limited LPL production or function, people with FCS cannot effectively break down chylomicrons, lipoprotein particles that are 90% triglycerides. People living with FCS are at high risk of acute pancreatitis in addition to other chronic health issues such as fatigue and severe, recurrent abdominal pain. People living with FCS are sometimes unable to work, adding to the burden of disease. About TRYNGOLZA® (olezarsen) TRYNGOLZA® is an RNA-targeted medicine designed to lower the body's production of apoC-III, a protein produced in the liver that is a key regulator of triglyceride metabolism. TRYNGOLZA ® (olezarsen) is approved in the United States as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). For more information about TRYNGOLZA, visit TRYNGOLZA is not yet approved for any indication in Europe. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS TRYNGOLZA is contraindicated in patients with a history of serious hypersensitivity to TRYNGOLZA or any of the excipients in TRYNGOLZA. Hypersensitivity reactions requiring medical treatment have occurred. WARNINGS AND PRECAUTIONS Hypersensitivity Reactions Hypersensitivity reactions (including symptoms of bronchospasm, diffuse erythema, facial swelling, urticaria, chills and myalgias) have been reported in patients treated with TRYNGOLZA. Advise patients on the signs and symptoms of hypersensitivity reactions and instruct patients to promptly seek medical attention and discontinue use of TRYNGOLZA if hypersensitivity reactions occur. ADVERSE REACTIONS The most common adverse reactions (incidence >5% of TRYNGOLZA-treated patients and >3% higher frequency than placebo) were injection site reactions, decreased platelet count and arthralgia. Please see full Prescribing Information for TRYNGOLZA. About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has marketed medicines and a leading pipeline in neurology, cardiology and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit and follow us on X (Twitter), LinkedIn and Instagram. Ionis Forward-looking Statements This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of our commercial medicines, olezarsen, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2024, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company. In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals ® and TRYNGOLZA ® are trademarks of Ionis Pharmaceuticals, Inc.
Cision Canada
25-04-2025
- Business
- Cision Canada
Sanofi and Sobi extend their partnership with the World Federation of Hemophilia Humanitarian Aid Program
Up to five-year contract extension to support the WFH Humanitarian Aid Program with medicine donations and financial assistance MONTREAL, April 25, 2025 /PRNewswire/ -- The World Federation of Hemophilia (WFH) and WFH USA, together with Sanofi, through its philanthropic organization Foundation S, and Sobi, announce the signing of a contract providing support to the WFH Humanitarian Aid Program in the form of medicine donations and financial assistance for up to 5 years. This is the second renewal of the collaboration, which has been in place for more than ten years and demonstrates the partners' commitment to providing the necessary care and resources for people with hemophilia around the world. The commitment includes the donation of up to 100 million international units (IU) of factor therapy per year that will be distributed by the WFH Humanitarian Aid Program worldwide to developing countries. This will enable the WFH to deliver aid to people with inherited bleeding disorders across the globe. The new signing comes as Sanofi and Sobi celebrate the tenth anniversary of answering the WFH call to action to support the global inherited bleeding disorders community through the donation of treatment products and financial aid. Between 2014 and 2024, Sanofi and Sobi provided one billion IUs of treatment product, enabling the treatment of 22,219 people with inherited bleeding disorders. Alaa Hamed Global Head of Medical Affairs, Rare Diseases, Sanofi "Building on the legacy of the original ten-year, one billion IU commitment, this new agreement reaffirms Sanofi and Sobi's dedication to creating a predictable, sustainable program that addresses unmet needs and advances the standard of care for hemophilia worldwide. Over the past decade, these contributions have expanded treatment availability and transformed patient outcomes. We are proud to leverage the Foundation S expertise and continue providing essential treatments and with this renewal ensuring improvement to the quality of life for thousands of individuals in developing countries for years to come." Lydia Abad-Franch Head of R&D and Chief Medical Officer, Sobi "We are immensely proud to continue our contributions to the Humanitarian Aid Program of the World Federation of Hemophilia (WFH). Since the initial pledge, over 22,000 people with hemophilia have been treated with factor donated by Sobi and Sanofi. We are thrilled to continue our partnership with the WFH and look forward to the positive impact it will have on the lives of those affected by inherited bleeding disorders." Cesar Garrido President, World Federation of Hemophilia " The contributions of Sanofi and Sobi to the WFH Humanitarian Aid Program can't be overstated — and especially in the last ten years after they answered the WFH call to action. This new five-year contract will continue that legacy of support for the global inherited bleeding disorders community, and it will help us get closer to our shared vision of Treatment for All." Mark Skinner Board Member, World Federation of Hemophilia, USA "The WFH Humanitarian Aid program is an extraordinary example of collaboration. The life-changing impact of this program is a testament to what can be achieved by working together with our industry partners. The visionary leadership of Sobi and Sanofi's Foundation S has provided inspiration for others in industry, hope for patients and their families, and elevated capacity of health care professionals around the world." About the World Federation of Hemophilia The World Federation of Hemophilia (WFH) is a non-profit organization dedicated to improving and sustaining care for people with inherited bleeding disorders around the world. At the WFH, national member organizations (NMOs) and health care professionals (HCPs) work together to provide care for people with inherited bleeding disorders around the world. We partner with governments and hemophilia treatment centres to enhance knowledge through training and provide tools they need to identify, support and treat people living with bleeding disorders in their communities, while promoting global advocacy and collaboration to achieve our common goals. The WFH Humanitarian Aid Program improves the lack of access to care and treatment by providing much-needed support for people with inherited bleeding disorders in developing countries. By providing patients with a more predictable and sustainable flow of humanitarian aid donations, the WFH Humanitarian Aid Program makes it possible for patients to receive consistent and reliable access to treatment and care. None of this would be possible without the generous support of Sanofi and Sobi, our Founding Visionary Contributors; Bayer, CSL Behring and Roche, our Visionary Contributors; Grifols, our Leadership Contributor; and Takeda, our Contributor. Our vision of Treatment for All is for a world where all people with inherited bleeding disorders have access to care, regardless of their type of bleeding disorder, gender, or where they live. Our mission is to improve and sustain care for people with inherited bleeding disorders around the world. To find out more about the WFH, please visit About WFH USA WFH USA furthers the mission of the WFH in the U.S. by deepening engagement of American citizens with our global vision of Treatment for All and raising funds to bring that vision to life. By working with the World Federation of Hemophilia, we help transform communities by equipping and empowering WFH national member organizations (NMOs) and healthcare providers with the knowledge and tools they need to identify, support and treat people living with bleeding disorders around the world. To find out more about WFH USA, please visit About Sanofi We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people's lives. Our team, across the world, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY About Foundation S – The Sanofi Collective Foundation S strives to create healthier futures for generations to come. We are driven by a singular purpose: to improve the lives of vulnerable populations by catalyzing community–based solutions, expanding access to medicines, and mobilizing collective action. About Sobi Sobi® is a global biopharma company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at and LinkedIn.
