Iptacopan

Max to immediately begin process to provide humanitarian access for Fabhalta® (iptacopan) in collaboration with its network of partner hematologists across 53 countries People living with the rare blood disease in low-middle income countries require frequent blood transfusions, experience debilitating symptoms of fatigue and without treatment may progress to more serious life-threatening diseases The Max Foundation and Novartis have collaborated to improve access to medicines in low-resource countries for more than two decades SEATTLE, Dec. 6, 2024 /PRNewswire/ -- The Max Foundation (Max), a global nonprofit organization dedicated to accelerating health equity by delivering medication, technology, and supportive services to patients worldwide, today announced it will expand its collaboration with Novartis to include Fabhalta® (iptacopan), providing access to innovative treatment in low resource countries for PNH, a rare and debilitating blood disease that causes red blood cells to break apart. "We are thrilled to expand our reach to include people living with PNH in low-resource countries without access to an innovative treatment for this life-threatening condition," said Pat Garcia-Gonzalez, CEO of Max. "This collaboration reaffirms the deeply shared value of health equity for both organizations by making the latest treatment approved for the disease available at no cost, fulfilling our mission to accelerate health equity and enabling people to live with dignity and hope." PNH is caused by a genetic flaw that affects how red blood cells and platelets work. Only about 6 per 1 million people are diagnosed with it each year. People with PNH have sudden symptoms, including fatigue, weakness, increased heart rate, or shortness of breath, triggered by stressors like physical exertion or infection. During these episodes, red blood cells are broken down earlier than they should be, which causes patients to pass dark-colored urine. If PNH isn't treated, it can cause anemia, chronic kidney disease, or blood clots. "For more than 20 years, Novartis has successfully partnered with The Max Foundation to advance health equity by providing access to our innovative oncology medicines. Together, we have reached more than 100,000 patients by now, delivering sustainable and profound impact on communities around the globe. Novartis is proud to further expand our strong partnership, thereby accelerating early access for PNH patients in need," said Lutz Hegemann, President, Global Health at Novartis. The program will initially focus on identifying partner physicians ready to treat PNH and creating access pathways into 53 countries in Sub-Saharan Africa, Latin America, the Caribbean, and Southeast Asia and Asia Pacific. In addition to medication access, Max is seeking to collaborate with medical experts to strengthen health systems by providing education and training, identifying other potential collaborators in low- and middle-income countries, building capacity at medical institutions and facilitating conversations and partnership engagement to expand access to treatment. Max and Novartis have collaborated to improve access to innovative medicines since 2001. Starting with access to medicines for chronic myeloid leukemia and gastrointestinal stromal tumors, the collaboration has expanded to include advanced breast cancer and multiple rare diseases. Novartis is a member of Max's Humanitarian Partnership for Access to Critical Treatments (Humanitarian PACT), a collaboration among professional, nonprofit, and commercial organizations that share the commitment of the foundation to increase global access to treatment, care, and support for people living with cancer and other critical illnesses. Members of the Humanitarian PACT agree to invest resources and/or their unique organizational knowledge and capabilities to support the expansion of Max's proven treatment access model. About The Max Foundation The Max Foundation is a global health nonprofit organization dedicated to accelerating health equity. For 26 years, Max has pioneered practical, scalable, high-quality solutions to bring lifesaving treatments and patient-centered health care to more than 100,000 people living with cancer and critical illness in low- and middle-income countries. Max believes in a world where all people can access high-impact medicines, where geography is not destiny, and where everyone can strive for health with dignity and with hope. Learn more at . SOURCE The Max Foundation WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Novartis’ Fabhalta improved hemoglobin levels in patients with a rare blood disorder who switched from anti-C5 therapies, bolstering the drug’s case against AstraZeneca’s competing treatments. Fabhalta has been approved for exactly one year in paroxysmal nocturnal hemoglobinuria, a disorder that causes red blood cells to break down and can lead to anemia or blood clots. Some patients require transfusions. AstraZeneca’s C5 inhibitors Soliris and Ultomiris have long been the standard of care, each generating roughly $3 billion in 2023 sales. But on Friday, Novartis said patients in the single-arm Phase 3b APPULSE-PNH study saw their average hemoglobin levels improve from baseline over 24 weeks after switching from Soliris or Ultomiris, meeting the primary endpoint. “These data reinforce our confidence in Fabhalta, the first and only oral monotherapy currently available for the treatment of adults with PNH, to provide meaningful hemoglobin improvement, regardless of previous treatment experience,” David Soergel, global head of Novartis’ cardiovascular, renal and metabolism development unit, said in a news release . The company declined to provide specifics, though a spokesperson told Endpoints News that the data will be presented at a medical meeting in 2025. The results build on data from Novartis’ APPLY-PNH study. At last year’s American Society of Hematology conference, Novartis said that 94% of patients who switched from C5-inhibitors to Fabhalta in the study’s extension period achieved transfusion avoidance, and “mean hemoglobin levels increased to near-normal.” In that group, Novartis said mean hemoglobin levels were 9.1 g/dL at the start of the extension period, which is lower than patients’ average levels at the start of the APPULSE-PNH study. Soliris and Ultomiris are infusions, while Fabhalta is approved as a twice-daily oral pill. Novartis CEO Vas Narasimhan said in January that the company saw “very positive early launch signals” for Fabhalta, but added that he expected a “modest ramp for this brand given the dynamics within the PNH market,” according to an AlphaSense transcript. Fabhalta generated $44 million in third-quarter 2024 sales. In August, it was granted an accelerated approval in adults with primary immunoglobulin A nephropathy (IgAN), a rare autoimmune disease that affects the kidneys.