Trientine tetrahydrochloride

PARIS, Dec. 12, 2025 /PRNewswire/ -- Orphalan, an international pharmaceutical company specializing in the development and commercialization of orphan drugs, has acquired Orphelia Pharma, a company committed to treating rare and severe pediatric diseases, notably in neurology and oncology. This strategic transaction strengthens Orphalan's position as a European leader with a global commercial footprint in rare diseases, expanding its therapeutic portfolio. Key highlights of the acquisition include: Expansion of our capabilities in rare neurological and oncological pediatric diseases Greater reach and improved access to medicines for children and adults with rare conditions A unique platform combining scientific excellence, agility, and global commercialization By bringing together complementary expertise, the companies are now positioned to accelerate innovation and improve access to transformative treatments for patients around the world. As they enter this new stage, their teams remain fully committed to ensuring continuity of care and supporting their partners and patients. Dr. Naseem Amin, Chief Executive Officer of Orphalan said: "The acquisition of Orphelia Pharma reinforces our mission: to provide innovative and accessible treatments to patients living with rare diseases, wherever they are. "Together, we are building a unique platform that brings together scientific excellence, agility, international development and commercial capabilities to advance care for patients of all ages suffering from orphan diseases." Dr. Hugues Bienaymé, Founder and Deputy Chief Executive Officer of Orphelia Pharma, added: "At Orphelia Pharma, we have dedicated ourselves to developing safe and effective pediatric medicines that address major unmet medical needs in rare diseases. "Joining Orphalan will allow us to increase the impact of our treatments and make them available more rapidly to the children who need them most." Gilles Alberici, Chief Executive Officer of Orphelia Pharma, commented: "Bringing together our two complementary companies marks an important milestone, both for our organizations and for the broader community of patients living with rare diseases." About Orphalan Founded in 2011, Orphalan SA is an international pharmaceutical company dedicated to the development and commercialization of orphan drugs. Originating from a collaboration with AGEPS and Hôpital Lariboisière, Orphalan developed Cuprior®, a treatment for Wilson's disease, now authorized in 38 countries and marketed in 28. Orphalan's ambition is to reduce diagnostic delays and make innovative orphan drugs accessible to as many patients as possible through a patient-centered approach and close collaboration with healthcare professionals. Orphalan's shareholders include Bpifrance, Advent, and the management team. About Orphelia Pharma Orphelia Pharma is a pharmaceutical company based in Paris and Lyon, specializing in the development and commercialization of medicines for rare and severe pediatric diseases. With one authorized medicine in the European Union ( Kigabeq®) and a second under regulatory review ( Kizfizo®), Orphelia Pharma has established regional distribution agreements across Europe and other regions and collaborates with academic and industrial partners to advance research in rare diseases. Further information: Bien Commun Advisory M. Benedict Brodrick +33.6.76.14.02.02 [email protected] SOURCE Orphalan 21% more press release views with Request a Demo

New Delhi: In a bid to address the steep cost of imported rare disease therapies , the government is considering modifications to its Promotion of Research and Innovation in Pharma MedTech Sector ( PRIP ) scheme to support domestic development, said Amit Agrawal, Secretary, Department of Pharmaceuticals. Delivering the special guest address at the FICCI Rare Disease Conference 2025, Agrawal said, “Rare diseases in India are as significant as any other public health issue. While we are known as the pharmacy of the world, the need of the hour is to invest in research and development." “Under this vision we launched PRIP and we are at very final stages of making some tweaks to make it better and one of the key focus is the inclusion of rare diseases and orphan drugs,” he said adding that the government is considering rare diseases as a strategic focus with a higher policy support under the scheme.” Launched in 2023 with an allocation of Rs 5,000 crore, PRIP is a Research-Linked Incentive (RLI) initiative aimed at driving innovation in pharmaceuticals and MedTech. Of the scheme’s total outlay Rs 700 crore is earmarked under component ‘A’ for establishing 7 CoEs at NIPER and the rest Rs 4,250 crore is under component ‘B’ for promoting drug development in areas like New Chemical Entities, Complex generics including biosimilars, medical devices, stem cell therapy, orphan drugs, Anti-microbial resistance etc.” Agrawal also highlighted progress under the Production-Linked Incentive (PLI) scheme, noting that eight rare disease drugs—including Trientine (Wilson’s disease), Nitisinone (Type 1 Tyrosinemia), Eliglustat and Miglustat (Gaucher’s), Hydroxyurea (sickle cell anemia), Sapropterin (Phenylketonuria), Sodium Phenylbutyrate, and Carglumic Acid (Hyperammonemia) are now produced domestically. Stating that PLI scheme has brought down treatment costs significantly, he noted, “the annual cost of therapy for Gaucher’s disease has dropped from around Rs 3.6 crore to Rs 3 to 6 lakhs." The production of these drugs began in 2023 following the addition of pharmaceuticals in the PLI scheme with a financial outlay of ₹15,000 crore for the production period of fiscal year 2022-2023 to 2028-29. However, patient groups argue that the therapies currently covered are largely off-patent small molecules, whereas the the greater hurdle for patients is posed by advanced therapies such as large molecules, biologics, and gene therapies , that are still under patent protection. For instance, a disease-modifying therapy Risdiplam marketed by Swiss drugmaker Roche under the brand name Evrysdi for spinal muscular atrophy (SMA)–a neuromuscular disease–costs Rs 60–80 lakh annually, while Novartis’ one-time gene therapy Zolgensma (onasemnogene abeparvovec) entails Rs 14–16 crore. India’s National Policy for Rare Diseases (NPRD), introduced in 2021, has established 13 CoEs—including the latest at AIIMS Patna—to provide treatment and counselling for 63 listed rare diseases. According to the Ministry of Health and Family Welfare, as of August 2024, over 1,100 patients have benefited. Yet advocacy groups stress that the Rs 50 lakh financial support cap is inadequate for advanced therapies , and access to centers remains limited. By Abhijeet Singh ,