- In October 2024, Theranexus, a biopharmaceutical company focused on rare neurological diseases, partnered with Synerlab Group, a contract development and manufacturing organization (CDMO), to manufacture the first commercial batches of an exclusive pediatric formulation of miglustat. In this partnership, Theranexus developed this formulation for the treatment of Niemann-Pick disease type C (NPC), and the European Medicines Agency (EMA) approval will be sought, with commercialization expected by 2026
- In September 2024, IntraBio Inc., a leader in the development of drugs for rare neurodegenerative diseases, announced that the U.S. Food and Drug Administration (FDA) approved AQNEURSA (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in both adults and pediatric patients weighing ≥15 kg. In this approval, AQNEURSA becomes the only FDA-approved stand-alone therapy for NPC
- In September 2024, Zevra Therapeutics received approval from the U.S. Food and Drug Administration (FDA) for Miplyffa (arimoclomol) to treat Niemann-Pick disease type C (NPC). In this case, the capsules, for patients aged two years and older, are used in conjunction with miglustat to address neurological symptoms of NPC. Miplyffa also received a rare pediatric disease priority review voucher (PRV)
- In September 2024, Orsini announced that Zevra Therapeutics selected them as the exclusive specialty pharmacy partner for Miplyffa™ (arimoclomol), the first FDA-approved treatment for Niemann-Pick disease type C (NPC), a rare and progressive neurodegenerative disease
