- 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
Frequently Asked Questions
The market is segmented based on Global Niemann-Pick Market Segmentation, By Disease Type (Type A, Type B, Type C, and Type E), Diagnosis (Physical Examination, Skin Biopsy, Biochemical Testing, and Genetic Testing), Treatment (Miglustat, Physical Therapy, and Others), Age Group (Perinatal, Early Infantile, Late Infantile, and Adult) End-Users (Clinics, Hospitals, Diagnostic Centers, and Others), Distribution Channel (Direct Tenders, Hospital Pharmacy, Retail Pharmacy, Online Pharmacy, and Others) – Industry Trends and Forecast to 2032
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The Global Niemann Pick Market size was valued at USD 923.81 USD Million in 2024.
The Global Niemann Pick Market is projected to grow at a CAGR of 5.5% during the forecast period of 2025 to 2032.
The market report covers data from the U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E., South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.
