LYSOSOMAL STORAGE DISEASES THERAPEUTICS MARKET ANALYSIS

Leading Companies

Major players operating in the global lysosomal storage diseases therapeutics market include Shire plc, Pfizer, Inc., Sanofi, BioMarin Pharmaceutical Inc., Actelion Ltd., Raptor Pharmaceutical Corp., Protalix Biotherapeutics Inc., Quest Diagnostics, and Amicus Therapeutics, Inc.

Global Lysosomal Storage Diseases Therapeutics Market : Key Developments

 On February 9, 2022, Avrobio, a U.S.-based pharmaceutical company, announced that its gene therapy for rare lysosomal storage disorder showed potential durability in the first three patients more than one year after infusion. The gene therapy is given for cystinosis which is treated with cysteamine, a common treatment that reduces the buildup of cystine crystals in the kidneys for people with the inherited disease. The first three patients on the gene therapy with AVR-RD-04, remain off oral cysteamine between 12 and 26 months after the treatment infusion. A fourth patient received the therapy in November 2021. None of the patients have experienced adverse events due to the treatment, but have had mild or moderate side affects due to stem cell mobilization, underlying disease, preexisting conditions or myeloablative condition.

On March 1, 2023, Sanofi, a France-based pharmaceutical company, on occasion of Rare Disease Day announced that it will launch new products for rare disease in India by end of 2023 or beginning of next year. The company has also received a recommendation from the Subject Expert Committee to import and market two new products in India, Nexviazyme (Avalglucosidase alfa powder) and Xenpozyme (Olipudase alfa powder) for Pompe disease and Niemann-Pick disease (ASMD) respectively.