Spinal Muscular Atrophy Market, by Type (Type I, Type II, Type III, and Type IV), by Treatment (Gene Therapy and Disease-modifying drugs), by Age Group (Infant and Adult), by Route of Administration (Oral and Injectables), by Distribution Channel (Hospital Pharmacies, Retail Pharmacies, and Online Pharmacies), and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) - Size, Share, Outlook, and Opportunity Analysis, 2024 - 2031

Published In : Mar 2024

Code : CMI1920

Pages :156

Formats :

Excel and PDF

Industry : Pharmaceutical

Market News

Recent Developments

In June 2023, Biogen Inc., a multinational biotechnology company, announced new SPINRAZA (nusinersen) data that will address crucial issues for the spinal muscular atrophy (SMA) community. The data were presented at the SMA Research & Clinical Care Meeting hosted by Cure SMA in Orlando, U.S. SPINRAZA is licensed in over 60 countries to treat newborns, children, and adults with spinal muscular atrophy (SMA). More than 14,000 people throughout the world have received SPINRAZA as a cornerstone of care for SMA.

In March 2023, Novartis, a multinational pharmaceutical corporation, presented new data demonstrating the transformational and long-term benefits of Zolgensma (onasemnogene abeparvovec), a one-time gene therapy for spinal muscular atrophy (SMA). The most recent findings from two Long-Term Follow-Up (LTFU) trials, LT-001 and LT-002, revealed that Zolgensma continues to be efficacious and durable across a wide range of patient demographics, with an overall benefit-risk profile that remains favorable.

In February 2023, Biohaven Ltd., a pharmaceutical company, announced that it had received Fast Track designation from the U.S. Food and Drug Administration (FDA) for taldefgrobep alfa, a new anti-myostatin adnectin for the treatment of spinal muscular atrophy (SMA). Fast Track designation enables critical new pharmaceuticals to reach patients sooner by allowing for more regular discussions with the FDA and expedited review of a drug that treats a serious ailment and meets an unmet medical need.

In August 2020, PTC Therapeutics, Inc., a pharmaceutical company, announced that the U.S. FDA had approved Evrysdi (risdiplam), the first at-home, orally administered treatment for spinal muscular atrophy (SMA) in adults and children aged 2 months and older. Evrysdi demonstrated clinically relevant improvements in motor function and achievement of developmental milestones in two trials involving patients aged two months and up and across all degrees of illness severity, including types 1, 2, and 3.

Increasing product launches are expected to drive the market growth during the forecast period.

Increasing product launches are expected to drive the market growth over the forecast period. For instance, in June 2021, F. Hoffmann-La Roche Ltd, a multinational healthcare company, launched prescription medicine Evrysdi, indicated for the treatment of spinal muscular atrophy (SMA) in adults and children aged 2 months and older, in India.