Spinal muscular atrophy (SMA) being a rare genetic neuromuscular disorder results in progressive muscle weakness and atrophy. It affects the part of the nervous system that controls voluntary muscle movement.
Spinal Muscular Atrophy Current therapeutic management revolves around supportive and multidisciplinary care with a focus on reducing complications and improving quality of life. The current market holds alternatives for symptomatic relief with curative therapy being a high unmet need.
Approaches for management of Spinal Muscular Atrophy revolves aroundNutrition, Respiratory Care Assessment, Orthopedic Care, Rehabilitation, along with few medications. Proper nutrition provides several benefits such as improved growth, better breathing, improved motor functions, and a better quality of life to those living with Spinal Muscular Atrophy. Respiratory Care Assessment, on the other hand, recommends evaluation of cough effectiveness, observation of breathing, and monitoring gas exchange. The anticipatory respiratory care includes management techniques, respiratory support, nutrition and hydration management and Routine immunisations.
Respiratory muscle weakness is a significant problem in several forms of SMA. Availability of portable, effective ventilation devices has helped with the management of Respiratory muscle weakness associated with different types of SMA. Rehabilitation is another crucial aspect for the patients of spinal muscular atrophy. The care centre constituting both orthopaedic and neurological departments provides multidisciplinary therapy to improve the remaining motor capabilities.
Very few medications are available to aid the patients of spinal muscular atrophy. The only approved and commercialised drug available for the management of Spinal Muscular Atrophy pediatric and adult patients is Spinraza (Nusinersen) developed by Biogen. The U.S. FDA approved this ‘antisense oligonucleotide’ medicine drug was in December 2016.
The past decade saw many advances in the development of Spinal Muscular Atrophy Drugs. At present we have a dozen companies involved in bringing expertise to lead to a cure.
Spinal Muscular Atrophy pipeline possesses multiple potential drugs in late and mid-stage developments to be launched in the near future. Zolgensma (AVXS-101), which belongs to Novartis, is a proprietary gene therapy of a one-time intravenous treatment for SMA Type 1 and a one-time intra-thecal treatment for SMA Type 2 designed to prevent further muscle degeneration. It is currently in Phase III stage of development and has been awarded Orphan Drug Designation, Breakthrough Therapy Designation, and Fast Track Designation by the U.S. Food and Administration (FDA).
Owned by Roche, Risdiplam (RG7916) is an investigational, oral medicine that is systemically distributed and designed to increase SMN protein levels in the CNS and throughout the body. It is in phase II/III trial. A collaborative product of Astellas Pharma and Cytokinetics, Reldesemtiv (CK- 2127107) is in phase II clinical development. The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CK-2127107 for the potential treatment of spinal muscular atrophy (SMA). Branaplam, in phase I/II investigational studies, is a Novartis’s mid-stage product. It is an orally available drug which aims to correct the splicing of SMN2, the “back-up” gene, thereby increasing the amount of SMN protein levels.
Among all these upcoming years, Zolgensma, Risdiplam, Reldesemtiv are expected to hold the largest market share after their launch in the market in 2019, 2022 and 2022 respectively.
