Genentec announced the launch of Evrysdi (risdiplam) for Spinal Muscular Atrophy Market

 

Growing initiatives to enhance the treatment option for rare diseases is propelling the growth of the Spinal Muscular Atrophy Market. For instance, in 2017, the U.S. FDA granted an orphan drug license to RG7916, a joint development program by Roche, SMA Foundation, and PTC Therapeutics for the treatment of SMA. Similarly, the Orphan Drug Designation was also granted to Cytokinetics’ CK-2127107 by U.S. FDA in May 2017. Moreover, increasing initiatives by people and non-profit organizations for creating awareness regarding SMA is another key factor augmenting the growth of the market. For instance, in January 2018, the National Institute for Health and Care Excellence (NICE) invited Biogen to submit a single technology appraisal for Spinraza to enable NHS funding. NICE and NHS, U.K. are developing a managed access agreement for a long-term reimbursement plan for Biogen’s Spinraza.

1. In January 2019, Biogen announced that the first spinal muscular atrophy (SMA) patient had been treated in its Phase IV RESPOND study evaluating the efficacy and safety of Spinraza (nusinersen) in patients with a suboptimal clinical response to Novartis’ Zolgensma (onasemnogene abeparvovec)

2. In May 2019, AveXis, a Novartis company, announced the US Food and Drug Administration (FDA) has approved Zolgensma® (onasemnogene abeparvovec-xioi) for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA)

3. In August 2020, Genentech, a Roche company, announced the U.S. Food and Drug Administration (FDA) had approved its Evrysdi (risdiplam) for spinal muscular atrophy (SMA)

Spinal Muscular Atrophy, also known as Spinal Neuromuscular Atrophy (SMAA), is a degenerative disease of the central nervous system that results in atrophy of the spinal musculature and other degenerative changes at the level of the spinal cord and surrounding tissues. Symptoms of spinal muscular atrophy vary significantly depending on the form of SMA that is present. Milder cases of SMA have less serious symptoms, which may not be apparent until the child has reached an advanced age or is well into middle age. In severe cases, the affected children may be completely unable to perform normal daily functions. The exact onset of SMA varies greatly, but it is normally noted during childhood and is more commonly diagnosed in boys than girls.

North America is expected to lead the global Spinal Muscular Atrophy Market and this is attributed to the increasing approval for the launch of a novel drug for the treatment of SMA. For instance, in August 2020, the U.S. Food and Drug Administration approved Evrysdi (risdiplam) to treat patients two months of age and older with spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement. This is the second drug and the first oral drug approved to treat this disease.

See Full Report@ https://bit.ly/3jAmKHb