Launches of First Therapies Approved for Spinal Muscular Atrophy and Friedreich's Ataxia Will Revolutionize Treatment and Drive Growth of These Rare Disease Markets

BURLINGTON, Mass., Oct. 25, 2016 /PRNewswire/ -- Decision Resources Group finds that the anticipated launches of the first therapies for the treatment of spinal muscular atrophy (SMA) or Friedreich's ataxia (FA) will transform treatment of these diseases and lead to their markets expanding dramatically. Medical practice in SMA and FA is currently dominated by supportive care or symptomatic treatment. However, the complete lack of efficacious treatment options translates into high unmet need and represents an as-yet untapped and lucrative market opportunity for drug developers.

Ionis/Biogen's antisense oligonucleotide nusinersen, which targets the underlying cause of SMA, will be the first agent ever approved for this disease. Based on efficacy data from clinical trials, interviewed pediatric neurologists expect rapid and widespread use of nusinersen. Assuming high-premium pricing, the launch of nusinersen, expected in 2017, will be a key driver of market expansion.

Following the anticipated label expansion of Horizon Pharma's Actimmune (interferon gamma-1b), the FA market is forecasted to grow significantly over the next ten years. However, interviewed neurologists' perceived limitations of Actimmune, including its unclear mechanism of action, variable effect on frataxin protein levels, and modest preservation of neurological function, could constrain its uptake and allow competitors to challenge Actimmune's position.

Other key findings from the Niche & Rare Disease Landscape & Forecast reports entitled Spinal Muscular Atrophy and Friedreich's Ataxia:

• In both SMA and FA, first-to-market therapies will likely benefit patients, but commercial opportunity remains for novel therapies that can offer improvements in efficacy, safety, and delivery. Further, physicians expect new therapies with different targets and mechanisms of action would be used in combination with existing agents.  
• During the second half of the 2015-2025 study period, nusinersen may face competition from AveXis's investigational gene therapy AVXS-101, which has shown tremendous promise in an ongoing clinical trial. AVXS-101 could potentially be delivered in a single intravenous administration, in contrast to nusinersen's triannual intrathecal administration, and may be curative.
• According to interviewed experts, gene therapy will also transform the FA treatment landscape, possibly negating the need for drug treatment. However, gene therapies being developed by Agilis Biotherapeutics, Pfizer, and RaNA Therapeutics are in preclinical testing and unlikely to launch during the study period.

Comments from Decision Resources Group Analysts, Ian Love, Ph.D., and Tara M. Stewart, M.A., Ph.D., respectively:

• "SMA is a serious and often fatal rare disease usually afflicting children, and there are no useful therapies available to treat this devastating condition. Thanks to the continued commercial interest in developing treatments for severe rare diseases, SMA patients may soon have access to effective therapies. Nusinersen and AVXS-101 have the potential to revolutionize care for SMA patients."
• "Despite the lack of competing brands entering the market in the near term, Actimmune's high U.S. cost may be an obstacle for its rapid adoption among patients with FA. If Actimmune can show that it delays disease progression or improves neurological function over a year or more, prescribers, patients, and payers are likely to accept its very high price."

For more information on purchasing these reports, please email [email protected].

About Decision Resources Group
Decision Resources Group offers best-in-class, high-value data, analytics and insights products and services to the healthcare industry, delivered by more than 1,000 employees across 14 global locations. DRG provides the pharmaceutical, biotech, medical device, financial services and payer industries with the tools, insights and advice they need to compete and thrive in an increasingly complex and value-based marketplace. DecisionResourcesGroup.com.

Media contact:
Ashton Verzone
Decision Resources Group
[email protected]
781-993-2690

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