The SMA Market: Assessing The Unknowns

Treatment Progress Leads To New Challenges In Spinal Muscular Atrophy

The introductions of Spinraza and Zolgensma in SMA offer new insights into how to address neurodegenerative diseases. But more real world evidence is needed.

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Uncertainty Ahead In SMA • Source: Shutterstock

The landmark FDA approval of Novartis AG’s Zolgensma (onasemnogene abeparvovec-xioi) in May 2019 shook the biopharma world in several ways including its price ($2.1 million per dose) and, as important, the very small data set on which FDA primarily based its decision – an ongoing open-label single arm trial of 21 infantile-onset patients with spinal muscular atrophy (SMA) under two years old. Biogen Inc.’s Spinraza (nusinersen) had already been approved in SMA in December 2016. As disease-modifying therapies, these compounds are a rarity in the field of neuromuscular diseases of genetic origin. They are also at the core of a fascinating, ongoing real-world case study in how the natural course of a disease can change rapidly. How companies’ SMA drug development and market access strategies evolve, both in terms of new disease-modifying agents and supportive therapies that address residual symptoms, could become a blueprint for other neuromuscular diseases like Duchenne’s Muscular Dystrophy (DMD) or Huntington’s Disease.

Spinal muscular atrophy (SMA) is a genetic disease caused by an absence of or defect in the SMN1 gene, which encodes the survival motor neuron (SMN) protein. A back-up gene,...

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