Roche reports new advances for people with rare neuromuscular disorders

Swiss pharma giant Roche (ROG: SIX) today announced data from across its growing neuromuscular portfolio at the World Muscle Society (WMS) Virtual Congress 20–24 September 2021.

The presentations included additional results from the RAINBOWFISH study, evaluating the efficacy and safety of Evrysdi (risdiplam) in babies with pre-symptomatic spinal muscular atrophy (SMA) from birth to six weeks of age and data supporting the continued clinical investigation of gene therapy, SRP-9001, in Duchenne muscular dystrophy (DMD).

Driven by its novel and convenient mode of administration, analysts have forecast that Evrysdi could generate up to $2 billion at peak global sales, competing with existing treatments such as Biogen’s (Nasdaq: BIIB) Spinraza (nusinersen) and Novartis’ (NOVN: V) Zolgensma (onasemnogene abeparvovec).

“These new data for Evrysdi may help extend the potential benefits of this medicine to the youngest SMA patients. Also, the data from SRP-9001 have helped to optimize the design of the upcoming Phase III trial for DMD,” said Dr Levi Garraway, Roche’s chief medical officer and head of global product development.”

At WMS 2021, data from the ongoing open label RAINBOWFISH study were presented. Four out of five of those treated with Evrysdi for at least 12 months achieved standing and walking independently within the World Health Organization windows for healthy children. In addition, all five babies maintained the ability to swallow and were able to feed exclusively orally after 12 months of treatment.