FDA delays decision on Roche's spinal muscular atrophy drug risdiplam to review more data

Roche said Tuesday that the FDA has extended its priority review of the company's filing for risdiplam in spinal muscular atrophy (SMA) from May 24 originally to August 24. According to Roche, the extension stems from the recent submission of additional data by the company, including results from the pivotal SUNFISH Part 2  study, which is says could help ensure access to risdiplam for "a broad range of people living with the condition, if approved."

The Swiss drugmaker said its FDA filing now includes 12-month efficacy and safety data from SUNFISH Part 2 study in people aged two to 25 years with Type 2 or 3 SMA (for related analysis, see KOL Views Results: Leading neurologist sees risdiplam as a real problem for Spinraza in SMA). "Given the volume of additional data submitted, the FDA requires more time for review," the company said. Chief medical officer Levi Garraway remarked "we strongly believe in the potential of risdiplam as a new therapeutic option," and added that the company's goal is to bring orally administered SMN2 splicing modifier "to infants, children and adults living with SMA as quickly as possible."

Meanwhile, Roche said it also submitted regulatory applications for risdiplam in Brazil, Chile, Indonesia, Russia, South Korea and Taiwan. It indicated that a filing in China is "imminent" and that it is currently on track to submit a marketing application to the European Medicines Agency in mid-2020, as well as other international markets.

If approved, analysts have forecast that risdiplam could eventually top $1 billion in annual sales, competing against Biogen's and Ionis Pharmaceuticals' Spinraza (nusinersen), which was approved by the FDA in 2016, as well as Novartis' gene therapy Zolgensma (onasemnogene abeparvovac-xioi), which was cleared by the US regulator last May and is marketed at $2.1 million per patient. Risdiplam, also known as RG7916, is being developed in partnership with PTC Therapeutics and the SMA Foundation.


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