Roche’s headquarters in Basel, Switzerland

The Food and Drug Administration has delayed its review of a drug developed by Roche to treat spinal muscular atrophy, the company said.

The Swiss drugmaker said Wednesday that the FDA had pushed back the deadline for completing its review of risdiplam following Roche’s submission of new data, which included 12-month safety and efficacy data from the registration-directed study of the drug, SUNFISH Part 2. The FDA had originally planned to decide whether or not to approve the drug by May 24, when it accepted Roche’s approval application in November 2019, but will now have until Aug. 24.

Shares of Roche were down 3.5% on the Swiss Exchange when the market closed Wednesday and have fallen further since then. Roche is leading the drug’s development, under a partnership with PTC Therapeutics and the SMA Foundation.

“We strongly believe in the potential of risdiplam as a new therapeutic option and recognize that unmet need remains in the treatment of SMA,” Roche chief medical officer Levi Garraway said in a statement. “We are working closely with the FDA to support the review of risdiplam. Our goal is to bring this therapy to infants, children and adults living with SMA as quickly as possible.”

Other approved therapies for SMA include Biogen’s Spinraza (nusinersen) and Novartis’ gene therapy Zolgensma (onasemnogene abeparvovec-xioi).

In a note to investors Wednesday, Cowen analyst Joseph Thome wrote that a delay for the FDA’s decision date is “unfortunate” given the enthusiasm for the drug on the part of patients with SMA and physicians who treat it. However, he wrote, there is a continued expectation that risdiplam will receive FDA approval this year. Moreover, he added, the timing for Part 2 of SUNFISH and another pivotal trial, FIREFISH, and the real-world population in the former, Roche’s decision is prudent as it guarantees a broad label for the drug. Thome wrote that at the firm’s healthcare conference in March, a panelist said he expected risdiplam to see uptake in Type 2 and Type 3 SMA patients with severe scoliosis and thus unable to take Spinraza, with the market being shared evenly between risdiplam and the aforementioned two drugs in the coming years.

Roche announced positive data from the Part 2 portion of SUNFISH in November, among patients aged 2-25 with Type 2 or Type 3 SMA, saying that the study met its primary endpoint of showing a change from baseline in the Motor Function Measure 32, or MFM-32 scale after one year of treatment with the drug. FIREFISH is a two-part trial in infants with Type 1 SMA.

Photo: Roche

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