The Food and Drug Administration is expected to rule by late spring next year on whether to approve a new drug for spinal muscular atrophy.
Swiss drugmaker Roche said Monday that the agency had accepted and granted priority review to its new drug application for risdiplam for SMA, setting a target action date of May 24, 2020. The drug is a survival motor neuron-2, or SMN-2 splicing modifier, designed to work by increasing and sustaining SMN protein levels throughout the central nervous system and peripheral tissues of the body.
The company’s regulatory submission was based on results from the Phase II/III FIREFISH and SUNFISH trials. FIREFISH enrolled 48 infants aged 1-7 months with Type 1 SMA, while SUNFISH enrolled 231 children and adults aged 2-25 with Type 2 and Type 3 SMA. The application includes data from the Part 1 portions of each study, as well as from the Part 2 confirmatory portion of SUNFISH.
“The FIREFISH and SUNFISH trials were designed to represent the real world spectrum of people living with SMA and include many people previously underrepresented in clinical trials,” Roche Chief Medical Officer Levi Garraway said in a statement. “We look forward to working closely with the FDA to explore broad access to risdiplam for all individuals in the community who might benefit.”
Roche said Nov. 11 that Part 2 of SUNFISH had produced positive data, meeting its primary endpoint of a change from baseline in the Motor Function Measure-32 scale after one year of treatment with the drug. In May, it presented data from the first parts of both trials at the American Academy of Neurology’s annual meeting. Among the 17 infants in FIREFISH who received the dose of drug used in the confirmatory portion, 41.2 percent were able to sit without support for at least five seconds, while 64.7 percent were able to sit with or without support, and 52.9 percent achieved upright head control after 12 months of treatment.
The drug will face competition particularly from two established players in the SMA market, namely Biogen’s Spinraza (nusinersen) and Novartis’ gene therapy Zolgensma (onasemnogene abeparvovec-xioi). Zolgensma received approval from the FDA earlier this year, particularly for infants with SMA, while Spinraza is approved for children as well as adults.
Photo: Roche
