The United States’ Food and Drug Administration (FDA) has granted priority review for the New Drug Application (NDA) for risdiplam (RG7916) for the treatment of SMA.
The FDA’s decision will be announced on 24th May 2020. If approved, risdiplam, an orally administered liquid, would be the first at-home administered medicine for people living with SMA.
A Priority Review status is given for drugs that, if approved, would represent significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. In addition, the FDA is expected to review this application within 6 months instead of the standard 10 months.
The FDA's acceptance of the NDA is an important step towards making risdiplam available to SMA patients in the U.S. This NDA filing includes results from a broad SMA patient population, including type 1, type 2 and type 3 SMA patients demonstrating improvements in motor functions and developmental milestones and a compelling safety profile.
Risdiplam is an investigational medicine being studied in a broad range of patients with SMA from birth to 60 years of age. It is designed to provide a sustained increase in SMN protein centrally and peripherally, through daily dosing and is being evaluated for its potential ability to help the SMN2 gene produce more functional SMN protein throughout the body. Risdiplam is being studied in a clinical trial for patients with type 1 SMA, called FIREFISH, in pre-symptomatic babies, RAINBOWFISH, in people with SMA Type 1, 2 or 3, aged 6 months to 60 years, who have previously been treated with SMA therapy, gene therapy or olesoxime and in SUNFISH, a placebo-controlled study in people aged 2-25 years with Type 2 or 3 SMA.
In addition to the FDA, Roche will work with health authorities around the world to determine appropriate pathways for filing a new drug application for risdiplam. In Europe, they anticipate filing a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) by the middle of 2020.
For both regulatory submissions, Roche is seeking a broad label for the treatment of SMA in paediatric and adult patients. They collaborate with regulatory agencies throughout their clinical development programme and during preparation for any application submission and/or review.
Roche is grateful to the whole community, especially the patients and families who participate in their studies, as well as the patient groups around the world who have supported and worked with them to achieve this milestone.