Dear SMA Community,
As part of our ongoing partnership and following your request to receive important information about the risdiplam clinical development program, we wanted to update you on the status of regulatory filings and reviews of risdiplam by the U.S. Food and Drug Administration.
In November 2019, the U.S. Food and Drug Administration (FDA) granted Priority Review for risdiplam with a decision for approval expected by May 24, 2020. As requested by the FDA, in February of this year we submitted additional data which included 12-month data from the pivotal SUNFISH Part 2 study, the only placebo-controlled study ever undertaken in people aged 2-25 years with Type 2 or 3 SMA. These data were presented at the 2nd International Scientific and Clinical Congress on Spinal Muscular Atrophy in February 2020.
The FDA recently informed us that the volume of additional data submitted by Genentech/Roche has resulted in a 3-month review extension of the New Drug Application (NDA) of risdiplam. We therefore expect a decision by the FDA no later than August 24, 2020. The FDA confirmed the extension is not related to any current efficacy or safety concerns of risdiplam nor to the COVID-19 pandemic. We want to reassure you that we are working collaboratively with the FDA and will do everything in our power to support the review process in order to make risdiplam available to all patients that can benefit from the treatment, as soon as possible.
We appreciate that the SMA community is facing a number of new and unexpected challenges during these unprecedented times. Please know that we are working tirelessly around the clock to advance treatment options for families impacted by SMA. We want to thank everyone in the community, especially the patients and families who have participated in the risdiplam clinical development program. It is because of you that we are here today, and we are incredibly grateful.