Responding to our request for an update on risdiplam FDA filing, Fani Petridis from Roche wrote to us:
Dear members of the SMA community,
As part of our ongoing partnership and following your request to receive timely information about the risdiplam clinical development program, we are pleased to share with you that the U.S. Food and Drug Administration (FDA) has accepted the filing of the New Drug Application (NDA) for risdiplam for the proposed use in people living with Spinal Muscular Atrophy (SMA). The FDA has granted the application a Priority Review, which means that the agency is currently expected to review the application within 6 months instead of the standard 10 months. The FDA is expected to make a decision on approval by May 24, 2020.
The risdiplam NDA submission incorporates 12-month data from the dose-finding Part 1 sections of the FIREFISH and SUNFISH studies, as well as data from the confirmatory Part 2 of SUNFISH. In addition to the FDA, we will work with health authorities around the world with the objective to determine appropriate pathways for filing a new drug application for our investigational medicine, risdiplam. In Europe, we anticipate to file a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) by the middle of 2020. For both regulatory submissions, we are currently seeking a broad label for the treatment of SMA in paediatric and adult patients. We are grateful for the opportunity to collaborate with regulatory agencies throughout our clinical development program and during our preparation for any application submission and/or review.
Advances in medical science are only meaningful when they reach the people who need them. As a company that is driven by the potential to improve the lives of patients and families touched by SMA, we are very excited to take this next step forward toward making risdiplam available to all patients that can benefit from the treatment.
We thank everyone in the community, especially the patients and families who participate in our studies, as well as the patient groups around the world who have supported and worked with us to achieve this milestone. Our journey to develop treatments for people with SMA continues to be inspired by you. We look forward to providing further updates about our program as they become available.
If you have any questions about this update, please do not hesitate to contact me.
Fani Petridis, on behalf of the Roche Global SMA Team
Global Patient Partnership Director, Rare Diseases
We thank the Roche Global SMA Team for the update. It is disappointing to learn that the risdiplam approval timeline in Europe will be so much behind the US, likely stretching into 2021. We acknowledge the fact that the European Medicines Agency prefers to base its recommendations on larger datasets than the FDA, and data collection takes time as the risdiplam trials are still ongoing. Nevertheless we must remind Roche and the European Medicines Agency that there is a high unmet medical need among the SMA community in our country and that, given its high therapeutic potential, risdiplam must be made available to people without delay.