Community Statement from Genentech: FDA Grants Priority Review to Risdiplam for Spinal Muscular Atrophy

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Genentech, a member of the Roche Group, today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted Priority Review for risdiplam, an investigational survival motor neuron-2 (SMN-2) splicing modifier for spinal muscular atrophy (SMA). If approved, risdiplam, an orally administered liquid, would be the first at-home administered medicine for people living with SMA. Below please find a community statement from Genentech.


Dear SMA community,

As part of our ongoing partnership, and following your request to receive important and timely information about the risdiplam clinical development program, we would like to provide an update regarding the regulatory status of our investigational medicine, risdiplam.

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 decide 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. We are grateful for the opportunity to collaborate with the regulatory agency throughout our clinical development program and during our preparation for application submission and review.

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.


J.P. Sacksteder, on behalf of the Genentech and Roche SMA Team

Frequently Asked Questions and Answers:

What is Risdiplam?

Risdiplam is an investigational, orally administered (liquid) survival motor neuron-2 (SMN-2) splicing modifier for SMA. It is designed to increase and sustain SMN protein levels throughout the central nervous system and peripheral tissues of the body. It is being evaluated for its potential ability to help the SMN2 gene produce more functional SMN protein throughout the body. Genentech and Roche lead the clinical development of risdiplam as part of a collaboration with the SMA Foundation and PTC Therapeutics. Genentech would commercialize the medicine in the United States if approved.

What are the FIREFISH and SUNFISH studies?

FIREFISH is an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants aged one to seven months. The primary objective of Part 1, which evaluated initial efficacy as an exploratory endpoint, was to assess the safety profile of risdiplam in infants and determine the dose. Part 2 is a pivotal, single-arm trial evaluating risdiplam in 41 infants with Type 1 SMA for 24 months, followed by an open-label extension. SUNFISH is a two-part, double-blind, placebo-controlled pivotal clinical trial in children and young adults (2-25 years old) with Type 2 or 3 SMA. Part 1 determined the dose for the confirmatory Part 2, and evaluated efficacy as an exploratory endpoint. SUNFISH Part 2 is the largest placebo-controlled trial to date evaluating treatment for people with Type 2 or 3 SMA. SUNFISH Part 2 recently met its primary endpoint of change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year of treatment with risdiplam, compared to placebo. Results will be presented at an upcoming medical congress.

Are there plans for pre-approval access or compassionate use?

We understand the urgency of addressing the needs and daily challenges of people with SMA and their families, and the concerns they have about accessing investigational medicines as soon as possible. We are working with regulatory authorities to explore the feasibility of options for compassionate use and pre-approval access. We are committed to come back to the community and share updates once information is available.

About risdiplam

Risdiplam is an investigational, oral medicine that is systemically distributed and designed to increase SMN protein levels in the central nervous system (CNS) and throughout the body. It is designed to help the SMN2 gene produce more functional SMN protein, to better support motor neurons and muscle function. Roche and Genentech are leading the clinical development of risdiplam in collaboration with the SMA Foundation and PTC Therapeutics.


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