News

The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. Roche provides an update on the clinical development of risdiplam

    risdiplam

    Summary

    • 12-month data from the pivotal SUNFISH Part 2 study submitted to the FDA in February, leading to a 3 month review extension of the New Drug Application (NDA)
    • On track to submit a Marketing Authorisation Application (MAA) to the European Medicines Agency in mid-2020
    • Regulatory filings have been submitted in Brazil, Chile, Indonesia, Russia, South Korea and Taiwan.

    In November 2019, the U.S. Food and Drug Administration (FDA) granted Priority Review for risdiplam with a decision for approval expected by 24th May 2020. As agreed with the FDA, in February of this year Roche 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 our International Scientific and Clinical Congress on Spinal Muscular Atrophy in February 2020.

    The FDA recently informed Roche that the volume of additional data submitted by the company requires a three-month review extension of the New Drug Application (NDA) of risdiplam. Roche therefore expects a decision from the FDA by 24th August 2020.

    Additionally, regulatory filings have been submitted in Brazil, Chile, Indonesia, Russia, South Korea and Taiwan. Furthermore, a filing submission in China is imminent and Roche is currently on track to submit a Marketing Authorisation Application (MAA) to the European Medicines Agency in mid-2020, as well as in other countries around the world.

    Roche would like our community to know that members of its staff are working expeditiously to make risdiplam available to all patients that can benefit from the treatment, as soon as possible.  They thank everyone in the community, especially the patients and families who have participated in the risdiplam clinical development programme.

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