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RISDIPLAM - Genentech

Basics, expectations and next steps.


What is Genentech?

Genentech is a biotechnology company, subsidiary of Roche.

What is Risdiplam?

Risdiplam is a small molecule drug developed by Genentech.

What does RISDIPLAM do?

Administered orally, it is shown to increase the amount of SMN protein made by the SMN2 gene.



August 7th, 2020

RISDIPLAM (Evrysdi) has been approved by the United States Food and Drug Administration (FDA) as the third treatment for Spinal Muscular Atrophy, suitable for the use in patients with SMA of all types from ages 2 months and up.

Feb 6, 2020 - Risdiplam "showed significant improvement in motor function" in a placebo controlled trial consisting of people aged 2 to 25 with type 2 or 3 Spinal Muscular Atrophy!

Feb 6, 2020 - Risdiplam is now available to types 1 and 2 via Early Access Program (EAP).


The drug is currently being evaluated by the FDA, under priority review designation, as the third treatment for SMA and the second treatment for ALL SMA types and ages. The final decision on approval is expected by May 24, 2020.


  • For the first time ever, adults (neglected and underrepresented population in clinical trials) were both included in the study and had demonstrated significant improvement.

This is extremely positive departure from how clinical trials have been designed in the past. Given that we have huge and growing adult population, exclusion from trials means lack of data. Lack of data means problems with accessing drug post approval. Problems with accessing drug post approval means adults are prevented from receiving standard of care. Not receiving standard of care is unacceptable!

  • For the first time ever, stabilization of the disease is used as the meaningful & measurable goal of the drug therapy.

With symptomatic patients, especially those who have lived with SMA for years, the goal of treatment, first and foremost, is to stop the progression of the disease. Any gains are a bonus. This is the first study that translated this knowledge and shared understanding of what drug therapy should do into actual measures and outcomes. And they did it with success!

  • For the first time ever, the study included all types and ages - from pre symptomatic babies to 60 year old adults!

We are thrilled that Roche/Genentech pioneered this important innovation in framework for conducting drug trails in SMA, and we hope that all current and future studies will follow the suite!

Our expectations regarding approval:

Our expectation is that Risdiplam will be approved to for all.

The study design was comprehensive and inclusive, endpoints were achieved in SUNFISH & FIREFISH and Risdiplam is being reviewed by the FDA under the priority review designation.

Risdiplam (developed in collaboration with the SMA Foundation and PTC Therapeutics) is a small molecule used to increase the amount of SMN protein made by the SMN2 gene, commonly referred to as back up genes. Risdiplam is an orally given medication that prevents motor neuron degeneration and preserves muscle function. It is a liquid taken daily by mouth or feeding tube if required.

Risdiplam was tested in 4 trials, starting in December 2016, in SMA types 1, 2 & 3.

FIREFISH trial was for type 1 infants between 1 and 7 months of age.

JEWELFISH trial was a combination of type 1, 2 & 3 between the age of 6 months to 60 years old in SMA patients who have received Nusinersen or other SMN targeting therapy such as Olesoxime.

SUNFISH was a two part, double-blind, placebo controlled pivotal study in people aged 2 to 25 years old with SMA type 2/3.

RAINBOWFISH trial was for pre-symptomatic babies with SMA up to 6 weeks old.


Risdiplam is distributed throughout the body, raising the levels of SMN protein in various organs, not just the central nervous system (brain and spinal cord). The Risdiplam clinical development was sponsored by Roche/Genentech, and it included a wide variety of individuals from types 1-3, and multiple age groups, from birth to 60 years old. The study was conducted world-wide.

RISDIPLAM Trial Timeline

  • September 2017: Start of part 2 of SUNFISH study

  • October 2017: Interim results of Part 1 of the SUNFISH study indicate Risdiplam is well tolerated at all doses

  • March 2018: Start of pivotal part of FIREFISH for babies with Type 1 SMA

  • March 2018: Start of FIREFISH for babies with Type 1 SMA

  • May 2019: Update on FIREFISH & SUNFISH presented at the 2019 AAN conference showing promising results

  • November 2019: Risdiplam meets primary endpoint in pivotal SUNFISH trial in people with type 2 or 3 SMA

  • November 2019: FDA grants priority review to Risdiplam for the treatment of SMA. The FDA’s decision is expected by May 24, 2020

  • December 2019: Risdiplam meets primary endpoint in Firefish

  • January 2020: Roche announces global pre-approval access (compassionate use) plans for Risdiplam

  • August 7th, 2020 RISDIPLAM (Evrysdi) is approved by the USA FDA.

This page will be updated as new developments occur. Check back often.

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