Risdiplam - Roche's oral SMA drug

Promising results from SMA FIREFISH trail

Roche have announced positive results from Part 2 of its study. The trial evaluated the drug risdiplam in babies aged one to seven months old with Type 1 spinal muscular atrophy (SMA). It showed the drug to be safe and effective in increasing muscle function.

Data from the study will be presented at an upcoming medical congress.

For more information, read the press release from Roche.

14 January 2020

Roche announces global compassionate use plan for risdiplam

The announcement means that doctors will be able to apply for the potential treatment on behalf of their patients with SMA Type I. Roche plan to expand access to people with SMA Type II later in the year.  

Roche UK will operate this compassionate use plan until risdiplam has been approved via the Early Access to Medicines Scheme (EAMS).  This scheme, governed by the UK Medicines and Healthcare Products Regulatory Agency (MHRA), is designed to ensure people can access promising new medicines at the earliest stage.  

Risdiplam is an oral drug that increases SMN protein levels, the protein absent in people with SMA. The drug works by targeting the SMN2 gene. It is currently an investigational medicine as it has not yet been approved in the UK or elsewhere.  

Parents of children with SMA Type I should speak to their child’s doctor about gaining access to the treatment.  

For more information you can read the community letter from Roche and the UK statement from Roche UK (via the SMA UK website). 

26 November 2019

SMA drug granted Priority Review by FDA

Roche have announced that the U.S. Food and Drug Administration (FDA) have accepted the New Drug Application (NDA) and granted Priority Review for the drug risdiplam. We can expect a decision on approval from the FDA by 24 of May, 2020. Roche anticipate filing a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) by the middle of 2020.

For more information read the press release from Roche. 

11 November 2019

Promising results from SMA SUNFISH trial

Roche have today announced positive news from Part 2 of its study. The trial evaluated the drug risdiplam in people aged two to 25 years with Type 2 or 3 spinal muscular atrophy (SMA). It showed the drug to be safe and effective in increasing muscle function.

Data from the study will be presented at an upcoming medical congress.

For more information, read the press release from Roche.