SMA Newsroom

Aug 30, 2023

Community update from Roche

We are pleased to share the latest community update from Roche with several topics that are relevant to our SMA Community.

1. European Commission (EC) approves risdiplam for babies under two months old with SMA 

Following on from an earlier community letter on the CHMP opinion, Roche confirms that risdiplam’s EU label has been extended to include infants from birth to below two months.

At SMA Europe, we are pleased that our hard work engaging with all stakeholders to ensure that everyone who can benefit from treatment is included as early as possible is bearing fruit. The fact that another treatment option is now available for newborns is very good news for our community.

Please see the press release here, including statement issued by Nicole Gusset, President & CEO of SMA Europe.

2. Risdiplam EU Summary of Product Characteristics (SmPC) updated to include JEWELFISH - Please find a link to the updated SmPC here

JEWELFISH is an open-label study to investigate the safety, tolerability and pharmacokinetics/pharmacodynamics of risdiplam in adult and paediatric patients with SMA (previously treated with nusinersen, onasemnogene abeparvovec, or Roche’s previous IMPs RO6885247 or olesoxime) (NCT03032172).

The EMA has adopted the following new information in the risdiplam EU SmPC, based on the 24-months primary analysis of JEWELFISH:

  • Removal of the warning stating that there is no efficacy data in patients previously treated with gene therapy
  • Inclusion of safety results, and the conclusion that the safety profile of risdiplam in previously treated patients is consistent with the profile for people who have not previously been treated
  • Inclusion of pharmacokinetic and pharmacodynamic results from the study
  • Inclusion of patient characteristics and exploratory efficacy results

These changes were agreed with the EMA earlier this year, but have only recently become public in the updated SmPC.

3. MANATEE: recruitment for Cohort D commences & SMA Europe’s feedback was reflected in (link).

MANATEE is a two-part, seamless, randomised, placebo-controlled, double-blind study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of GYM-329 in combination with risdiplam in patients with SMA (NCT05115110). This cohort is now recruiting. Enrollment remains ongoing in the ambulant cohorts.

Cohort D will recruit at 33 sites in 11 countries. As of today, open European sites are located in Poland, Netherlands, Belgium and Italy and it is hoped that additional sites will open in Spain, Croatia, Portugal and the UK. As each site becomes ready to recruit, they will become visible on, ForPatients, and other local trial sites.   

Based on further feedback from SMA Europe, some changes in the trial description have been made to better explain the inclusion criteria (age & ambulation status) for each cohort.