Roche has presented newest data on safety and efficacy of risdiplam, the next drug being developed to treat all forms of spinal muscular atrophy. The data reinforces earlier observations that risdiplam appears to be safe and effective across the full spectrum of the disease.
The company also announced the dates of regulatory submission in the US and EU.
New safety and efficacy data of risdiplam made a splash at the 24th World Muscle Society Congress in Copenhagen last week.
Safety data was good – there have been no serious safety concerns about risdiplam in any of the ongoing clinical trials.
Efficacy data was also encouraging, to say the least. When looking at 21 babies who had completed 16 months of treatment in the FIREFISH trial, not only no child required tracheostomy or permanent ventilation but the vast majority have experienced major functional improvements. Fourteen out of seventeen infants (that is, 82%) who had been on the higher dose of risdiplam reached a CHOP-INTEND score of at least 40!
Here is an amazing video of a type 1 baby girl who has been taking risdiplam for a year as a part of FIREFISH trial. Our thanks go to Zosia’s family for sharing.
In parallel, children, teenagers and young adults with SMA types 2 and 3 who took part in Part 1 of the SUNFISH trial experienced “a clinically meaningful increase in total MFM32 score”. The increase was independent of age and functional status at the start of the trial, which means that meaninfful functional improvements were observed across all ages and SMA types. On the biochemical level, risdiplam led to a median two-fold increase in blood SMN protein level in those patients just after 4 weeks of treatment. As expected, the increase was sustained for 12 months and more.
In the 45 analysed participants of the JEWELFISH trial – which enrolled people who had previously been treated with drugs like olesoxime and Spinraza – risdiplam similarly led to a two-fold increase in blood SMN protein over 12 months of treatment compared to day one. This is an interesting proposition – that risdiplam may offer an additional benefit over Spinraza. We wait to see further long-term data from JEWELFISH.
Roche has also announced that their presymptomatic clinical trial RAINBOWFISH recruited the first patient last August, even as there are still many places left in the trial for presymptomatic newborns.
All this data compels the company to file for expedited regulatory approval of risdiplam. An application will first be submitted to the US Food and Drug Administration in late 2019. Folloing that, in early 2020, a complete risdiplam dossier will be submitted to the European Medicines Agency for approval on our continent.
TreatSMA is liaising with Roche on expediting access to risdiplam in the UK.
