Roche har delat en ny uppdatering av Risdiplam (RG7916), det nya namnet heter risdiplam, som har nått ett antal milstolpar.
Risdiplam är en SMN2 splicing modifierare som ges dagligen genom mun eller g-rör som en vätska och distribueras överallt i kroppen. Risdiplam har utformats för att hjälpa SMN2-genen att producera mer SMN-protein, eftersom människor med SMA har minskad nivå av SMN-protein. Risdiplam är en forskning-molekyl som utvecklas i samarbete med PTC Therapeutics och SMA Foundation.
Detta är en ny studie som kommer att utvärdera risdiplam vid pre-symptomatisk barn.
Next steps for JEWELFISH Study
The JEWELFISH study is currently ongoing in people aged 12 to 60 years and who have previously received an SMN2 targeting therapy as part of a clinical study. We will broaden the inclusion criteria and increase the number of participants in JEWELFISH to include those who
· received nusinersen as part of their regular medical care for SMA
· have SMA Type 1
· are 6 months of age or older.
Further plans for JEWELFISH include eligibility for people who are still actively taking part in the OLEOS study (Olesoxime long term safety and effiacacy study).
These updates to JEWELFISH will be made subject to approval by Health Authorities and Ethics Committees.
Next steps for SUNFISH Study
Information from Part 1 has allowed us to confirm the dose of risdiplam to be investigated in Part 2. Part 2 will assess the efficacy and safety of risdiplam. Enrolment into Part 2 of SUNFISH is almost complete, approximately 168 participants will be included. The main analysis of the efficacy and safety of risdiplam will take place after all participants receive one year of treatment.
Next steps for FIREFISH Study
Part 1 has been completed. Information from Part 1 has allowed us to confirm the dose of risdiplam to be investigated in Part 2, which will assess the safety and effiacy of risdiplam. Participants of Part 1 are receiving risdiplam and are still enrolled in FIREFISH as part of an open-label extension phase.
Part 2 is underway and will assess the safety and efficacy of risdiplam, including an additional 40 babies between the ages of 1 and 7 months. The main analysis of efficacy and safety of risdiplam will occur after all babies have completed one year of treatment, and efficacy will be determined by the number of babies that can sit without assistance after one year of treatment.