The FDA (US Food and Drug Administration) accepted Roche's application file on Risdiplam, gave priority review. Final approval is expected on May 24, 2020. Reference data are based on 12-month results of 'Firefish' and 'Sunfish' clinical trials evaluating Risdiplam in SMA type 1 and 2–3 patients. Accordingly, the FDA will complete its evaluation on the Risdiplam drug in 6 months instead of 10 months. If the drug is approved, it will be the first home drug for SMA patients orally. Roche is expected to begin procedures with the European Medicines Agency in the coming weeks.
Risdiplam is an investigational, oral drug that is systematically distributed and designed to increase SMN protein levels in the central nervous system (CNS) and body. It is designed to make the SMN2 gene better support motor neurons and muscle function and help produce a more functional SMN protein. Risdiplam has been tested in different groups from infants to adults.
Risdiplam is also being studied in people who have previously had a different SMN enhancing therapy. Persons participating in this trial must first go through a "washout" period - that is, they should stop taking the previous medication and wait for it to leave the body before enrolling.
Risdiplam is a daily oral medication. It can also be given through a g tube.
Risdiplam should be taken throughout the life of the individual.