The US Food and Drug Administration (known as the FDA) has accepted regulatory documentation for a drug called “Risdiplam”, as the most innovative therapeutic treatment for SMA types 1,2 and 3, and has been prioritized for evaluation of the impact that manifests on patients. The documentation is based on the results of the FIREFISH and SUNFISH clinical studies, and during a 12-month period, specialist were working on the results of the efficacy provided by the mentioned experimental drug. This is a special procedure, and its approval will be binding in the US and countries that automatically accept the FDA criteria. Also, health company “Roche” is expected to begin regulatory procedures with the European Medicines Agency (EMA) .
SMA Serbia Association is looking forward to great news for all our patients, families and participants in the SUNFISH study, as we are one step closer to the latest cure for all 3 types of SMA.