News

The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. Roche initiates phase 2 clinical trials for RG7916 in Types 1, 2 & 3 SMA.

    Roche initiates phase 2 clinical trials for RG7916Roche initiates phase 2 clinical trials for RG7916. The company will be testing RG7916, an orally available drug that aims to correct the splicing of SMN2, the “backup gene”, in types 1, 2 & 3 SMA, at sites in the US, Canada, Australia and some European countries.

    There will be two trials. One will be a multi-centre, randomised, double-blind, placebo-controlled study consisting of two parts: an exploratory dose finding part (Part 1) of RG7916 for 12-weeks and a confirmatory part (Part 2) of RG7916 for 24-months

    One trial (Firefish) will be enrolling approximately 50 infants with SMA type I at sites across the US as well as in France. The second trial (Sunfish) will be enrolling individuals aged 2-25 with SMA type II or III. This trial will recruit nearly 200 individuals across 34 sites in the US, Canada, Australia and some European countries (Belgium, France, Germany, Italy, Spain, Sweden, Switzerland and the UK).

    Patients who participated in the Moonfish study with RG7800 may soon be eligible to enrol into a third study, Jewelfish (BP39054), an open-label study with RG7916, provided they meet the inclusion criteria. More details will be shared as the study plans develop.

    Please read the community statement provided by Dr. Sangeeta Jethwa, MD.