The FDA has accepted new drug application (NDA) and granted priority review for risdiplam (Genentech, San Francisco, CA), an investigational, survival motor neuron-2 (SMN-2) splicing modifier for spinal muscular atrophy (SMA). The FDA is expected to make a decision on approval by May 24, 2020.
If approved, risdiplam, an orally administered liquid, would be the first at-home administered medicine for people living with SMA. Risdiplam is designed to increase and sustain SMN protein levels both throughout the central nervous system and peripheral tissues of the body.
In addition to the FIREFISH (NCT02913482) and SUNFISH (NCT02908685) studies included in the NDA submission, risdiplam is being studied in a broad clinical trial program in SMA, with participants ranging from newborns to 60 years old, and includes participants previously treated with SMA therapies.
Ilka Kleffner, MD; Catharina C. Gross, PhD; Marius Ringelstein, MD; Jörg Rehrmann, MD; Markus Kraemer, MD; and Jan Dörr, MD
Harold P. Adams Jr., MD
Shruti Bhandari, MD; Rohit Kumar, MD; Megan Nelson, MD; Donald Miller, MD; and Brian J. Williams, MD