Risdiplam: First Orally Administered Drug for Spinal Muscular Atrophy got Approval
Spinal Muscular Atrophy (SMA) is a rare genetic disease affecting motor nerve cells in spinal cord, resulting in progressive muscle wasting and weakness. It is the second major cause of Neuromuscular disease and is seen commonly in children. SMA is caused by a mutation in the survival motor neuron gene 1 (SMN1). The prevalence of SMA in Infants is the ratio of 1: 11000.
Males are more affected than females and also the clinical features are more seen men. Very typically adults do develop SMA, which is Type 4, which begins later 21 years of age. Though simple muscle atrophy can be reversed with balanced diet and exercise, SMA is not so easy to treat.
Though cure of SMA is not in existence as of, researchers across the world working on development of new treatments which can enhance the quality of life. Apart from the First FDA approved Drug, Nusinersen there exists a gene therapy medication, Onasemnogene abeparvovec for the treatment of SMA.
On August 7, 2020 the US FDA has approved the first Orally Administered drug for the treatment of SMA, Risdiplam developed by Genentech Inc. The acts as motor neuron 2-directed RNA splicing modifier. The efficacy of it was tested in the clinical trials for the treatment of patients with infantile-onset and later-onset SMA.
The drug was accorded the status of Orphan Drug and also attained Fast Track Designation with Priority Review by the FDA.
