US FDA approved another drug for rare Duchenne Muscular Dystrophy
Updated: Aug 16, 2020
The United States Food and Drug Administration has approved Viltepso (viltolarsen) for treating patients suffering from Duchenne muscular dystrophy (DMD) who have a mutation in the DMD gene that is amenable to exon 53 skipping. FDA has reported that this is the second drug approved for patients suffering from this mutation.
Approximately 8% of the patients with DMD have a mutation that is amenable to exon 53 skipping.
The approval has been granted to NS Pharma.
The drug approval is based on the two clinical trials enrolling 32 male patients and had genetically confirmed DMD. Improvement in the dystrophin levels was reported in one of the trial which enrolled sixteen patients, dystrophin level improved from 0.6% to 5.9% at week 25.
The clinical benefit of the drug has not been established.
The drug has received the accelerated approval, the company has to conduct a clinical trial confirming the efficacy of the product. The current study is planned to assessed the efficacy of Viltepso, if it can improve the time to stand in patients suffering from DMD and have the specific mutation.
The product has received Priority Review designation in addition to the accelerated approval.
About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy, a common type of muscular dystrophy is a genetic disorder which causes the muscle deterioration and weakness. The disease is caused because of the mutation in the DMD gene which results in the absence of dystriophin, protein responsible for keeping the muscle intact. Symptoms are reported at the age of three to five years which further wosen with age. The disease usually affects the males and rarely affects females.
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