The USFDA has approved viltolarsen injection as newer gene-targeting drug therapy for Duchenne Muscular Dystrophy (DMD) . The drug is indicated for patients with a confirmed mutation in the DMD gene. The drug targets the dystrophin gene that is susceptible to exon 53 skipping which is known to cause gene mutations, resulting in progressive muscle deterioration. The USFDA has given accelerated approval to the drug based on the data from two clinical studies. The study was conducted on 32 male patients who had genetically confirmed DMD.
The study assessed the levels of dystrophin from baseline to week-25. The study reported increased levels of dystrophin from 0.6 % at baseline to 5.9 % at week-25 in patients treated with viltolarsen. Upper respiratory tract infection, injection site reaction, cough, and fever were found to be the most common adverse effects. The trials also suggested…
