The U.S. Food and Drug Administration (FDA) has announced the accelerated approval of viltolarsen (Viltepso), developed by NS Pharma, for the treatment of Duchenne Muscular Dystrophy (DMD).
Last year, The FDA approved golodirsen (Vyondys 53), developed by Sarepta Therapeutics, for DM, according to the Medscape Medical News.
Dr. Billy Dunn, head of the Office of Neuroscience of the FDA’s Center for Drug Evaluation and Research, said, “The FDA is committed to fostering drug development for serious neurological disorders like Duchenne muscular dystrophy.”
The FDA approval of viltolarsen provides “an important treatment option for Duchenne muscular dystrophy patients with this confirmed mutation,” Dr. Dunn added.
Viltolarsen, an antisense oligonucleotide, promotes the production of functional dystrophin by masking exon 53 in the DMD gene. The drug was evaluated in two clinical studies that involved 32 DMD patients.
In one study, 50 percent of the patients who received viltolarsen had an increase in dystrophin production.
According to the FDA, the increase in dystrophin production is “reasonably likely to predict clinical benefit but a clinical benefit of the drug has not been established.”
The FDA approved Viltolarsen under its accelerated approval pathway, a process of drug approvals for serious and life-threatening diseases, which generally offers a meaningful advantage over the current treatments.
The FDA requires NS Pharma to do a large trial to confirm the clinical benefits of viltolarsen as part of its accelerated approval. If the trial does not verify the drug’s clinical benefits, the FDA may withdraw the approval.
Some of the most common adverse events of viltolarsen include upper respiratory tract infection, cough, fever, and injection-site reaction.
The clinical studies did not observe kidney toxicity in patients, although some antisense oligonucleotides potentially cause kidney toxicity, including severe glomerulonephritis. The FDA advised, “Kidney function should be monitored in patients taking Viltepso.”
