34. Viltepso

FDA Building
In greenlighting NS Pharma’s Viltepso, the FDA has kept its Duchenne muscular dystrophy approval standard consistent, albeit controversial. (FDA)

Active ingredient: Viltolarsen
Disease: Duchenne muscular dystrophy
Peak sales estimate: N/A
Approved: Aug. 12
Company: NS Pharma

The scoop: In greenlighting NS Pharma’s Viltepso, the FDA has kept its Duchenne muscular dystrophy (DMD) approval standard consistent, albeit controversial. Viltepso is meant for patients with a mutation of the DMD gene that’s amenable to exon 53 skipping, the same population as Sarepta Therapeutics' Vyondys 53. Both drugs’ approvals are based on data showing they can increase the levels of dystrophin, a crucial protein DMD patients fail to properly produce. The approval for Vyondys 53, driven by those biomarker data, previously fueled controversy even within the FDA. Questions have been focused on whether the increase in dystrophin can translate into actual improvement in symptoms. NS Pharma is running a confirmatory phase 3 trial to validate its efficacy. – Angus Liu

34. Viltepso