After a long regulatory road that included a complete response letter in May, Stealth Biotherapeutics Inc. finally got its Barth syndrome drug across the finish line, with the U.S. FDA granting accelerated approval to Forzinity (elamipretide HCl) to improve muscle strength in those with the ultra-rare pediatric mitochondrial cardioskeletal disease.

Nucleoside reverse transcriptase translocation inhibitors (NRTTIs) are a novel class of antiretroviral agents that inhibit HIV replication by targeting the viral reverse transcriptase enzyme and specifically blocking its translocation step during DNA synthesis, a critical process in the viral replication cycle.

When it won U.S. FDA accelerated approval more than eight years ago, Intercept Pharmaceuticals Inc.’s Ocaliva (obeticholic acid) was viewed as a breakthrough, becoming the first new treatment in 20 years for rare, progressive liver disease primary biliary cholangitis (PBC) and, for several years, the only second-line treatment for PBC patients failing to respond to ursodeoxycholic acid. More recently, however, Ocaliva has faced regulatory and safety stumbles, with Intercept now voluntarily pulling the farnesoid X receptor activator from the U.S. market.