FDA’s Expedited Review and Industry Moves on Rare Disease and Gene Therapy

The FDA has altered its review plans by canceling an advisory meeting for Biohaven’s troriluzole but signaled an impending late-year approval decision, a positive indicator amid historical precedents. Stealth BioTherapeutics’ rare Barth syndrome drug, elamipretide, received a fast-tracked review with a decision due by late September, reflecting regulatory urgency following multiple prior rejections and advocacy pressure. Concurrently, Ionis Pharmaceuticals achieved FDA approval for its antisense drug Dawnzera to prevent hereditary angioedema attacks, marking its second FDA nod in under a year. In gene therapy news, Gilead Sciences acquired Interius BioTherapeutics for $350 million to strengthen in vivo CAR-T capabilities, joining a pharma race to simplify and expand cell therapies. Meanwhile, Rocket Pharmaceuticals resolved FDA concerns allowing resumption of its gene therapy trial for Danon disease at a modified dose, restoring momentum in cardiovascular gene therapy development.