New FDA Approvals for Rare Disease Therapeutics

Ionis Pharmaceuticals secured FDA approval for Dawnzera (donidalorsen), an antisense therapy preventing attacks in hereditary angioedema (HAE), a rare genetic disease. The drug markedly reduces attack rates and supports less frequent self-administration dosing. This marks Ionis’ second recent FDA approval, reflecting growing momentum in rare disease treatment innovation. Concurrently, Stealth BioTherapeutics received FDA acceptance of a revised submission for elamipretide, targeting Barth syndrome, with an expedited review timeline indicated.