rare-disease

ARPA-H's new THRIVE program is funding seven teams to build reusable gene-editing platforms for rare pediatric diseases, aiming to move beyond one-patient-at-a-time therapies like the one made for infant KJ Muldoon.

An antisense drug stabilized walking speed in a 54-patient pivotal trial; the FDA accepted Ionis's NDA for Priority Review with a target action date of September 22, 2026.

The FDA granted accelerated approval to Hepcludex (bulevirtide-gmod) on May 22, 2026, making it the first treatment cleared for chronic hepatitis delta virus infection in the United States.

An AAV9 gene-regulation therapy delivered durable, dose-dependent seizure control and early signs of neurodevelopmental rescue through 52 weeks, just as the pivotal study begins dosing.

AstraZeneca's CALYPSO Phase III hit its composite primary endpoint with 31.1% of eneboparatide patients normalizing serum calcium and dropping supplements at week 24 versus 5.9% on placebo, with benefits sustained through a 52-week extension.

A 20% subcutaneous immunoglobulin paired with hyaluronidase delivered the same antibody exposure as HYQVIA in half the volume, clearing Takeda's path to global filings in fiscal 2026.

Italian drugmaker Chiesi will pay $27 a share for KalVista Pharmaceuticals to acquire Ekterly, the first oral on-demand treatment for hereditary angioedema, in a deal expected to close in the third quarter.

Regeneron's one-time intracochlear gene therapy for OTOF-related deafness cleared the FDA 61 days after BLA filing, the first gene therapy product approved under the Commissioner's National Priority Voucher pilot program.

Axsome Therapeutics' AXS-05 faces its FDA verdict on April 30 for Alzheimer's disease agitation, backed by four Phase 3 trials but complicated by one failed study.

Denali's tividenofusp alfa becomes the first FDA-approved biologic engineered to cross the blood-brain barrier, breaking a long rare-disease rejection streak and validating a transport-vehicle platform that could unlock CNS access for many more drugs.

Early-phase data in 81 children with Dravet syndrome showed large seizure reductions and manageable safety issues as a phase 3 trial moves ahead.

Prime Medicine is pursuing accelerated approval for PM359, an autologous prime editing therapy that restored immune function in both patients treated for chronic granulomatous disease, marking the first clinical use of prime editing technology.