accelerated approval

hearing audio waves
Favicon Fierce Pharma

Regeneron ushers in new era with inaugural gene therapy approval

The FDA today approved the company’s Otarmeni, the first gene therapy for a rare genetic form of hearing loss that affects about 50 newborns per year.
Darren Incorvaia Apr 23, 2026 3:22pm
MPS Families Carry Coffin to FDA

Rare disease advocates stage 'funeral' at FDA, make 4 demands

Mar 18, 2026 5:25pm
Three stacked cups with coffee spilling out of top one

Inovio trims team as trouble brews at FDA for rare disease drug

Mar 13, 2026 10:03am
opening door to brick wall

Senate hearing takes aim at FDA's rare disease review process

Feb 26, 2026 3:50pm
FDA

FDA outlines draft policy on MRD for accelerated approval

Jan 21, 2026 2:55pm
sarepta building
Favicon Fierce Pharma

Sarepta, despite key trial flop, will seek full nod for DMD meds

Nov 4, 2025 7:56am