Marty Makary

FDA
Favicon Fierce Pharma

FDA approves leucovorin without clinical trial—but not for autism

The FDA has used real-world data to approve leucovorin to treat cerebral folate deficiency in patients who have a confirmed variant in the FOLR1 gene.
Angus Liu , Darren Incorvaia Mar 10, 2026 9:00am
Prasad

CBER Director Vinay Prasad to depart FDA—again

Mar 6, 2026 5:30pm
FDA stop sign

Senior FDA official fires back at uniQure amid controversy

Mar 5, 2026 3:33pm
opening door to brick wall

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

Feb 26, 2026 3:50pm
The White House

Trump met with Makary before FDA’s Moderna walkback: report

Feb 19, 2026 11:30am
shards of a disc in the air

Disc's rare disease drug hit by FDA rejection

Feb 13, 2026 5:00pm