rare diseases

Capitol
Pharma

Orphan Cures Act dropped from Senate reconciliation bill

The Orphan Cures Act would allow drugs with multiple rare disease indications to be exempt from Medicare price negotiations.
Angus Liu Jun 17, 2025 11:30am
Sanofi

Sanofi buys Blueprint for $9.1B, its largest deal in 7 years

Jun 2, 2025 11:09am
rare diseases
Favicon Fierce Biotech

Stealth CEO questions FDA rejection of rare disease drug

May 29, 2025 2:27pm
Alnylam Pharmaceuticals dancer AHP video

Alnylam recruits dancer with rare disease for Givlaari promo

May 28, 2025 2:04pm
CRL RTF rejection

Savara's lung disease asset hit with FDA refuse-to-file letter

May 27, 2025 10:35am
Novo Nordisk

Novo heads to regulators after Sogroya posts positive results

May 13, 2025 9:50am