Rare Disease Therapeutics

Close up shot of the target from a dart game
Pharma

Alkermes’ Lumryz, from $2.4B Avadel buy, hits phase 3 mark in IH

If approved, Lumryz would add more pressure to Jazz’s blockbuster Xywav, which counts idiopathic hypersomnia as its main growth driver.
Angus Liu May 12, 2026 11:45am
two arrows pointing different directions

FDA to reconsider rejected cell therapy. Could uniQure be next?

May 7, 2026 10:40am
Human kidney anatomy

In another win for rare disease, Travere nabs FDA’s first FSGS nod

Apr 13, 2026 6:25pm
UCB sign

Fast-rising UCB scores FDA nod for rare disease drug Kygevvi

Nov 4, 2025 11:36am
FDA stop sign

FDA snubs Ultragenyx gene therapy over manufacturing issues

Jul 14, 2025 10:16am
Capitol

Orphan Cures Act dropped from Senate reconciliation bill

Jun 17, 2025 11:30am