Rare disease treatments, side by side

50 FDA-approved drugs across 10 rare conditions, each compared on how it is given, how often, who it is for, what its pivotal trial showed and what its label warns about, with the Phase 3 and under-review drugs listed below them. Every number comes from the current prescribing label or the trial record, and the sources are on each page.

Acromegaly

6 approved: Sandostatin LAR Depot, Somatuline Depot, Signifor LAR, Somavert, Palsonify, Mycapssa. 1 in development.

ATTR Amyloidosis (Transthyretin Amyloidosis)

5 approved: Onpattro, Amvuttra, Vyndaqel / Vyndamax, Attruby, Wainua. 1 in development.

Hemophilia A

4 approved: Alhemo, Hympavzi, Hemlibra, Qfitlia. 1 in development.

Hemophilia B

4 approved: Alhemo, Hympavzi, Qfitlia, Hemgenix.

Hereditary Angioedema

5 approved: Takhzyro, Orladeyo, Andembry, Ekterly, Dawnzera.

IgA Nephropathy

6 approved: Tarpeyo, Filspari, Fabhalta, Vanrafia, Trutakna, Voyxact. 5 in development.

Myasthenia Gravis

7 approved: Soliris, Vyvgart, Rystiggo, Zilbrysq, Ultomiris, Uplizna, Imaavy.

NTRK Fusion Cancer

3 approved: Vitrakvi, Rozlytrek, Augtyro. 4 in development.

Sickle Cell Disease

5 approved: Casgevy, Lyfgenia, Droxia (also Siklos), Adakveo, Endari. 1 in development.

Spinal Muscular Atrophy

5 approved: Isembyld, Spinraza, Zolgensma, Itvisma, Evrysdi.