Rare disease treatments, side by side

74 FDA-approved drugs across 17 conditions, 16 of them rare, 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 drugs: Sandostatin LAR Depot, Somatuline Depot, Signifor LAR, Somavert, Palsonify, Mycapssa. Plus 1 in development.

ATTR Amyloidosis (Transthyretin Amyloidosis)

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

Fabry Disease

3 approved drugs: Fabrazyme, Elfabrio, Galafold. Plus 1 in development.

Familial Chylomicronemia Syndrome

2 approved drugs: Tryngolza, Redemplo.

GastroparesisNot rare

1 approved drug in 2 forms: Reglan, Gimoti. Plus 1 in development, 1 approved only outside the U.S. and 1 turned down by the FDA.

Gaucher Disease

5 approved drugs: Cerezyme, VPRIV, Elelyso, Cerdelga, Zavesca. Plus 1 in development.

Hemophilia A

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

Hemophilia B

4 approved drugs: Alhemo, Hympavzi, Qfitlia, Hemgenix.

Hereditary Angioedema

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

Huntington Disease

3 approved drugs: Xenazine, Austedo and Austedo XR, Ingrezza and Ingrezza Sprinkle.

IgA Nephropathy

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

Immune Thrombocytopenia

6 approved drugs: Nplate, Promacta, Doptelet, Tavalisse, Alvaiz, Wayrilz. Plus 1 in development and 1 approved only outside the U.S..

Myasthenia Gravis

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

NTRK Fusion Cancer

3 approved drugs: Vitrakvi, Rozlytrek, Augtyro. Plus 2 in development and 2 approved only outside the U.S..

Sickle Cell Disease

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

Spinal Muscular Atrophy

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

Wilson Disease

4 approved drugs: Cuprimine, Depen, Syprine, Cuvrior, Galzin. Plus 2 in development.