5 Rare Diseases That Now Have Their First FDA-Approved Treatments

AJMC February 28, 2025
Giuliana Grossi

Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, signifying progress in closing treatment gaps for rare disease.

Since Rare Disease Day last year, several rare diseases have gained their first-ever FDA-approved drug, signifying progress in closing treatment gaps for these conditions.

1. Niemann-Pick Disease, Type C: Arimoclomol (Miplyffa)

Patients with Neimann-Pick disease, type C (NPC), received their first FDA-approved treatment in September.¹ Children with the rare genetic condition live for an average of 13 years and experience progressive neurological symptoms. In combination with miglustat, arimoclomol aims to mitigate the neurological symptoms of NPC with an indication for patients at least 2 years old.

Days after Zevra Therapeutics’ arimoclomol approval, IntraBio’s levacetylleucine (Aqneursa)...

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