Rare Disease Drug Approvals
CDER’s ARC Program | Center for Drug Evaluation and Research
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Rare Disease Drug Approvals
2025 | 2024 | 2023 | 2022 | 2021
This page describes select recent rare disease drug approvals announced by a CDER or FDA communication. To search all FDA-approved drugs, visit Drugs@FDA.
- Kygevvi (doxecitine and doxribtimine)
FDA approves 1st drug for thymidine kinase 2 deficiency, a very rare mitochondrial disease
FDA News Release – November 3, 2025 - Forzinity (elamipretide)
FDA Grants Accelerated Approval to First Treatment for Barth Syndrome
FDA News Release – September 19, 2025 - Fabhalta (iptacopan)
FDA approves first treatment for adults with complement 3 glomerulopathy, a rare kidney disease, to reduce proteinuria
FDA News Release – March 20, 2025 - Ctexli
FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease
FDA News Release – February 21, 2025
- Alhemo
FDA Approves Drug to Prevent or Reduce the Frequency of Bleeding Episodes for Patients with Hemophilia A with Inhibitors or Hemophilia B with Inhibitors
CDER Notable Approval – December 20, 2024 - Tryngolza (olezarsen)
FDA Approves Drug to Reduce Triglycerides in Adult Patients with Familial Chylomicronemia Syndrome
CDER Notable Approval – December 19, 2024 - Crenessity (crinecerfont)
FDA Approves New Treatment for Congenital Adrenal Hyperplasia
FDA News Release – December 19, 2024 - Attruby (acoramidis)
FDA Approves Drug for Heart Disorder Caused by Transthyretin-mediated Amyloidosis
CDER Notable Approval – November 25, 2024 - Aqneursa (levacetylleucine)
FDA Approves New Drug to Treat Niemann-Pick Disease, Type C
FDA News Release – September 24, 2024 - Miplyffa (arimoclomol)
FDA Approves First Treatment for Niemann-Pick Disease, Type C
FDA News Release - September 20, 2024 - Xolremdi (mavorixafor)
FDA Approves First Drug for WHIM Syndrome, a Rare Disorder That Can Lead to Recurrent, Life-Threatening Infections
CDER Notable Approval – April 30, 2024
- Veopoz (pozelimab-bbfg)
FDA Approves First Treatment for CD55-deficient Protein-losing Lnteropathy (CHAPLE disease)
CDER Notable Approval – August 18, 2023 - Sohonos (palovarotene)
FDA Approves First Treatment for Fibrodysplasia Ossificans Progressiva
CDER Statement – August 17, 2023 - Qalsody (tofersen)
FDA Approves Treatment of Amyotrophic Lateral Sclerosis Associated with a Mutation in the SOD1 Gene
CDER Notable Approval – April 25, 2023 - Joenja (leniolisib)
FDA Approves First Treatment for Activated Phosphoinositide 3-kinase Delta Syndrome
CDER Notable Approval – March 24, 2023 - Skyclarys (omaveloxolone)
FDA Approves First Treatment for Friedreich’s Ataxia
CDER Notable Approval – February 28, 2023 - Lamzede (velmanase alfa)
FDA Approves First Enzyme Replacement Therapy for Rare Alpha-mannosidosis
CDER Notable Approval – February 17, 2023
- Ztalmy (ganaxolone)
FDA Approves Drug for Treatment of Seizures Associated with Rare Disease in Patients Two Years of Age and Older
CDER Notable Approval – March 18, 2022 - Vonjo (pacritnib)
FDA Approves Drug for Adults with Rare Form of Bone Marrow Disorder
CDER Notable Approval – March 1, 2022 - Pyrukynd (mitapivat)
FDA Approves Treatment for Anemia in Adults with Rare Inherited Disorder
CDER Notable Approval – February 17, 2022 - Enjaymo (sutimlimab-jome)
FDA Approves Treatment for Adults with Rare Type of Anemia
CDER Notable Approval – February 4, 2022
- Tarpeyo (budesonide)
FDA Approves First Drug to Decrease Urine Protein in IgA Nephropathy, a Rare Kidney Disease
CDER Notable Approval – December 17, 2021 - Besremi (ropeginterferon alfa-2b-njft)
FDA Approves Treatment for Rare Blood Disease
FDA News Release – November 12, 2021