Five Pharmaceutical Companies With FDA-Approved Orphan Drugs: Selected Examples, 2021–2025
ACADIA, BioMarin, Novartis, Sarepta, and Servier appear in FDA records for the seven treatments below, covering conditions including Rett syndrome, achondroplasia, hemophilia A, Duchenne muscular dystrophy, and certain gliomas. The examples show which treatments are associated with each company name, the rare-disease uses approved, and when those approvals occurred.
This is a selected overview of approvals from January 1, 2021, through December 31, 2025, rather than a ranking by company totals. FDA treats orphan designation and marketing approval as separate processes; the examples below have documented approvals for the selected uses.
Table of contents
- Companies, treatments, and approved uses
- What these examples help you compare
- Scope and company attribution
- Explore related approval records
- Frequently asked questions
- Sources
Companies, treatments, and approved uses
Companies are listed alphabetically. Indications summarize the historical approval associated with each date, including key eligibility restrictions; they are not statements of current labeling.
| Company named in cited FDA records | Treatment | Indication at the listed approval | FDA approval date |
|---|---|---|---|
| ACADIA Pharmaceuticals Inc. | Daybue (trofinetide) | Rett syndrome in adults and children age 2 and older | March 10, 2023 |
| BioMarin Pharmaceutical, Inc. | Voxzogo (vosoritide) | Increased linear growth in children age 5 and older with achondroplasia and open growth plates | November 19, 2021 |
| BioMarin Pharmaceutical, Inc.† | Roctavian (valoctocogene roxaparvovec-rvox) | Severe hemophilia A in adults without antibodies to AAV5 detected by an FDA-approved test | June 29, 2023 |
| Novartis Pharmaceuticals Corporation | Vijoice (alpelisib) | Severe manifestations of PIK3CA-related overgrowth spectrum (PROS) requiring systemic therapy, from age 2 | April 5, 2022 |
| Sarepta Therapeutics, Inc. | Amondys 45 (casimersen) | Duchenne muscular dystrophy with a confirmed DMD gene mutation amenable to exon 45 skipping | February 25, 2021 |
| Sarepta Therapeutics, Inc.† | Elevidys (delandistrogene moxeparvovec-rokl) | Duchenne muscular dystrophy in ambulatory children ages 4–5 with a confirmed DMD gene mutation | June 22, 2023 |
| Servier Pharmaceuticals, LLC | Voranigo (vorasidenib) | Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation, following surgery, from age 12 | August 6, 2024 |
What these examples help you compare
The indication matters as much as the company or brand name. Sarepta’s two examples both concern Duchenne muscular dystrophy, but the listed approvals describe different eligible populations: Amondys 45 requires a mutation amenable to exon 45 skipping, while Elevidys’s original approval specified ambulatory children ages 4–5. Similarly, Vijoice’s approval concerns severe manifestations of PROS requiring systemic treatment, rather than every condition involving PIK3CA.
The dates also provide historical context. A later population expansion or label change does not alter the date of the selected approval. Use the linked FDA records and current prescribing information when assessing a treatment’s present indication or eligibility.
Scope and company attribution
The seven treatments are selected examples, not an exhaustive inventory of each company’s orphan-drug portfolio. Their number does not establish market share, commercial success, clinical superiority, or which company had the most approvals. No parent-company consolidation has been applied.
Company names follow the cited FDA records and do not establish current ownership or commercialization rights. For the Roctavian and Elevidys entries marked †, the cited Roctavian and Elevidys Purple Book pages identify applicants but do not independently establish the sponsor at the original 2023 approval. Those entries should therefore be read as company associations in FDA records, rather than reconstructed historical sponsor attribution.
Explore related approval records
QOPE’s FDA Approvals dataset supports exploration of application and product details, including sponsor, dosage form, and strength. It does not provide a complete submission history or host the orphan-designation records needed for a comprehensive orphan-approval comparison. Verify indication-specific approvals and their dates in the relevant FDA records.
Frequently asked questions
Does orphan designation mean a treatment is approved?
No. Designation establishes eligibility for certain orphan-drug benefits; marketing approval is a separate FDA decision for a specified use.
Can one treatment have more than one approval?
Yes. FDA may approve additional indications or expand an eligible population. The table identifies the selected historical approval, so a later approval should be assessed separately when comparing indications or dates.