Orphan drug approvals, in numbers.

An orphan drug is a medicine developed for a rare disease, defined in the United States as a condition affecting fewer than 200,000 people and in the European Union as one affecting no more than 5 in 10,000. This page counts the orphan approvals recorded on the two regulators' public registers. Both registers were captured on 31 July 2026.

Key figures

• The FDA register records 1,437 orphan approvals between 1983 and July 2026, granted under 1,075 orphan designations and covering 833 distinct active ingredients.
• 58% of those FDA orphan approvals (837) were granted from 2017 onwards.
• 2025 is the highest full year on the FDA register, with 97 orphan approvals. The register shows 45 for 2026 up to 22 July.
• 281 active ingredients hold more than one FDA orphan approval.
• The EMA lists 163 authorised medicines that hold orphan status in the European Union, from 99 marketing authorisation holders.

FDA orphan approvals by year

FDA orphan approvals ran at 52 or fewer a year until 2016 and at 75 or more in every full year since 2017. The count below is by FDA marketing approval date. Source: FDA Orphan Drug Product designation database.

Conditions with the most FDA orphan approvals

Multiple myeloma has the most FDA orphan approvals of any condition counted here, with 44. Each row counts the approvals whose FDA orphan designation text names the condition. One medicine approved for several uses in the same condition is counted once for each approval. Source: FDA Orphan Drug Product designation database.

Blood cancers and other cancers account for most of this table because the United States grants orphan designation to any condition under the 200,000 threshold, which includes many individual cancer types.

How concentrated FDA orphan approvals are

The FDA register names 448 sponsors across its 1,437 orphan approvals. 248 sponsors hold a single orphan approval, and 31 sponsors hold ten or more. Sponsors are counted as named on the register, with legal-form differences such as Corp. and Corporation folded together. Affiliates of one group that are registered under different names are counted separately. Source: FDA Orphan Drug Product designation database.

The active ingredients with the most FDA orphan approvals are nivolumab (19), pembrolizumab (17) and lisocabtagene maraleucel (12).

Orphan medicines authorised in the European Union

The EMA dataset lists 163 authorised medicines holding orphan status. 17 of them are advanced therapy medicinal products, 22 hold a conditional marketing authorisation and 22 were authorised under exceptional circumstances. 108 of the 163 were authorised from 2020 onwards. Source: EMA medicines dataset.

The EU figure is smaller than the US figure for a structural reason. A medicine leaves the EU orphan list when its market exclusivity ends or its designation is withdrawn, so the EMA list shows medicines that hold orphan status now. The FDA register keeps every orphan approval since 1983.

Method and limits

• Sources. The FDA Orphan Drug Product designation database, filtered to approved designations, and the EMA medicines dataset, filtered to authorised medicines with orphan status. Both were captured on 31 July 2026.
• Unit of count. An FDA orphan approval is one approved indication under an orphan designation. One designation can carry several approvals, and one medicine can hold several designations.
• Historical approvals are included. The FDA register lists approvals since 1983 whether or not the product is still marketed. 17 of the approvals sit under a designation that was later withdrawn or revoked.
• Condition counts depend on wording. Conditions are matched on the words of the FDA designation. A designation worded differently from the condition name is not counted in that row.
• One EMA record has no authorisation date in the dataset and is left out of the by-year count only.
• Phase 3 candidates are not counted here. AXLRx tracks them, but they are not published until each row has been checked against its trial registry record.

Questions this page answers

How many orphan drugs has the FDA approved?

The FDA Orphan Drug Product designation database records 1,437 orphan approvals from 1983 to July 2026. They were granted under 1,075 orphan designations and cover 833 distinct active ingredients.

How many orphan medicines are authorised in the European Union?

The EMA medicines dataset lists 163 authorised medicines that hold orphan status, as captured on 31 July 2026. Medicines whose orphan status has ended are no longer on that list.

Which rare disease has the most FDA orphan approvals?

Multiple myeloma has 44 FDA orphan approvals, followed by non-small cell lung cancer with 37 and melanoma with 34, counted by the condition named in the orphan designation.

Are orphan drug approvals increasing?

Yes. The FDA register shows 75 or more orphan approvals in every full year since 2017, against 52 or fewer in every year before. 58% of all FDA orphan approvals since 1983 were granted from 2017 onwards.

Related AXLRx analysis

AXLRx publishes commercial analysis for individual rare diseases, including paroxysmal nocturnal haemoglobinuria, myasthenia gravis, sickle cell disease, hereditary angioedema, spinal muscular atrophy, Dravet syndrome, Fabry disease and ATTR amyloidosis. The sourcing standard behind every figure is set out in the methodology, and the engine page explains how a question becomes a deliverable. To ask about the full rare disease company and asset list, use the contact page.