Rare disease pharmaceutical companies: who holds EU orphan medicines

What is an orphan medicine in the EU?

An orphan medicine in the EU is one designated for a life-threatening or chronically debilitating condition that affects no more than 5 in 10,000 people in the EU, where no satisfactory treatment exists or the new medicine offers significant benefit. The rules come from Regulation (EC) No 141/2000 on orphan medicinal products.

The European Medicines Agency's Committee for Orphan Medicinal Products (COMP) examines each application, and the European Commission grants the designation. A designation is not an approval: the medicine still needs a marketing authorisation. Once authorised, the EMA says the sponsor receives ten years of market exclusivity, plus two more years where the paediatric study requirements are met. Other incentives include protocol assistance, a form of scientific advice, and fee reductions.

Market exclusivity means that, for ten years, the EU will not accept another marketing authorisation application for a similar medicine for the same indication. Counting running exclusivities therefore shows which companies hold the most protected rare-disease products.

Rare disease pharmaceutical companies with the most EU orphan exclusivities

Novartis holds the most running EU orphan market exclusivities, with 16 across 11 medicines, according to the European Commission's Union Register as our register read it on 27 September 2026. Immedica Pharma follows with 8, then Pfizer, Chiesi and Bristol-Myers Squibb with 6 each. In total the register showed 203 running exclusivities across 162 medicines, held by 91 companies.

One medicine can hold several exclusivities, one for each orphan condition it is authorised for. Immedica's 8 come from three medicines, and Rhythm Pharmaceuticals holds 4 for a single medicine, Imcivree. The counts are by the holder entity named in the register, so a group with several EU entities can appear more than once.

Source: European Commission, Union Register of medicinal products, orphan designations linked to authorised medicines (CC BY 4.0), as held in our patent and exclusivity expiry tracker on 27 September 2026. Running means no end date recorded and an expiry on or after 18 October 2026. Holder name spellings merged where they differ only by Ltd/Limited or UC.

42 of the 203 exclusivities end by 18 October 2028, which opens those indications to similar medicines.

Orphan designation in the EU: what the register shows

The EMA has recorded 3,310 orphan designation decisions, of which 2,205 were positive and active when our EMA orphan designations register last read the EMA file on 27 September 2026. A further 981 had been withdrawn, 88 had expired and 36 were negative. The EMA file of 18 October 2026 still listed 3,310 records, with 2,204 positive.

The EMA's bulk designation file does not name the sponsor company, so we cannot rank companies by designations from it. Authorisations show more directly who sells rare-disease medicines.

Recent authorisations show the flow. Our EMA approvals register counts 15 orphan medicines among the 81 EU authorisations dated 17 October 2025 to 21 September 2026. Two of the 15 are held by Irish-registered companies: Redemplo (plozasiran), held by Arrowhead Pharmaceuticals Ireland, and Aqneursa (L-acetylleucine), held by IntraBio Ireland.

Orphan drug companies with an Irish presence

Alexion, AstraZeneca Rare Disease, makes rare-disease medicines in Ireland at two sites. Alexion says it employs over 1,300 people across two manufacturing facilities, at College Business and Technology Park, Blanchardstown, Dublin 15, and at Monksland, Athlone, County Roscommon, and that its global supply chain distributes its rare disease therapies from Ireland. Its medicines include eculizumab (Soliris) and ravulizumab; see also its clinical trial sponsor page.

BioMarin International Limited, the holder of four running orphan exclusivities, is registered at Shanbally, Ringaskiddy, County Cork, according to the EMA product page for Voxzogo. Our Cork pharma map lists BioMarin's site there for biologics drug substance manufacturing and packaging.

Eleven holders with Ireland in their registered name held 23 running orphan exclusivities on 27 September 2026. The largest are Vertex Pharmaceuticals (Ireland) Limited and Jazz Pharmaceuticals Ireland Limited with 5 each, and Takeda Pharmaceuticals International AG Ireland Branch with 3. The others include Gilead Sciences Ireland, Akcea Therapeutics Ireland, Immunocore Ireland, Kalvista Pharmaceuticals (Ireland), Leadiant Biosciences Ireland and Amivas Ireland. A holder registered in Ireland is the legal entity responsible for the medicine in the EU; it does not mean the medicine is made here. Our directory of companies in Ireland and the Ireland pharma map show where manufacturing happens, including Jazz Pharmaceuticals in Roscommon and Takeda in Dublin and Wicklow.

Top companies developing achondroplasia treatments

BioMarin holds the only EU-authorised achondroplasia medicine, Voxzogo (vosoritide), authorised on 26 August 2021, and Ascendis Pharma's Yuviwel (navepegritide) received a positive CHMP opinion on 15 October 2026. BridgeBio's oral infigratinib is the next candidate, with a US application under review and an EU filing planned for the fourth quarter of 2026.

Achondroplasia is an inherited condition caused by a change in the FGFR3 gene, as the EMA describes it. Voxzogo is authorised for patients aged 4 months and older whose growth plates are not closed, and its orphan market exclusivity runs to 1 September 2033 under the Union Register, including a two-year paediatric extension recorded on 10 April 2026. For Yuviwel, the CHMP recommended use in children aged 2 and older whose growth plates are not closed; the European Commission decision is pending and the EMA will review whether the orphan designation can be maintained. The US FDA approved Yuviwel on 27 February 2026 under accelerated approval.

Sources: EMA product pages for Voxzogo and Yuviwel; EMA CHMP highlights, 12-15 October 2026; FDA Novel Drug Approvals 2026; BridgeBio release of 6 October 2026; ClinicalTrials.gov records read 18 October 2026. Status only; no comparison of the medicines is implied.

Ireland has a part in the trials. Four Ascendis navepegritide studies list a site in Ireland on ClinicalTrials.gov, including a Phase 2 study in infants under 2 (NCT06079398) and one in adolescents aged 12 to 18 (NCT06732895). Kyowa Kirin, which licensed infigratinib for Japan in February 2024, runs a separate Phase 3 study there, AOBA (NCT06926491). Trial records are on our achondroplasia trials page, and the sponsors have pages for BioMarin, Ascendis Pharma, QED Therapeutics and Tyra Biosciences.

Rare disease medicines policy in Ireland

Ireland's Budget 2027 will fund a pilot scheme to support earlier access to new medicines for rare diseases, the Department of Health announced on 7 October 2026, within a total health allocation of €29.1 billion. The press release does not give the pilot's cost or start date. Our report on the Budget 2027 health allocation and the rare disease medicines pilot has more on the announcement.

The pilot follows the National Rare Disease Strategy 2025-2030, which estimates that about 300,000 people in Ireland live with a rare disease. The strategy aligns with the Programme for Government commitment to review options for earlier reimbursement of orphan medicines and early access schemes. We cover how Irish reimbursement works for orphan medicines in our article on Ireland's drug approval system and orphan medicines reform, and trial capacity in our report on rare disease trial bottlenecks in Ireland. Trials recruiting in Ireland are listed on our Irish clinical trials register.

Method and sources

Company counts come from the European Commission's Union Register list of active orphan designations linked to authorised medicines, as our tracker read it on 27 September 2026. We counted one exclusivity per medicine and orphan condition, kept only those with no end date and an expiry on or after 18 October 2026, and merged holder names that differ only by Ltd/Limited or UC. Irish holders are those with Ireland in the registered name. Designation totals come from the EMA orphan designations file. Achondroplasia statuses come from the EMA, the FDA, company releases and ClinicalTrials.gov, read on 18 October 2026. Corrections are logged on our corrections page and the registers are explained on our methodology page.