The EMA has published its 2025 results: 16 new drugs for rare diseases have been recommended in the EU.
On January 15, 2026, the European Medicines Agency (EMA) presented its annual review of medicinal product recommendations for 2025 – Human Medicines in 2025.
At the end of the year, the EMA recommended 104 medicinal products for registration in the EU, including 38 containing a new active substance not previously registered in the European Union.
Special emphasis is placed on orphan diseases: in 2025, the EMA recommended 16 drugs for the treatment of rare diseases.
Orphan Disease Drugs Recommended by the EMA in 2025
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Ezmekly (mirdametinib) — symptomatic, inoperable plexiform neurofibromas associated with NF1 in adults and children aged ≥2 years.
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Ziihera (zanidatamab) — HER2-positive biliary tract cancer (BTC) after ≥1 line of therapy.
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Ogsiveo (nirogacestat) — progressive desmoid tumors in adults.
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Romvimza (vimseltinib) — symptomatic TCT after exhaustion of surgical options.
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Voranigo (vorasidenib) — IDH1/2-mutated Grade 2 gliomas after surgery (≥12 years).
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Zemcelpro (dorocubicel) — allogeneic HSCT for hematological malignancies in the absence of a suitable donor.
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Vyjuvek (beremagene geperpavec) — wound treatment for dystrophic epidermolysis bullosa (DEB) from birth.
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Wayrilz (rilzabrutinib) — refractory immune thrombocytopenia (ITP) in adults.
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Ekterly (sebetralstat) — acute attacks of hereditary angioedema (HAE) in patients ≥12 years of age.
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Waskyra (etuvetidigene autotemcel) — Wiskott-Aldrich syndrome in the absence of an HLA-matched related donor (≥6 months).
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Aqneursa (levacetylleucine) — neurological manifestations of Niemann-Pick disease type C (≥6 years).
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Maapliv (amino acids) — acute decompensation of MSUD when enteral nutrition is impossible.
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Sephience (sepiapterin) — hyperphenylalaninemia in phenylketonuria (PKU).
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Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) — cystic fibrosis in the presence of ≥1 CFTR mutation (non-Class I).
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Tryngolza (olezarsen) — familial chylomicronemia syndrome (FCS) in adults.
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Duvyzat (givinostat) — Duchenne muscular dystrophy in outpatients ≥6 years of age.
Biosimilars and Pharmacovigilance
The EMA also reports the registrationof 41 biosimilars, emphasizing their importance for funding sustainability and expanding access to therapy. Following authorization by the European Commission, continuous post-registration surveillance to monitor quality, safety, and the benefit-risk balance continues.
Source: EMA — Human Medicines in 2025 (full version of the overview and report) (https://www.ema.europa.eu/en/documents/report/human-medicines-2025_en.pdf)
