A total of 37 medicinal products containing a new active substance (NAS) received marketing authorization from the European Commission in 2024, a figure only marginally higher than the 36 products that were approved in 2023.
The 2024 and 2023 figures for NAS-containing medicines were significantly lower than the 55 products approved in 2022, although that year marked a record high for the EU. The 2023 figure had been the lowest since 2019, when just 28 NAS-containing drugs gained an EU-wide marketing authorization.
In 2025, to date, three NAS-containing products have been approved and 12 other products are expected to be authorized shortly, having been recommended for approval by the European Medicine Agency last year.
Among the products expected to be approved soon are Leqembi (lecanemab), Eisai/Biogen’s disease modifying treatment for early Alzheimer’s disease, and MSD’s Welireg (belzutifan), which could become the first medicine approved in the EU to treat the rare genetic disorder, von Hippel-Lindau disease.
The EU regulator defines a NAS as a substance that is not contained in any other medicinal product that has received a marketing authorization in the EU.
Fifteen Orphans, Two ATMPs, Two Vaccines
Of the 37 new products approved, 15 were orphan medicines, compared with the 11 rare disease drugs approved in 2023, according to an analysis by the Pink Sheet.
Among the orphan drugs approved last year were Ipsen’s Iqirvo (elafibranor), for the treatment of primary biliary cholangitis, and GSK’s Omjjara (momelotinib), for treating splenomegaly or other disease-related symptoms in adults who have myelofibrosis and moderate to severe anemia.
Two advanced therapy medicinal products (ATMPs) were approved in 2024, both of which were gene therapies. One of these was Vertex Pharmaceuticals/CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel), which became the first gene editing therapy for beta thalassemia and severe sickle cell disease to be approved for marketing in the EU. The other was Pfizer’s Beqvez (fidanacogene elaparvovec), for hemophilia B.
Both gene therapies received a conditional marketing authorization (CMA), meaning that the companies marketing them will need to generate additional data if they are to gain a full marketing authorization. A CMA may be granted based on less comprehensive clinical data than normally required, where the benefit of immediate availability of the medicine outweighs the risk inherent in the fact that additional data are still required.
A total of two vaccines were given the all clear last year – Valneva’s Ixchiq, which was the first chikungunya vaccine to be approved in the EU, and mRESVIA, from Moderna, for preventing lower respiratory tract disease caused by respiratory syncytial virus in adults 60 years of age and older.
Cancer & Blood Lead Therapeutic Categories
As in previous years, NAS-containing cancer therapies led the approvals, with 11 products (see table below for approvals by therapeutic areas). This was followed closely by treatments for blood disorders with 10 drugs.
Among the oncology drugs was AbbVie’s platinum-resistant ovarian cancer treatment, Elahere (mirvetuximab soravtansine), which became the first folate receptor alpha-directed antibody drug conjugate medicine approved in the EU.
AstraZeneca’s Truqap (capivasertib) became the first AKT inhibitor to be approved in the EU for breast cancer patients with specific biomarker alterations (PIK3CA, AKT1 or PTEN).
Meanwhile, Loqtorzi (toripalimab), Junshi Biosciences/Coherus BioSciences' anti-PD-1 monoclonal antibody, became the first approved treatment for nasopharyngeal carcinoma, and the first first-line treatment for advanced or metastatic esophageal squamous cell carcinoma, regardless of PD-L1 status.
As for the blood disorder drugs, in addition to Casgevy and Beqvez, the approved therapies included Sobi/Sanofi’s hemophilia A drug, Altuvoct (efanesoctocog alfa), the hemophilia A and B treatments, Alhemo (concizumab) and Hympavzi (marstacimab), from Novo Nordisk and Pfizer respectively, and Takeda’s Adzynma, (apadamtase alfa/cinaxadamtase alfa), for treating ADAMTS13 deficiency in children and adult patients with congenital thrombotic thrombocytopenic purpura.
There were also three therapies approved for paroxysmal nocturnal hemoglobinuria: Novartis’s Fabhalta (iptacopan), Alexion/AstraZeneca’s Voydeya (danicopan), and Roche’s Piasky (crovalimab).
Approval Pathways
At six, slightly fewer products in total received a CMA in 2024 compared with the previous two years - seven in 2023 and eight in 2022.
In addition to Casgevy and Beqvez, these were Ipsen’s Iqirvo (elafibranor), Regeneron Pharmaceuticals’ Ordspono (odronextamab), Mirati Therapeutics/Bristol Myers Squibb’s Krazati (adagrasib), and CSL Vifor/Travere Therapeutics’s Filspari (sparsentan).
Two products received an approval under exceptional circumstances. One of these was Biogen’s antisense oligonucleotide Qalsody (tofersen), an orphan drug for the treatment of adults with amyotrophic lateral sclerosis associated with a mutation in the superoxide dismutase 1 gene. The other was Takeda’s Adzynma.
The exceptional circumstances approval is used where the applicant is unable to provide comprehensive data on safety and efficacy under normal conditions of use because the condition to be treated is rare or because the collection of full information is not possible or is unethical.
Accelerated Assessment & PRIME
Six of the 37 approved products had been developed under PRIME, the EMA’s priority medicines scheme for drugs for unmet medical needs. These were Beqvez, Casgevgy, Ixchiq, Fabhalta, Voydeya and MSD’s Winrevair, which became the first activin signaling inhibitor therapy for pulmonary arterial hypertension approved in the EU.
PRIME is designed to get medicines to patients faster by helping developers optimize their development plans and increasing the likelihood of having their products fast-tracked under the EU’s accelerated assessment pathway when they are eventually filed for regulatory review.
That said, only one of the 37 products approved in 2024 had been reviewed under the accelerated assessment mechanism. This was Ixchiq.
2025
In 2025, the commission had as of 21 January approved three products containing NASs. One was InflaRx’s Gohibic (vilobelimab), the first drug approved in the EU for the treatment of SARS-CoV-2-induced acute respiratory distress syndrome. The other was Augtyro (repotrectinib, Bristol Myers Squibb’s treatment for patients whose solid tumors have a neurotrophic tyrosine receptor kinase gene fusion, or patients with ROS1-positive advanced non-small cell lung cancer.
Siiltibcy (mycobacterium tuberculosis derived antigens (rdESAT-6/rCFP-10)), Serum Life Science/Serum Institute of India’s product for diagnosing Mycobacterium tuberculosis infection was approved on 13 January. According to data from the EMA, Siiltibcy is a NAS.
In addition to Leqembi and Welireg, other NAS-containing products that received a positive recommendation from the EMA last year and which are expected to be approved by the commission soon are:
- Henlius Biotech’s Hetronifly (serplulimab).
- Janssen-Cilag’s Lazcluze (lazertinib).
- BridgeBio Pharma/AstraZeneca’s Beyonttra (acoramidis).
- Galderma/Chugai Pharmaceutical’s Nemluvio (nemolizumab).
- Gilead Sciences' Seladelpar Gilead ((seladelpar lysine dihydrate).
- Geron‘s Rytelo (imetelstat).
- CSL’s Andembry (garadacimab).
- AstraZeneca/Ionis Pharmaceuticals’ Wainzua (eplontersen).
- AstraZeneca’s Kavigale (sipavibart).
- Arcturus Therapeutics’ Kostaive (zapomeran/ARCT-154).
Once granted, approvals by the commission are valid in all EU member states as well as in the European Economic Area countries of Iceland, Liechtenstein and Norway.
*Products categorized as Medicine in the table below are classified as chemical drugs by the EMA. Also, some medicines might fall into more than one therapeutic area but have been reflected only in one.
Details of all EU approvals of NAS-containing products are provided in the Pink Sheet’s New Drug Approvals Tracker.
