13 new medicines recommended for approval
EMA’s human medicines committee (CHMP) recommended 13 medicines for approval at its July 2025 meeting.
The committee recommended granting a marketing authorisation for Aqneursa* (levacetylleucine), for the treatment of Niemann-Pick type C, a rare, progressive, fatal genetic disorder caused by mutations encoding lysosomal proteins that are essential for the intracellular transport and metabolism of body fats, including cholesterol. See more details in the news announcement in the grid below.
Ekterly* (sebetralstat) received a positive opinion from the CHMP for the treatment of acute attacks of hereditary angioedema, a rare, chronic, genetic, debilitating, and potentially life-threatening disorder characterised by recurrent and often unpredictable attacks of swelling in many parts of the body. The medicine is administered orally at the earliest sign of an attack. This is the first oral treatment for hereditary angioedema recommended for approval in the European Union (EU), reducing the burden of injection from patients and speeding up the time between onset of attack and administration of the medicine.
The CHMP recommended granting a marketing authorisation for Romvimza* (vimseltinib), for the treatment of adults with symptomatic tenosynovial giant cell tumour, a condition where the tissue surrounding the joints and tendons, called the synovial lining or synovium, expands abnormally forming outgrowths of the joint.
A positive opinion was adopted for Tryngolza* (olezarsen), for the treatment of adults with familial chylomicronemia syndrome, a rare, serious autosomal recessive inherited disease characterised by elevated triglyceride levels in the blood.
The CHMP recommended granting a marketing authorisation for Voranigo* (vorasidenib), a medicine intended for the treatment of low-grade astrocytoma or oligodendroglioma, two rare malignant brain tumours.
The committee recommended granting a marketing authorisation for Yeytuo (lenacapavir), for pre-exposure prophylaxis (PrEP) in combination with safer sex practices to reduce the risk of sexually acquired human immunodeficiency virus type 1 (HIV-1) infection in adults and adolescents at high risk of becoming infected. This medicine will facilitate PrEP uptake and compliance because it only has to be administered twice a year via a subcutaneous injection. The committee simultaneously reviewed the medicine for the EU market, under the centralised procedure, and for non-EU countries, under the EU-Medicines for all (EU-M4all) programme that enables EMA to support global regulatory capacity building and contribute to the protection and promotion of public health beyond the EU. See more details in the news announcement in the grid below.
Zurzuvae (zuranolone) received a positive opinion from the CHMP for the treatment of postpartum depression in adults following childbirth.
The committee adopted positive opinions for four biosimilar medicines:
- Bildyos (denosumab), for the treatment of osteoporosis and bone loss.
- Bilprevda (denosumab), for the prevention of skeletal-related events in adults with advanced malignancies involving bone.
- Eyluxvi (aflibercept), for the treatment of age-related macular degeneration and visual impairment.
- Usrenty (ustekinumab), for the treatment of Crohn’s disease, plaque psoriasis and paediatric plaque psoriasis, and psoriatic arthritis.
Two generic medicines received a positive opinion: Macitentan Accord (macitentan) and Macitentan AccordPharma (macitentan). Both medicines are intended for the treatment of pulmonary arterial hypertension.
Negative opinion for three medicines
The committee recommended not granting a marketing authorisation for Elevidys* (delandistrogene moxeparvovec) for the treatment of Duchenne muscular dystrophy, a rare, ultimately lethal genetic disease in which the muscles progressively weaken and lose function.
Jelrix (cartilage-forming cells, autologous) received a negative opinion from the CHMP for the treatment of cartilage defects in the knee.
The CHMP adopted a negative opinion for Nurzigma* (pridopidine), intended for the treatment of adults with Huntington’s disease, an inherited condition that worsens over time and causes brain cells to die.
For more information on these negative opinions, see the question-and-answer documents in the grid below.
Recommendations on extensions of therapeutic indication for 8 medicines
The committee recommended another eight extensions of indication for medicines that are already authorised in the EU: Alhemo, Baqsimi, Clopidogrel Zentiva, Invokana, mResvia, Sirturo, Taltz and Tevimbra.
Withdrawal of applications
Applications for initial marketing authorisation for three medicines were withdrawn:
- Aplidin (plitidepsin), a medicine expected to be used to treat multiple myeloma.
- Ifinwil (eflornithine), for the treatment of adults and children from one year of age with high-risk neuroblastoma, a cancer of nerve cells in different parts of the body.
- Nidlegy (bifikafusp alfa / onfekafusp alfa), for the treatment of adult patients with locally advanced fully resectable melanoma.
Question-and-answer documents on the withdrawals of these medicines are available in the grid below.
Outcome of re-examination
Following a re-examination, the committee has recommended granting a marketing authorisation for Kisunla (donanemab), a medicine intended for the treatment of early Alzheimer’s disease. The committee has recommended granting a marketing authorisation in people who do not have a copy of the ApoE4 gene, a certain form of the gene for the protein apolipoprotein E, or people who have only one copy of the gene.
For more information, see the question-and-answer document in the grid below.
Conclusion of referral
The CHMP endorsed the recommendation of the Agency’s safety committee (PRAC) following a review of serious side effects of Ixchiq, a live attenuated chikungunya vaccine. The previous temporary restriction on vaccinating people aged 65 years and above, which was put in place during the review, will now be lifted. For more information, see the public health communication in the grid below.
Start of referral
The committee started a review of Tecovirimat SIGA to consider emerging data from recent clinical trials suggesting a lack of effectiveness in the treatment of mpox, a disease caused by the monkeypox virus. The review of Tecovirimat SIGA was initiated at the request of the European Commission, under Article 20 of Regulation (EC) No 726/2004. For more information, see the public health communication in the grid below.
Other updates
The CHMP gave a positive opinion to update the composition of the mRNA vaccines Comirnaty and Spikevax to target the new SARS-CoV-2 LP.8.1 variant of the virus that causes COVID-19. The revision of these vaccines is in line with the recommendation issued by EMA’s Emergency Task Force to update COVID-19 vaccines to target the SARS-CoV-2 variant LP.8.1 for the 2025/2026 vaccination campaign.
The CHMP has approved a change in the composition of Trixeo Aerosphere and its duplicate product Riltrava Aerosphere to replace the existing gas propellant with a low global warming potential gas alternative. This is the first reformulation of an inhaled medicine with a more environmentally friendly propellant. See more details in the news announcement in the grid below.
The committee finalised its assessment of an application to extend the use of the diagnostic medicine Neuraceq in adults to monitor their response to treatments that reduce beta amyloid plaques. These plaques are abnormal clumps of protein that build up in the brain of people with Alzheimer’s disease, leading to problems with brain function. Although the CHMP did not recommend this use, it agreed that the medicine’s product information should be updated so that healthcare professionals have access to up-to-date information. A question-and-answer document on the update is available in the grid below.
Agenda and minutes
The agenda of the July 2025 CHMP meeting is published on EMA’s website. Minutes of the meeting will be published in the coming weeks.
*This product was designated as an orphan medicine during its development. Orphan designations are reviewed by EMA’s Committee for Orphan Medicinal Products (COMP) at the time of approval to determine whether the information available to date allows maintaining the medicine’s orphan status and granting the medicine ten years of market exclusivity.
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