Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 21-24 July 2025
13 new medicines recommended for approval; another 8 medicines recommended for extension of their therapeutic indications
NewsHumanMedicines
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.
CHMP statistics
Key figures from the July 2025 CHMP meeting are represented in the graphic below.
July 2025 statistics - monthly and cumulative figures for CHMP opinions and withdrawn applications:
-
13 positive opinions on new medicines: 2 new non-orphan medicine, 5 orphan medicines, 4 biosimilars, and 2 generic, hybrid or informed consent medicine. Total in 2025: 69
-
3 negative opinions on new medicines. Total in 2025: 7
-
8 positive opinions on extensions of therapeutic indication. Total in 2025: 59
-
3 withdrawn applications for new medicines. Total in 2025: 13
Positive recommendations on new medicines
Aqneursa
- International non-proprietary name (INN)
L-Acetylleucine
- Marketing authorisation applicant
Intrabio Ireland Limited
- Therapeutic indication
Chronic treatment of Niemann-Pick Type C (NPC) in adults and children from birth
- More information
- News announcement
Ekterly
- INN
sebetralstat
- Marketing authorisation applicant
Kalvista Pharmaceuticals (Ireland) Limited
- Therapeutic indication
Treatment of hereditary angioedema (HAE) attacks in adult and adolescents aged 12 years and older
- More information
Romvimza
- INN
vimseltinib
- Marketing authorisation applicant
Deciphera Pharmaceuticals (Netherlands) B.V.
- Therapeutic indication
Treatment of adult patients with tenosynovial giant cell tumour (TGCT) who are not amenable to surgery
- More information
Tryngolza
- INN
olezarsen
- Marketing authorisation applicant
Ionis Ireland Limited
- Therapeutic indication
Treatment of familial chylomicronemia syndrome
- More information
Voranigo
- INN
vorasidenib
- Marketing authorisation applicant
Les Laboratoires Servier
- Therapeutic indication
Treatment of predominantly non-enhancing astrocytoma or oligodendroglioma
- More information
Yeytuo
- INN
lenacapavir
- Marketing authorisation applicant
Gilead Sciences Ireland Unlimited Company
- Therapeutic indication
Pre-exposure prophylaxis to prevent HIV-1
- More information
- News announcement
Zurzuvae
- INN
zuranolone
- Marketing authorisation applicant
Biogen Netherlands B.V
- Therapeutic indication
Treatment of postpartum depression (PPD) in adults
- More information
Positive recommendations on new biosimilar medicines
Bildyos
- INN
denosumab
- Marketing authorisation applicant
Henlius Europe GmbH
- Therapeutic indication
Treatment of osteoporosis and bone loss
- More information
Bilprevda
- INN
denosumab
- Marketing authorisation applicant
Henlius Europe GmbH
- Therapeutic indication
Prevention of skeletal related events in adults with advanced malignancies involving bone
- More information
Eyluxvi
- INN
aflibercept
- Marketing authorisation applicant
Biolitec pharma Limited Zweigniederlassung Jena
- Therapeutic indication
Treatment of age-related macular degeneration (AMD) and visual impairment
- More information
Usrenty
- INN
ustekinumab
- Marketing authorisation applicant
Biosimilar Collaborations Ireland Limited
- Therapeutic indication
Treatment of Crohn’s disease, treatment of plaque psoriasis and paediatric plaque psoriasis, and psoriatic arthritis
- More information
Positive recommendations on new generic medicines
Macitentan Accord
- INN
macitentan
- Marketing authorisation applicant
Accord Healthcare
- Therapeutic indication
Treatment of pulmonary arterial hypertension (PAH)
- More information
Macitentan AccordPharma
- INN
macitentan
- Marketing authorisation applicant
Accord Healthcare
- Therapeutic indication
Treatment of pulmonary arterial hypertension (PAH)
- More information
Positive recommendation on medicine for use outside the European Union
Lenacapavir Gilead
- INN
lenacapavir
- Marketing authorisation applicant
Gilead Sciences Ireland Unlimited Company
- Therapeutic indication
Pre-exposure prophylaxis to prevent HIV-1
- More information
- News announcement
Aplidin
- INN
plitidepsin
- Therapeutic indication
Treatment of multiple myeloma
- Marketing authorisation applicant
PharmaMar
- More information
Ifinwil
- INN
eflornithine
- Marketing authorisation applicant
Norgine B.V.
- Therapeutic indication
Treatment of adults and children from one year of age with high-risk neuroblastoma
- More information
Nidlegy
- INN
bifikafusp alfa / onfekafusp alfa
- Marketing authorisation applicant
Philogen S.p.A.
- Therapeutic indication
Neoadjuvant treatment of adult patients with locally advanced fully resectable melanoma
- More information
Elevidys
- INN
delandistrogene moxeparvovec
- Marketing authorisation applicant
Roche Registration GmbH
- Therapeutic indication
Treatment of ambulatory patients aged 3 to 7 years old with Duchenne muscular dystrophy
- More information
Jelrix
- INN
autologous cartilage-derived articular chondrocytes, in-vitro expanded
- Marketing authorisation applicant
TETEC Tissue Engineering Technologies AG
- Therapeutic indication
Repair of symptomatic, localised, full-thickness cartilage defects of the knee joint grade III or IV
- More information
Nurzigma
- INN
pridopidine
- Marketing authorisation applicant
Prilenia Therapeutics B.V.
- Therapeutic indication
Treatment of Huntington's disease
- More information
Positive recommendations on extensions of therapeutic indications
Alhemo
- INN
concizumab
- Marketing authorisation holder
Novo Nordisk A/S
- More information
Baqsimi
- INN
glucagon
- Marketing authorisation holder
Amphastar France Pharmaceuticals
- More information
Clopidogrel Zentiva
- INN
clopidogrel
- Marketing authorisation holder
Zentiva k.s.
- More information
Invokana
- INN
canagliflozin
- Marketing authorisation holder
Janssen-Cilag International N.V.
- More information
mResvia
- INN
single-stranded 5' capped mRNA encoding the respiratory syncytial virus glycoprotein F stabilized in the prefusion conformation
- Marketing authorisation holder
Moderna Biotech Spain, S.L.
- More information
Sirturo
- INN
bedaquiline
- Marketing authorisation holder
Janssen-Cilag International N.V.
- More information
Taltz
- INN
ixekizumab
- Marketing authorisation holder
Eli Lilly and Co (Ireland) Limited
- More information
Tevimbra
- INN
tislelizumab
- Marketing authorisation holder
Beone Medicines Ireland Limited
- More information
Re-examinations of initial applications
Kisunla
- INN
donanemab
- Marketing authorisation applicant
Eli Lilly Nederland B.V.
- More information
Start of referral procedure
Tecovirimat SIGA
- INN
tecovirimat
- More information