EMA’s human medicines committee (CHMP) recommended 16 medicines for approval at its April 2025 meeting.
The CHMP recommended granting a marketing authorisation for Alyftrek* (deutivacaftor / tezacaftor / vanzacaftor), a medicine intended for the treatment of cystic fibrosis in people aged six years and older who have at least one non class I mutation in the cystic fibrosis transmembrane conductance regulator gene. Cystic fibrosis is an inherited disease that damages the lungs, the digestive system and other organs.
A positive opinion was adopted for Attrogy* (diflunisal), for the treatment of hereditary transthyretin-mediated amyloidosis, a disease in which abnormal proteins called amyloids build up in tissues around the body including around the nerves.
The committee recommended granting a conditional marketing authorisation for Duvyzat* (givinostat), as a treatment for Duchenne muscular dystrophy (DMD) in patients from the age of six who can walk. DMD is a rare, ultimately lethal genetic disease in which the muscles progressively weaken and lose function. See more details in the news announcement in the grid below.
Sephience* (sepiapterin) received a positive opinion from the CHMP for the treatment of hyperphenylalaninaemia in adults and children with phenylketonuria, an inherited disease where people cannot process the amino acid phenylalanine, causing it to build up in the blood and brain, which can be harmful.
Tepezza (teprotumumab) received a positive opinion from the CHMP for the treatment of adults with moderate to severe Thyroid Eye Disease (TED), also known as Graves’ Eye Disease, a rare autoimmune disease that triggers inflammation of muscles, fat, and other tissues around and behind the eyes. Treatment options for moderate-to-severe TED are limited, most patients are treated with corticosteroids and some patients need multiple reconstructive surgeries. See more details in the news announcement in the grid below.
The CHMP recommended granting a conditional marketing authorisation for Ziihera* (zanidatamab), for the treatment of adults with unresectable locally advanced or metastatic HER2-positive biliary tract cancer, a collective term for a group of rare, often fatal gastrointestinal tract cancers, accounting for approximately 1% of all adult cancers.
The CHMP recommended granting a marketing authorisation for Oczyesa* (octreotide), for the treatment of adults with acromegaly, a disease that leads to excessive growth, particularly of bones in the hands, feet and face caused by too much growth hormone. This medicine was submitted in a hybrid application, which relies in part on the results of pre-clinical tests and clinical trials of an already-authorised reference product and in part on new data.
The committee adopted positive opinions for nine biosimilar medicines:
The committee recommended not granting a marketing authorisation for Winlevi (clascoterone), a medicine intended for treating acne vulgaris.
For more information on this negative opinion, see the question-and-answer document in the grid below.1
The committee recommended extensions of indication for ten medicines that are already authorised in the European Union (EU): Adcetris, Adempas, Amvuttra*, Calquence, Cystadrops*, Jivi, Veklury, Vyvgart*, Xofluza and Zoonotic Influenza Vaccine Seqirus.
The marketing authorisation holder for Kisunla (donanemab), a medicine intended for the treatment of early Alzheimer’s disease, has requested a re-examination of the opinion adopted during the committee’s March 2025 meeting. Upon receipt of the grounds of the request, the CHMP will re-examine its opinion and issue a final recommendation.
An application for an initial marketing authorisation was withdrawn. Dazluma (troriluzole hydrochloride monohydrate) was intended for the treatment of spinocerebellar ataxia genotype 3 (SCA3), an inherited brain disorder that affects coordination and balance.
An application to extend the therapeutic indication of Ngenla (somatrogon) in the treatment of adults with growth hormone deficiency was also withdrawn.
Question-and-answer documents on the withdrawals of these medicines are available in the grid below.
The agenda of the April 2025 CHMP meeting is published on EMA's website. Minutes of the meeting will be published in the coming weeks.
Key figures from the April 2025 CHMP meeting are represented in the graphic below.
*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.
1 This news announcement was updated on 30 April 2025 to include the negative opinion for Winlevi (clascoterone)
deutivacaftor / tezacaftor / vanzacaftor
Vertex Pharmaceuticals (Ireland) Limited
Treatment of cystic fibrosis.
diflunisal
Purpose Pharma International AB
Treatment of ATTR amyloidosis.
givinostat
Italfarmaco S.p.A.
Treatment of Duchenne muscular dystrophy (DMD).
sepiapterin
PTC Therapeutics International Limited
Treatment of hyperphenylalaninaemia (HPA) in adult and paediatric patients with phenylketonuria (PKU).
teprotumumab
Amgen Europe B.V.
Treatment of moderate to severe Thyroid Eye Disease (TED).
zanidatamab
Jazz Pharmaceuticals Ireland Limited
Treatment of biliary tract cancer.
trastuzumab
CuraTeQ Biologics s.r.o.
Treatment of metastatic and early breast cancer.
denosumab
Mabxience Research S.L.
Prevention of skeletal related events in adults with advanced malignancies, treatment of adults and skeletally mature adolescents with giant cell tumour of bone.
denosumab
Biosimilar Collaborations Ireland Limited
Treatment of osteoporosis and bone loss.
denosumab
Zentiva k.s.
Prevention of skeletal related events in adults with advanced malignancies, treatment of adults and skeletally mature adolescents with giant cell tumour of bone.
denosumab
Mabxience Research S.L.
Treatment of osteoporosis and bone loss.
denosumab
Gedeon Richter Plc.
Treatment of osteoporosis and bone loss.
denosumab
Biosimilar Collaborations Ireland Limited
Prevention of skeletal related events in adults with advanced malignancies, treatment of adults and skeletally mature adolescents with giant cell tumour of bone.
denosumab
Gedeon Richter Plc.
Prevention of skeletal related events in adults with advanced malignancies, treatment of adults and skeletally mature adolescents with giant cell tumour of bone.
denosumab
Zentiva k.s.
Treatment of osteoporosis and bone loss.
octreotide
Camurus AB
Treatment of acromegaly.
brentuximab vedotin
Takeda Pharma A/S
riociguat
Bayer AG
vutrisiran
Alnylam Netherlands B.V.
acalabrutinib
AstraZeneca AB
mercaptamine
Recordati Rare Diseases
damoctocog alfa pegol
Bayer AG
remdesivir
Gilead Sciences Ireland UC
efgartigimod alfa
Argenx
baloxavir marboxil
Roche Registration GmbH
zoonotic influenza vaccine (H5N8) (surface antigen, inactivated, adjuvanted)
Seqirus S.r.l.
clascoterone
Cassiopea S.p.A.
Indicated for the topical treatment of acne vulgaris in adults and adolescents
donanemab
Eli Lilly Nederland B.V.
troriluzole
Biohaven Biosciences Ireland Limited
somatrogon
Pfizer Europe MA EEIG