CHMP meeting highlights – April 2025

The Committee for Medicinal Products for Human Use (CHMP) recommended the approval of 16 medicines during its meeting this month.

The EMA’s human medicines group gave a positive opinion for approval of two conditional marketing authorisations. The first, for Duvyzat (givinostat) to treat Duchenne muscular dystrophy in patients from six years old who can walk. The second recommendation was for Ziihera (zanidatamab), to treat adults with unresectable locally advanced or metastatic HER2-positive biliary tract cancer.

This includes Alyftrek (deutivacaftor / tezacaftor / vanzacaftor) to treat cystic fibrosis in individuals from six years old and over, with at least one non class I mutation in the cystic fibrosis transmembrane conductance regulator gene.

The CHMP also adopted a positive opinion for Attrogy (diflunisal) as a treatment for hereditary transthyretin-mediated amyloidosis. Separately, the MHRA has approved Beyonttra (acoramidis) to treat adults with wild-type or variant forms of the condition, who have cardiomyopathy.

Additionally, Sephience (sepiapterin) received a recommendation to treat individuals who have hyperphenylalaninaemia with phenylketonuria. This inherited condition is defined by an inability to process the amino acid phenylalanine, causing it to build up in the blood and brain.

Tepezza (teprotumumab) received a positive opinion to treat adults with the rare autoimmune disorder thyroid eye disease (Graves’ eye disease), who have a moderate to severe form of the condition.

The CHMP also recommended Oczyesa (octreotide) to treat adults with acromegaly, a disorder characterised by excessive growth hormone in the body.

CHMP’s opinion for biosimilar medicines

Additionally, the committee recommended nine biosimilar medicines. This included Dazublys (trastuzumab) for metastatic and early breast cancer.

The CHMP adopted several positive options for denosumab biosimilars for bone-related disorders:

• Denbrayce (denosumab), Enwylma (denosumab), Vevzuo (denosumab) and Yaxwer (denosumab), as a preventative drug for adults who experience skeletal related events in advanced malignancies, and as a treatment for adults and skeletally mature adolescents with giant cell tumour of bone
• BBL (denosumab), Izamby (denosumab), Junod (denosumab) and Zadenvi (denosumab), to treat osteoporosis and bone loss.

Indication extension recommendations by the CHMP

The CHMP recommended extending the indications in the EU for ten medicines: Adcetris, Adempas, Amvuttra, Calquence, Cystadrops, Jivi, Veklury, Xofluza, Zoonotic Influenza Vaccine Seqirus and Vyvgart.

“Vyvgart is the first and only targeted IgG Fc-antibody fragment for [chronic inflammatory demyelinating polyneuropathy] and if approved, would mark the first treatment in Europe with a novel, precision mechanism of action for [these] patients in 30 years,” Dr Luc Truyen, PhD, Chief Medical Officer, argenx, explained following the CHMP’s decision.

Initial marketing authorisation application re-examinations

Furthermore, the CHMP will issue a final recommendation Kisunla (donanemab), as a treatment for early Alzheimer’s disease. This is following a request by the marketing authorisation holder to re-examine its opinion adopted during the committee’s March 2025 meeting. Details of approval recommendations granted by the CHMP in its February 2025 meeting can be viewed here.

Marketing authorisation application withdrawals

The CHMP withdrew marketing authorisation applications for two treatments. The first was an initial application for Dazluma (troriluzole hydrochloride monohydrate) to treat spinocerebellar ataxia genotype 3 (SCA3). The committee declined to extend the indication of Ngenla (somatrogon) for adults with growth hormone deficiency.