14 new medicines recommended for approval; another seven medicines are recommended for extension of their therapeutic indications

EMA’s human medicines committee (CHMP) recommended 14 medicines for approval at its May 2024 meeting.

The CHMP recommended granting a marketing authorisation under exceptional circumstances for Adzynma* (rADAMTS13) an enzyme replacement therapy indicated for the treatment of children and adult patients with congenital thrombotic thrombocytopenic purpura, a rare, life-threatening blood disorder characterised by blood clotting in small blood vessels throughout the body that can result in organ damage and premature death.

Akantior* (polyhexanide) received a positive opinion for the treatment of acanthamoeba keratitis, a severe, progressive and sight threatening corneal infection characterised by intense pain and photophobia. Acanthamoeba keratitis is a rare disease primarily affecting contact lens wearers.

The committee adopted a positive opinion for Cejemly (sugemalimab) for the treatment of adults with metastatic non-small-cell lung cancer.

A recommendation for a conditional marketing authorisation was made for Durveqtix (fidanacogene elaparvovec), a new gene therapy treatment for haemophilia B, a rare inherited bleeding disorder. This medicine was supported through EMA’s Priority Medicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support for promising medicines with a potential to address unmet medical needs. See more details in the news announcement in the grid below.

Fluenz (influenza vaccine (live attenuated, nasal)) received a positive opinion for the prophylaxis of influenza in children and adolescents from 24 months to less than 18 years of age.

The CHMP gave a positive opinion for GalliaPharm (germanium (68Ge) chloride / gallium (68Ga) chloride), to be used for radiolabelling of various kits used for positron emission tomography (PET) imaging.

Ixchiq (chikungunya vaccine (live)), the first vaccine in the European Union (EU) to protect adults against disease caused by Chikungunya virus transmitted to humans by infected mosquitoes received a positive CHMP opinion. Chikungunya is endemic in many (sub)tropical countries and causes recurrent epidemics. Due to climate change, it may also spread to regions so far spared. Ixchiq was supported through the PRIME scheme and was assessed under the OPEN framework to promote global public health. See more details in the news announcement in the grid below.

The CHMP recommended granting a marketing authorisation for Zegalogue (dasiglucagon) for the treatment of severe hypoglycaemia in adults, adolescents, and children aged six years and over with diabetes mellitus.

A biosimilar medicine, Avzivi (bevacizumab), received a positive opinion for the treatment of carcinoma of the colon or rectum, breast cancer, non-small cell lung cancer, renal cell cancer, epithelial ovarian, fallopian tube or primary peritoneal cancer, and carcinoma of the cervix.

Five generic medicines also received a positive opinion from the committee: Apexelsin (paclitaxel) for the treatment of metastatic breast cancer; Dasatinib Accord Healthcare (dasatinib) for the treatment of chronic myelogenous leukaemia; Pomalidomide Accord (pomalidomide), Pomalidomide Krka (pomalidomide) and Pomalidomide Zentiva (pomalidomide) for treatment of multiple myeloma.

Recommendations on extensions of therapeutic indication for seven medicines

The committee recommended extensions of indication for seven medicines that are already authorised in the EU: Dupixent, Eliquis, Kinpeygo*, Livmarli*, Skyrizi, Tagrisso and Tevimbra.

Withdrawal of applications

One application for an initial marketing authorisation was withdrawn. Kinharto was intended for the treatment of adult patients with symptomatic chronic heart failure and reduced ejection fraction when the heart muscle does not contract effectively.

The application to extend the therapeutic indication of Scenesse in adolescents with erythropoietic protoporphyria, a rare disease that causes intolerance to light, was withdrawn.

Question-and-answer documents on these withdrawals are available in the grid below.

Outcome of re-examination

Following a re-examination, the CHMP confirmed its initial recommendation to refuse the granting of a marketing authorisation for Nezglyal. This medicine was intended for the treatment of cerebral adrenoleukodystrophy, a genetic condition that damages the membrane covering nerve cells in the brain and spinal cord.

Other updates

Evaluation of application to renew marketing authorisation of Translarna

The CHMP has restarted the evaluation of an application to renew the conditional marketing authorisation for Translarna (ataluren), a medicine authorised for the treatment of Duchenne muscular dystrophy.

In January 2024, the CHMP recommended not renewing the conditional marketing authorisation for the medicine, based on its evaluation of all the available data.

The European Commission has now asked the Committee to further consider whether the data available on Translarna are sufficiently comprehensive to conclude on the medicine’s benefit-risk balance, and whether additional real-world data brought to the attention of the Commission during its decision-making process may change the negative outcome previously reached by the CHMP.

In addition, following the appellate judgment of the Court of Justice of the European Union of 14 March 2024 in Case C-291/22 P, EMA has decided to convene a new scientific advisory group on neurology (SAG-N) for Translarna. The assessment is therefore being reset to this stage of the initial renewal procedure. A SAG is a group of scientific experts that is called upon to respond to specific questions posed by EMA’s committees during the evaluation of a medicine. 

A revised CHMP recommendation on the renewal of Translarna’s marketing authorisation is expected in the next few months. The marketing authorisation for Translarna is currently still valid.   

Outcome of assessment to extend the use of Valdoxan

The CHMP has finalised its assessment of an application to extend the use of the antidepressant Valdoxan to include the treatment of adolescents with moderate to severe depression. Although the company decided not to pursue this use, the committee agreed for the product information to be updated to include the study results submitted for the application. A question-and-answer document is available in the grid below.

Reflection paper on Creutzfeldt-Jakob disease

The CHMP adopted the revision of the reflection paper on Creutzfeldt-Jakob disease and plasma-derived and urine-derived medicinal products. The main change in this revision is that it is no longer recommended to exclude donors who have spent at least one year in the United Kingdom (UK) between 1980 and 1996 from donating blood/plasma for fractionation. Cases of variant Creutzfeldt–Jakob disease in the UK declined in the last two decades. The last known UK case was reported in 2016 and no transfusion-transmitted infections have been reported from the UK since 1999.

Agenda and minutes

The agenda of the May 2024 CHMP meeting is published on EMA's website. Minutes of the meeting will be published in the coming weeks.

CHMP statistics

Key figures from the May 2024 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.