CHMP approves haemophilia B gene therapy, chikungunya vaccine in May meeting decisions

At the European Medicines Agency's (EMA) recent May gathering, significant recommendations and decisions were made regarding the approval and market applications of various medicines. The Committee for Medicinal Products for Human Use (CHMP), the agency's drug advisory body, endorsed 14 new medications, including one biosimilar and five generics, and supported label extensions for seven existing treatments. They also confirmed a negative opinion on a re-examined drug and noted the withdrawal of two marketing applications. Here’s a detailed overview of the decisions:

New Medicines Approved

Adzynma (rADAMTS13): Takeda’s enzyme replacement therapy for congenital thrombotic thrombocytopenic purpura in both children and adults received a positive recommendation.

Akantior (polyhexanide): SIFI’s treatment for acanthamoeba keratitis was also endorsed.

Cejemly (sugemalimab): CStone Pharmaceuticals’ anti-PD-L1 monoclonal antibody was approved for treating adults with metastatic non-small-cell lung cancer (NSCLC).

Durveqtix (fidanacogene elaparvovec): Pfizer’s gene therapy for haemophilia B was recommended for approval.

Fluenz: AstraZeneca’s live attenuated influenza vaccine for children and adolescents aged 24 months to <18 years was endorsed for influenza prophylaxis.

GalliaPharm (germanium [68Ge] chloride / gallium [68Ga] chloride): Eckert & Ziegler’s product was approved for radiolabelling various kits used in positron emission tomography (PET) imaging.

Ixchiq (chikungunya vaccine [live]): Valneva’s vaccine to protect adults from chikungunya virus, which is transmitted by infected mosquitoes, received approval.

Zegalogue (dasiglucagon): Novo Nordisk’s treatment for severe hypoglycaemia in diabetes patients aged six years and older was endorsed.

Avzivi (bevacizumab): Bio-Thera Solutions’ biosimilar version of Avastin was approved for multiple cancers, including those of the colon, rectum, breast, and kidney.

Generic Medicines: Positive opinions were given to five generic medicines. These include Apexelsin (paclitaxel) for metastatic breast cancer, Dasatinib Accord Healthcare (dasatinib) for chronic myelogenous leukaemia, and generic versions of pomalidomide from Accord Healthcare, Krka, and Zentiva for multiple myeloma treatment.

Extensions of Therapeutic Indication

Dupixent (dupilumab): Sanofi/Regeneron’s drug was recommended as an add-on maintenance treatment for adults with uncontrolled chronic obstructive pulmonary disease (COPD) characterized by raised blood eosinophils.

Eliquis (apixaban): Bristol Myers Squibb/Pfizer’s medication was recommended for treating venous thromboembolism (VTE) and prevention of recurrent VTE in pediatric patients from 28 days to <18 years of age.

Kinpeygo (budesonide): Calliditas Therapeutics/Stada’s treatment for primary immunoglobulin A nephropathy (IgAN) in adults received a positive opinion.

Livmarli (maralixibat): Mirum Pharmaceuticals’ drug was endorsed for treating progressive familial intrahepatic cholestasis in patients aged three months and older.

Skyrizi (risankizumab): AbbVie’s medication was recommended for adults with moderately to severely active ulcerative colitis (UC) who have had an inadequate response or intolerance to conventional or biologic therapy.

Tagrisso (osimertinib): AstraZeneca’s drug was endorsed for use in combination with pemetrexed and platinum-based chemotherapy for first-line treatment of adults with advanced NSCLC with specific EGFR mutations.

Tevimbra (tislelizumab): BeiGene’s drug was recommended for various NSCLC treatments, both alone and in combination with other chemotherapies for adults with locally advanced or metastatic disease.

Negative Opinions and Withdrawals

Nezglyal (leriglitazone): The CHMP confirmed its earlier decision to refuse marketing authorisation for Neuraxpharm and Minoryx Therapeutics' treatment for cerebral adrenoleukodystrophy.

Kinharto (omecamtiv mecarbil): Cytokinetics withdrew its application for this chronic heart failure treatment based on EMA feedback indicating insufficient evidence of benefit over risk.

Scenesse (afamelanotide): Clinuvel withdrew its application to extend use to adolescents with erythropoietic protoporphyria, citing an inability to meet EMA’s comprehensive safety and efficacy data requirements.

These decisions underscore the ongoing efforts of the EMA to ensure the availability of effective and safe medicinal treatments within the European Union.

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