CHMP nods for haemophilia B gene therapy, chikungunya vaccine lead decisions from May meeting

31 May 2024
Drug ApprovalVaccineClinical Result
At its May meeting, the European Medicines Agency's drug advisory body recommended 14 new medicines for approval, including one biosimilar and five generics, while it also backed extended labelling for seven treatments. In addition, the Committee for Medicinal Products for Human Use (CHMP) confirmed a negative opinion following a re-examination, and there were two marketing application withdrawals. All of the decisions can be found detailed below.
Positive recommendations on new medicines:
Takeda’s enzyme replacement therapy Adzynma (rADAMTS13) for the treatment of children and adult patients with congenital thrombotic thrombocytopenic purpura.
SIFI’s Akantior (polyhexanide) for the treatment of acanthamoeba keratitis.
CStone Pharmaceuticals’ anti-PD-L1 monoclonal antibody Cejemly (sugemalimab) for the treatment of adults with metastatic non-small-cell lung cancer (NSCLC).
Pfizer’s gene therapy Durveqtix (fidanacogene elaparvovec) for the treatment of haemophilia B.
AstraZeneca’s Fluenz (influenza vaccine [live attenuated, nasal]) for the prophylaxis of influenza in children and adolescents from 24 months to
Eckert & Ziegler’s GalliaPharm (germanium [68Ge] chloride / gallium [68Ga] chloride) for radiolabelling of various kits used for positron emission tomography (PET) imaging.
Valneva’s Ixchiq (chikungunya vaccine [live]) to protect adults against disease caused by Chikungunya virus transmitted to humans by infected mosquitoes.
Novo Nordisk’s Zegalogue (dasiglucagon) for the treatment of severe hypoglycaemia in adults, adolescents and children aged six years and over with diabetes mellitus.
Five generic medicines also received a positive opinion: Apexelsin (paclitaxel) for the treatment of metastatic breast cancer; Dasatinib Accord Healthcare (dasatinib) for the treatment of chronic myelogenous leukaemia; and generic versions of pomalidomide from Accord Healthcare, Krka and Zentiva for treatment of multiple myeloma.
Positive recommendations on extensions of therapeutic indication:
Sanofi/Regeneron’s Dupixent (dupilumab) as an add-on maintenance treatment in adults with uncontrolled chronic obstructive pulmonary disease (COPD) characterised by raised blood eosinophils.
Bristol Myers Squibb/Pfizer’s Eliquis (apixaban) to include the treatment of venous thromboembolism (VTE) and prevention of recurrent VTE in paediatric patients from 28 days to
Calliditas Therapeutics/Stada’s Kinpeygo (budesonide) for the treatment of adults with primary immunoglobulin A nephropathy (IgAN).
Mirum Pharmaceuticals’ Livmarli (maralixibat) to include progressive familial intrahepatic cholestasis in patients 3 months of age and older.
AbbVie’s Skyrizi (risankizumab) for the treatment of adults with moderately to severely active UC who have had an inadequate response, lost response, or were intolerant to either conventional or biologic therapy.
AstraZeneca’s Tagrisso (osimertinib) in combination with pemetrexed and platinum-based chemotherapy for the first-line treatment of adults with advanced NSCLC whose tumours have EGFR exon 19 deletions or exon 21 (L858R) substitution mutations.
BeiGene’s Tevimbra (tislelizumab) alone for the treatment of adults with locally advanced or metastatic NSCLC after prior platinum-based therapy; in combination with pemetrexed and platinum‑containing chemotherapy for the first-line treatment of certain adults with non-squamous NSCLC whose tumours have PD-L1 expression on ≥50% of tumour cells with no EGFR or ALK positive mutations; and in combination with carboplatin and either paclitaxel or nab-paclitaxel for the first-line treatment of certain adults with squamous NSCLC.
Negative opinions on new medicines:
The CHMP confirmed its initial recommendation to refuse the granting of a marketing authorisation for Neuraxpharm and Minoryx Therapeutics’ Nezglyal (leriglitazone) for the treatment of cerebral adrenoleukodystrophy.
Withdrawal of applications:
Cytokinetics pulled a filing seeking approval of Kinharto (omecamtiv mecarbil) for the treatment of adults with symptomatic chronic heart failure and reduced ejection fraction. The decision was based on feedback from the EMA that supporting results were not sufficient to conclude that the benefits of the cardiac myosin activator outweigh the risks. Cytokinetics recently outlined plans to conduct another study of Kinharto.
Clinuvel withdrew an application to expand use of Scenesse (afamelanotide) into adolescents with erythropoietic protoporphyria. The drug has been approved in this indication in the EU since 2014 for adults. However, the company determined that it was not possible to provide the comprehensive safety and efficacy data expected by the EMA in the expanded population.
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