Fifteen new medicines recommended for approval

EMA’s human medicines committee (CHMP) recommended fifteen medicines for approval at its January 2020 meeting.

The Committee recommended granting a marketing authorisation for Givlaari* (givosiran), the first treatment for acute hepatic porphyria in adults and adolescents aged 12 years and older. Acute hepatic porphyria is a rare life-threatening genetic condition that causes attacks of severe abdominal pain, vomiting and nervous system disorders, such as seizures, depression and anxiety.

Givlaari benefited from support of the PRIME scheme, EMA’s platform for early and enhanced dialogue with developers of promising new medicines that address unmet medical needs. This interaction led to a more robust application package to demonstrate the medicine’s benefits and risks, which allowed its accelerated assessment.

The CHMP adopted a positive opinion for Rybelsus (semaglutide) for the treatment of adults with insufficiently controlled type 2 diabetes to improve glycaemic control as an adjunct to diet and exercise. It is the first glucagon-like peptide (GLP-1) receptor agonist treatment – a class of non-insulin medicines for people with type 2 diabetes – developed for oral use, providing patients with another option to treat the disease without injections.

The Committee recommended granting a marketing authorisation for Vaxchora (Cholera vaccine (recombinant, live, oral)) for prophylaxis against cholera, a very serious disease caused by Vibrio cholerae, in adults and children.

Liumjev (insulin lispro) received a positive opinion from the CHMP for the treatment of diabetes mellitus in adults.

The CHMP recommended granting marketing authorisations for Nilemdo (bempedoic acid) and Nustendi (bempedoic acid / ezetimibe) for the treatment of primary hypercholesterolaemia (high blood cholesterol that has no identifiable cause) and mixed dyslipidaemia (abnormally levels of fat in the blood).

Nubeqa (darolutamide) received a positive opinion for the treatment of prostate cancer.

The CHMP adopted a positive opinion for Staquis (crisaborole) for the treatment of atopic dermatitis.

The biosimilar medicine Ruxience (rituximab) received a positive opinion for the treatment of non-Hodgkin’s lymphoma, chronic lymphocytic leukaemia, rheumatoid arthritis, granulomatosis with polyangiitis and microscopic polyangiitis, and Pemphigus vulgaris.

The CHMP recommended granting marketing authorisations for four generic medicinesAzacitidine betapharm (azacytidine) and Azacitidine Mylan (azacitidine), for the treatment of myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia; Arsenic trioxide Mylan (arsenic trioxide), for the treatment of acute promyelocytic leukaemia; and Cinacalcet Accordpharma (cinacalcet), for the treatment of secondary hyperparathyroidism, parathyroid carcinoma and primary hyperparathyroidism.

The CHMP recommended granting marketing authorisations for two hybrid medicinesBudesonide/Formoterol Teva Pharma B.V. (budesonide / formoterol fumarate dihydrate), for the treatment of asthma and chronic obstructive pulmonary disease; and Trepulmix* (treprostinil sodium), for the treatment of chronic thromboembolic pulmonary hypertension. Hybrid applications rely in part on the results of pre-clinical tests and clinical trials of an already authorised reference product and in part on new data.

Six recommendations on extensions of therapeutic indication

The Committee recommended extensions of indication for AmeluzMabTheraRezolstaSuliquaTybost and Venclyxto.

Withdrawals of applications

The application for an initial marketing authorisation for Idhifa (enasidenib) was withdrawn. This medicine was intended for the treatment of acute myeloid leukaemia, a cancer of white blood cells.

The application to extend the use of Keytruda (pembrolizumab) in the treatment of cancer of the oesophagus was also withdrawn.

Posted on the EMA website on 31 January 2020