Nine medicines recommended for approval, including the first two CAR-T cell therapies in the EU
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended nine medicines for approval, including six orphan medicines, at its June 2018 meeting.
The CHMP recommended granting marketing authorisations for the first two chimeric antigen receptors (CAR) T-cell therapies in the European Union: Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel) are both advanced therapy medicinal products (ATMPs) intended for the treatment of certain blood cancers. Kymriah and Yescarta were designated as orphan medicines during their development. They are also the first medicines supported through EMA’s PRIority MEdicines (PRIME) scheme to receive a positive opinion from the Committee.
Together with the approval recommendation for the first CAR-T cell therapies, the Committee also recommended an extension to the therapeutic indication for RoActemra (tocilizumab) to include the treatment of CAR-T-cell-induced cytokine release syndrome (CRS), a known serious side effect of CAR-T cell therapies.
Four other orphan medicines were recommended for approval by the Committee: Cablivi (caplacizumab) for the treatment of acquired thrombotic thrombocytopenic purpura; Mepsevii (vestronidase alfa) for the treatment of mucopolysaccharidosis type VII; Veyvondi (vonicog alfa) for the treatment of von Willebrand disease and Vyxeos (daunorubicin / cytarabine) for the treatment of acute myeloid leukaemia.
Duzallo (lesinurad / allopurinol) received a positive opinion for the treatment of hyperuricaemia in patients with gout.
The CHMP granted a positive opinion for Ulipristal Acetate Gedeon Richter (ulipristal acetate), an informed consent application for the pre-operative treatment of uterine fibroids. An informed consent application makes use of data from the dossier of a previously authorised medicine, with the marketing authorisation holder of that medicine giving consent for the use of their data in the application.
Positive recommendation on a new medicine following re-examination
The CHMP recommended granting a marketing authorisation for Nerlynx (neratinib), for the adjuvant treatment of adult patients with breast cancer, after re-examining its negative opinion for this medicine adopted in February 2018.
Seven recommendations on extensions of therapeutic indication
The Committee recommended extensions of indications for Dexdor, Inovelon, Jinarc, Lenvima, Opdivo, Rapamune and RoActemra (see above).
Start of referrals
The CHMP started a review of the effectiveness of bacterial lysate medicines in reducing the number and severity of respiratory infections in adults and children who experience repeated infections.
The Committee also started a review of Septanest (articaine / adrenaline solutions for injection) to harmonise the way the medicine is used in the countries where it is available. Septanest is a local anaesthetic used to prevent pain and discomfort in the mouth during dental procedures.
Withdrawal of application
The application for an initial marketing authorisation for Graspa (L-asparaginase) was withdrawn by the company. This medicine was intended to be used for the treatment of acute lymphoblastic leukaemia.