Seven medicines recommended for approval, including an advanced therapy

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended seven medicines for approval at its December 2017 meeting, including two orphan medicines, one of which is also an advanced therapy medicinal product (ATMP).

The CHMP recommended granting a marketing authorisation for the ATMP Alofisel (darvadstrocel), for the treatment of complex perianal fistulas in patients with Crohn’s disease. Alofisel has an orphan designation.

The Committee recommended granting a paediatric-use marketing authorisation (PUMA) for Alkindi (hydrocortisone), for the treatment of primary adrenal insufficiency, a rare hormonal disorder in infants, children and adolescents. PUMAs can be granted for medicines which are already authorised but no longer under patentprotection, and have been developed specifically for children.

The CHMP recommended granting a conditional marketing authorisation for Crysvita (burosumab), for the treatment of X-linked hypophosphataemia with radiographic evidence of bone disease in children and adolescents with growing skeletons. Crysvita has an orphan designation.

Ozempic (semaglutide) received a positive opinion for the treatment of type 2 diabetes.

One biosimilar medicine was recommended for approval by the Committee: Herzuma (trastuzumab), for the treatment of breast and gastric cancer.

Two generic medicines received a positive opinion from the CHMPAnagrelide Mylan (anagrelide), for the reduction of elevated platelet counts in at risk essential thrombocythaemia patients; and Efavirenz/Emtricitabine/Tenofovir disoproxil Krka (efavirenz / emtricitabine / tenofovir disoproxil), for the treatment of HIV infection.

Negative opinion on new medicine

The CHMP adopted a negative opinion for Aplidin (plitidepsin). Aplidin was expected to be used to treat multiple myeloma.

Three recommendations on extensions of therapeutic indication

The Committee recommended extensions of indications for Taltz, Truvada and Yervoy.

Outcome of review on mycophenolate

The CHMP concluded that current evidence does not indicate a risk of malformations or miscarriages during pregnancy when the father has taken mycophenolate medicines (used to prevent rejection of transplanted organs), although the risk of genotoxicity cannot be completely ruled out. For male patients, the CHMP now recommends that either the male patient or his female partner use reliable contraception (it is no longer required that they both use contraception). For female patients, the risk is unchanged.

Withdrawal of application

The application for an initial marketing authorisation for Qizenday (biotin) was withdrawn. This medicine was intended to be used for the treatment of progressive multiple sclerosis.

Posted on the EMA website on 15 December 2017