Ten new medicines, including two enzyme replacement therapies for rare diseases, recommended for approval

Ten new medicines have been recommended for approval at the June 2015 meeting of the Committee for Medicinal Products for Human Use (CHMP).

Two enzyme replacement therapies for the treatment of rare genetic diseases received a positive opinion from the CHMP: Kanuma (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency, and Strensiq (asfotase alfa), the first therapy for the bone disease hypophosphatasia that started in childhood. For more information on these two enzyme replacement therapies, both of which have an orphan designation, please see the press releases in the grid below.

The CHMP also recommended granting a marketing authorisation for Farydak (panobinostat) for the treatment of relapsed and/or refractory multiple myeloma. Farydak is the first cancer medicine that targets enzymes known as histone deacetylases. Farydak has an orphan designation. For more information on Farydak, please see the press release in the grid below.

Odomzo (sonidegib) received a position opinion from the CHMP for the treatment of basal cell carcinoma.

The CHMP also recommended Respreeza (human alpha1-proteinase inhibitor) for the treatment of alpha1-proteinase inhibitor deficiency.

Three generic medicines received positive opinions from the CHMP: Docetaxel Hospira UK Limited (docetaxel) for the treatment of breast cancer, non-small cell lung cancer, prostate cancer, gastric adenocarcinoma, and head and neck cancer, Duloxetine Zentiva (duloxetine) for the treatment of major depressive disorder, diabetic peripheral neuropathic pain and generalised anxiety disorder and Pregabalin Accord (pregabalin) for the treatment of epilepsy and generalised anxiety disorder.

Two ‘hybrid’ medicines received positive opinions from the Committee: Aripiprazole Sandoz (aripiprazole) for the treatment of schizophrenia and the prevention and treatment of manic episodes in bipolar 1 disorder, and Raxone (idebenone) for the treatment of visual impairment in patients with Leber’s hereditary optic neuropathy. Raxone has an orphan designation. Hybrid applications rely in part on the results of pre-clinical tests and clinical trials carried out with the reference product and in part on new data.

Negative opinion on new medicine

The CHMP adopted a negative opinion recommending the refusal of a marketing authorisation for the advanced therapy medicinal product Heparesc (human heterologous liver cells). Heparesc was intended to be used for the treatment of certain urea cycle disorders.

Four recommendations on extensions of therapeutic indication

The Committee recommended extensions of indication for Humira, Levemir, Perjetaand Voncento. For more information on the extension of indication for Humira, please see the press release in the grid below.

Outcome of review on adrenaline auto-injectors

The CHMP recommended several measures, including the introduction of educational material, to ensure that patients and carers use adrenaline auto-injectors successfully. An adrenaline auto-injectors are potentially life-saving treatments for anaphylaxis (severe allergic reactions) while the patient waits for emergency medical assistance. For more information on the outcome of this review, please see the public health communication in the grid below.


Posted on the EMA website on 26 June 2015