CHMP Recommends 10 Medicines for Approval

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Enzyme replacement therapies for rare diseases and cancer treatments are recommended for approval.


The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency recommended 10 new medicines for approval at its June 2015 meeting, including treatments for rare diseases and cancer.

Two enzyme replacement therapies for the treatment of rare genetic diseases received a positive opinion from the CHMP. The recommend therapies, Synageva BioPharma’s Kanuma (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency and Alexion Europe’s Strensiq (asfotase alfa)-the first therapy for the bone disease hypophosphatasia that started in childhood-have orphan designations.

The CHMP also recommended granting a marketing authorization for Farydak (panobinostat) from Novartis Europharm Ltd, for the treatment of relapsed and/or refractory multiple myeloma. Farydak, the first cancer medicine that targets enzymes known as histone deacetylases, has an orphan designation.

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

The CHMP also recommended Respreeza (human alpha1-proteinase inhibitor) from CSL Behring 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 generalized anxiety disorder; and Pregabalin Accord (pregabalin) for the treatment of epilepsy and generalized 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) from Santhera Pharmaceuticals (Deutschland) GmbH  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 recommended the refusal of a marketing authorization for the advanced therapy medicinal product Heparesc (human heterologous liver cells) from Cytonet GmbH & Co KG. Heparesc was intended to be used for the treatment of certain urea cycle disorders.

Other actions
The Committee recommended extensions of indication for Humira (AbbVie Ltd), Levemir (Novo Nordisk A/S), Perjeta (Roche Registration Ltd) and Voncento (CSL Behring GmbH).

The CHMP recommended several measures, including the introduction of educational material, to ensure that patients and carergivers 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.

Source: European Medicines Agency