Regulatory advisors in Europe have backed the approval of 10 new treatments, including two enzyme replacement therapies for rare diseases, cancer drugs, generics and ‘hybrid’ medicines.

Among this month’s batch of Committee for Medicinal Products for Human Use recommendations is Alexion’s Strensiq (asfotase alfa), the first therapy for hypophosphatasiais - a rare inherited metabolic disorder affecting the bones - in patients who have developed the disease in childhood.

Strensiq, which has also been designated orphan drug status, is being backed under ‘exceptional circumstances’ because safety and efficacy data are still limited due to the extreme rarity of the disease. Therefore, the CHMP has requested that further data be collected and submitted following authorisation.

Synageva BioPharma, which was acquired by Alexion last month, saw its enzyme replacement therapy Kanuma (sebelipase alfa) win favour as a treatment of lysosomal acid lipase deficiency, a rare and life-threatening genetic disease that can cause multiple organ damage. There is currently no approved therapy for this condition.

A marketing authorisation was also recommended for Novartis’ Farydak (panobinostat) for the treatment of relapsed and/or refractory multiple myeloma. 

The drug, which also carries orphan designation, is the first cancer medicine to target enzymes known as histone deacetylases. Clinical trials showed that adding Farydak to Janssen’s Velcade (bortezomib) and dexamethasone slowed disease progression in patients with relapsed and/or refractory multiple myeloma who have received at least two prior standard therapies.

Elsewhere, Novartis’ Odomzo (sonidegib) picked up a positive opinion for basal cell carcinoma, while CSL Behring’s Respreeza (human alpha1-proteinase inhibitor) was put forward as a therapy for alpha1-proteinase inhibitor deficiency.

From the generic field, the CHMP supported approval of: Hospira’s Docetaxel Hospira UK (docetaxel) for breast cancer, non-small cell lung cancer, prostate cancer, gastric adenocarcinoma, and head and neck cancer; Zentiva’s Duloxetine Zentiva (duloxetine) for major depressive disorder, diabetic peripheral neuropathic pain and generalised anxiety disorder; and Accord Healthcare’s Pregabalin Accord (pregabalin) for the treatment of epilepsy and generalised anxiety disorder.

Two ‘hybrid’ medicines (when authorisation depends partly on test results with the reference medicine and partly on new data from clinical trials) received positive opinions from the Committee: Sandoz’ Aripiprazole Sandoz (aripiprazole) for the treatment of schizophrenia and the prevention and treatment of manic episodes in bipolar 1 disorder, and Santhera’s Raxone (idebenone) for the treatment of visual impairment in patients with Leber’s hereditary optic neuropathy.

On the downside, the Committee rejected Cytonet’s Heparesc (human heterologous liver cells) as a treatment for certain urea cycle disorders, because concerns on the design and conduct of the studies cast doubt on data, it said.