CHMP recommends EU approval of seven medicines
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, and one biosimilar.
The CHMP recommended granting a marketing authorisation for TiGenix NV’s adipose stem cell therapy Alofisel (darvadstrocel) for the treatment of complex perianal fistulas in patients with Crohn’s disease. Once activated, the allogenic stem cells impair proliferation of lymphocytes and reduce the release of pro-inflammatory cytokines at inflammation sites. This immunoregulatory activity reduces inflammation and may allow the tissues around the fistula tract to heal. Alofisel, which will be marketed outside the US by Takeda Pharmaceutical Co. Ltd’s, according to a 2016 licence deal, has an orphan designation.
The Committee granted 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. The treatment is intended to restore normal phosphate reabsorption from the kidney and increase the production of vitamin D, which enhances intestinal absorption of phosphate and calcium. Ultragenyx and Kyowa Hakko Kirin Co., Ltd. entered into a collaboration and license agreement in August 2013 to develop and commercialise the monoclonal antibody that targets fibroblast growth factor 23. Crysvita has an orphan designation.
Following FDA approval of Mylan’s/Biocon’s biosimilar of Roche’s Herceptin, an additional biosimilar medicine to Roche’s blockbuster antibody is set to cut the revenues of the originator. The CHMP recommended Celltrion Inc’s Her2 targeting antibody Herzuma (trastuzumab) for the treatment of breast and gastric cancer. Herzuma follows on the heels of the EU market authorisation of Samsung Bioepis Co. Ltd.’s Herceptin biosimilar Ontruzant, which was approved for market in November.
In addition, Ozempic (semaglutide) from Novo Nordisk A/S, which recently licenced a diabetes drug from reMYND that prevents death of pancreatic beta cells, received a positive opinion for the treatment of metformin-refractive type 2 diabetes patients.
Futhermore, the CHMP positively assessed a paediatric-use marketing authorisation (PUMA) for Alkindi (hydrocortisone), a replacement therapy of the rare hormonal disorder primary adrenal insufficiency for pediatric use. Hydrocortisone was already authorised in the EU but had lost its patent protection, and thus could be developed specifically for children.
Two generic received a positive opinion from the CHMP: Anagrelide 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.
The CHMP adopted a negative opinion for PharmaMar SA’s Aplidin (plitidepsin) but did not immediately report the reasons. Aplidin was expected to be used to treat multiple myeloma. The application of MedDay Pharmaceuticals SA for an initial marketing authorisation for Qizenday (biotin) was withdrawn. The medicine, which is thought to support remyelination of nerve cells, was intended to be used for the treatment of progressive multiple sclerosis. The CHMP considered that the clinical data of two trials that enroled 253 patients to assess the medicine’s effectiveness were not robust enough, and that „there were uncertainties regarding the medicine’s safety given the small number of patients“ treated with Qizenday. In addition, the Committee said in a press release that more information was needed about how the medicine is absorbed, modified and removed from the body