EMA recommends seven new drugs for market

About 5% to 7% of patients with hemophilia A develop a resistance to treatment with recombinant factor VIII products because they carry so-called factor VIII inhibitors. At its January 2018 meeting, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended Roche’s first-in-class antibody emicizumab (brand name Hemlibra) to treat this patient group. The recommendation was based on the results of two Phase III studies that enroled about 160 patients. Roche was granted accelerated approval because Hemlibra ist the first new drug for this subpopulation that has been developed in the past 20 years.

On the orphan drug side, The CHMP positively assessed the long-term enzyme replacement therapy velmanase alfa (brand name: Lamzede), developed by Chiesi Farmaceutica to treat patients with mild to moderate forms of alpha-mannosidosis. As the pivotal study involved only 25 patients and alpha-mannosidosis is very rare, the CHMP recommended market approval under exceptional circumstances, that is it doesn’t expect Chiesi to table the same evidence for efficacy than for more common diseases.

Furthermore, the Committee recommended granting a marketing authorisation for Shingrix, a recombinant, adjuvanted Herpes zoster vaccine developed by GlaxoSmithkline Biologicals SA to prevent herpes zoster and post-herpetic neuralgia in adults 50 years of age or older. 

In the highly competitive market for type 2 diabetes, three drug candidates developed by Merck Sharp & Dohme took the CHMP hurdle: Segluromet (ertugliflozin / metformin), Steglatro (ertugliflozin)  as well as Steglujan (ertugliflozin / sitagliptin). Also, one biosimilar type II diabetes medicine was recommended for approval by the Committee: Semglee (insulin glargine) developed by Mylan S.A.S. 

The CHMP confirmed its previous positive opinion and recommended the granting of a marketing authorisation for Lokelma (sodium zirconium cyclosilicate), for the treatment of hyperkalaemia. Its developer, AstraZeneca AB, had already received a positive opinion in February 2017. However, the European Commisson suspended its decision-making following some concerns relating to good manufacturing practices (GMP) but a new inspection proved GMP compliance.

In contrast, the Committee adopted a negative opinion for EnCyzix (enclomifene) due to bad documentation of symptom-related outcomes.  The drug menufactured by Renable Pharma Ltd was expected to be used to treat hypogonadotrophic hypogonadism in men.

The CHMP also announced the start of the re-examination of PharmaMar’s cancer med Aplidin (plitidepsin), which got a negative opinion at the Committee’s December 2017 meeting.