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Givlaari, Rybelsus lead crowded CHMP recommendations

EU regulator delivered 15 positive opinions and no rejections


The latest meeting of the Committee for Medicinal Products for Human Use (CHMP) dished out positive recommendations all round, with no medicines receiving a negative opinion. 

The European Medicines Agency’s (EMA) CHMP recommended an impressive 15 new medicines for approval, as it convened for its first meeting of the year.

Leading the flurry of recommendations was Givlaari (givosiran), Alnylam’s second RNAi drug, for acute hepatic porphyria.

Porphyrias are genetic metabolic disorders caused by an enzyme deficiency in the haem pathway, that leads to a build-up of toxic porphyrin molecules in the body, causing attacks that lead to tissue and organ damage. Hepatic porphyrias are those in which the deficiency occurs in the liver.

Following the CHMP recommendation, it will become the first treatment for acute hepatic prophyria in adults and adolescents aged 12 years and older.

Joining Givlaari with a positive recommendation was Rybelsus (semaglutide), Novo Nordisk’s once-daily oral formulation of its injectable GLP-1 agonist semaglutide.

The diabetes treatment is bolstered not only by its convenience, but also by data which shows that it outperformed other drugs for type 2 diabetes.

In Novo’s Pioneer programme of the drug, people treated with Rybelsus achieved greater blood glucose reductions compared to Merck & Co’s DPP-4 inhibitor Januvia (sitagliptin), Boehringer Ingelheim and Eli Lilly’s SGLT2 inibitor Jardiance (empagliflozin) and Victoza, as well as placebo.

The CHMP also recommended PaxVax’s cholera vaccine Vaxchora for prophylaxis against choleam and Eli Lilly’s new fast-acting mealtime insulin Liumjev, to improve glycemic control in diabetes in adults.

Other notable recommendations include Bayer’s Nubeqa (darolutamide), which has been recommended for approval as a treatment for prostate cancer. Nubeqa will go head-to-head in the non-metastatic castration-resistant prostate cancer market with J&J’s Erleada (apalutamide), as well as Pfizer/Astellas’ Xtandi (enzalutamide).

Biosimilar and generic medicines also had a wildly favourable turn in this month’s recommendations – this includes Pfizer’s biosimilar Ruxience (rituximab) for the treatment of non-Hodgkin’s lymphoma and a host of other indications, as well as azacitidine betapharm (azacytidine) and azacitidine Mylan (azacitidine) for the treatment of a number of blood cancers.

Also of note was the quiet withdrawal of an application for extended use of Keytruda (pembrolizumab) in oesophageal cancer. The EMA reportedly determined that the data supporting the application were not sufficient to prove the treatment increased survival in the targeted population.

Article by
Lucy Parsons

3rd February 2020

From: Regulatory



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