Novo Nordisk's Drug Approved in Europe After FDA Rejection!

Last year, Novo Nordisk's new hemophilia treatment Concizumab won approval from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) after being rejected by the US Food and Drug Administration (FDA). Meanwhile, PTC Therapeutics' treatment for Duchenne Muscular Dystrophy, which performed poorly in a recent FDA review, is in trouble.

Novo Nordisk's anti-tissue factor pathway Inhibitor (TFPI) antibody has been recommended by the CHMP for marketing as A drug to prevent bleeding in hemophilia A or B patients using FVIII or FIX inhibitors. The drug, which is expected to be marketed under the brand name Alhemo after final approval from the European Commission, will be the first once-daily subcutaneous prophylactic treatment for hemophilia in patients 12 years of age and older who are accompanied by inhibitors.

In hemophilia treatment, patients may develop inhibitors due to the immune response, limiting treatment options. Alhemo is unique in that it promotes the formation of blood clots in the presence of inhibitors in the patient's body. Stephanie Seremetis, Novo Nordisk's chief medical officer for hemophilia, said in a press release that the CHMP recommendation is an "important milestone" for patients, noting that Alhemo will provide hemophiliacs with a new option for daily bleeding prevention while being easy to deliver via a pre-filled portable pen.

Concizumab, the lead drug candidate against TFPI, was rejected by the FDA last year. In its response letter, the FDA asked Novo Nordisk to provide more information about the drug's monitoring, patient dosing and manufacturing process. Pfizer's competitors have since gained FDA approval. Novo Nordisk has resubmitted its application for Concizumab to the FDA and it has been approved in several countries.

However, the CHMP has again expressed its displeasure with PTC Therapeutics' Translarna drug, the latest in a series of setbacks for Translarna in Europe. The CHMP first rejected Translarna's full approval last September, citing a lack of efficacy, and reached the same conclusion in another review in January. Although the European Commission requested another review in May, the latest negative opinion from the CHMP could leave Translarna's future in Europe in uncertainty.

Matthew B. Klein, MD, CEO of PTC Therapeutics, said in a press release that the CHMP's opinion is contrary to the wishes of European physicians, patients and their families. The PTC will continue to ensure that boys and young men with the nonsense mutation Duchenne muscular dystrophy have access to Translarna and will provide the European Commission with all possible evidence to support continued authorization.

Citi analysts predicted in the report that the European Commission is likely to accept the CHMP's opinion after its review. Although Translarna received conditional authorization in Europe in 2014, the FDA has rejected applications for the drug twice. PTC Therapeutics said it plans to apply for FDA approval again this year.