Novo Nordisk Drug Approved in Europe After FDA Rejection

Last year, Novo Nordisk's new treatment for hemophilia, Concizumab, received approval from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP), despite having been previously rejected by the US Food and Drug Administration (FDA). Meanwhile, PTC Therapeutics faces difficulties with its treatment for Duchenne Muscular Dystrophy following a poor performance in a recent FDA review.

Novo Nordisk's anti-tissue factor pathway inhibitor (TFPI) antibody has been recommended by the CHMP for marketing as a treatment to prevent bleeding in patients with hemophilia A or B who use FVIII or FIX inhibitors. The drug, expected to be marketed under the name Alhemo after receiving final approval from the European Commission, will be the first once-daily subcutaneous prophylactic treatment for hemophilia in patients aged 12 and older with inhibitors.

In hemophilia treatment, the development of inhibitors due to the immune response can limit treatment options. Alhemo is unique because it promotes blood clot formation even in the presence of inhibitors in the patient's body. Stephanie Seremetis, Novo Nordisk's chief medical officer for hemophilia, stated in a press release that the CHMP recommendation represents an "important milestone" for patients. She emphasized that Alhemo would offer hemophiliacs a new option for daily bleeding prevention and noted its ease of use, as it can be delivered via a pre-filled portable pen.

Concizumab, the leading drug candidate against TFPI, had been rejected by the FDA last year. The FDA had requested additional information about the drug's monitoring, patient dosing, and manufacturing process in its response letter. Since then, Pfizer's competitors have obtained FDA approval. Novo Nordisk has resubmitted its application for Concizumab to the FDA, and the drug has been approved in several other countries.

In contrast, the CHMP remains dissatisfied with PTC Therapeutics' Translarna, a drug designed for the treatment of Duchenne Muscular Dystrophy. This marks another setback for Translarna in Europe. The CHMP initially rejected Translarna's full approval last September, citing insufficient efficacy. The same conclusion was reached in another review in January. Although the European Commission requested yet another review in May, the latest negative opinion from the CHMP casts doubt on Translarna's future in Europe.

Matthew B. Klein, MD, CEO of PTC Therapeutics, expressed in a press release that the CHMP's opinion contradicts the wishes of European physicians, patients, and their families. PTC Therapeutics remains committed to ensuring that boys and young men with nonsense mutation Duchenne Muscular Dystrophy have access to Translarna. The company plans to provide the European Commission with all available evidence to support the continued authorization of the drug.

Citi analysts have predicted that the European Commission is likely to accept the CHMP's opinion after its review. Despite receiving conditional authorization in Europe in 2014, the FDA has rejected applications for Translarna twice. PTC Therapeutics has announced plans to apply for FDA approval once again this year.