After hitting a wall with the FDA last year, Novo Nordisk has picked up backing from European drug reviewers for its novel hemophilia treatment concizumab. Meanwhile, PTC Therapeutics' troubled Duchenne Muscular Dystrophy therapy didn’t fare as well in its latest attempt to pass muster with the agency.
Novo’s anti-tissue factor pathway inhibitor (TFPI) antibody bagged a marketing recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) to prevent bleeding in hemophilia A or B patients with FVIII or FIX inhibitors, respectively.
The drug, which will carry the brand name Alhemo after a final approval from the European Commission, is the first once-daily, under-the-skin prophylactic treatment for those 12 and older living with the disease and inhibitors, according to Novo Nordisk.
Hemophilia patients can develop inhibitors as an immune response to the clotting factors in typical hemophilia treatment, which aims to replace clotting factors through routine intravenous infusions. Such inhibitors can eventually limit patients' treatment options.
Alhemo, for its part, works by allowing blood clots to form even in the presence of inhibitors, according to the company.
The CHMP opinion is a “major milestone” for patients, Novo’s chief medical officer for hemophilia, Stephanie Seremetis, said in a company press release.
“If approved, Alhemo would be an important addition to our growing hemophilia portfolio as it offers the potential of everyday prophylaxis to prevent bleeds for people living with hemophilia who have developed the complication of inhibitors,” Seremetis added, noting that the drug is conveniently delivered in a pre-filled portable pen.
Concizumab was once the leading candidate in the anti-TFPI race until the FDA rejected the therapy last year. In its complete response letter, the U.S. agency requested additional information around proposed monitoring and dosing of patients and the drug's manufacturing process. Since then, a rival from Pfizer has gained an FDA nod.
Novo has refiled concizumab with the FDA, Novo’s development chief Martin Holst Lange told investors during the company’s second-quarter earnings call. So far, the drug is approved in Canada, Australia, Switzerland and Japan.
While Novo collected good news from CHMP's latest review cycle, the committee once again turned its nose up at PTC Therapeutics’ Translarna, the latest in a series of European setbacks for the drug.
CHMP first rejected a full approval last September, citing a lack of efficacy. Another review in January bore the same results, but the European Commission requested another examination in May, leaving Translarna on the market in the meantime.
Now, with CHMP’s latest negative opinion, the European Commission (EC) is expected to make the final call in approximately 67 days.
“The CHMP opinion is clearly against the expressed wishes of physicians, patients and families throughout Europe," said PTC’s CEO Matthew B. Klein, M.D., in a release. "As Translarna will remain authorized in Europe pending review by the European Commission, we will continue to ensure Translarna is available to boys and young men living with nonsense mutation Duchenne muscular dystrophy. In addition, PTC will ensure the EC has all possible evidence to support continued authorization."
Citi analysts expect the EC to “likely ratify” CHMP’s opinion following its review, the analysts wrote in a note to clients.
While it picked up its initial conditional authorization in Europe in 2014, the FDA has rejected the drug twice. PTC in March said it plans to file for potential FDA approval again this year.