Novo faces delay in US review of haemophilia drug

The FDA has extended its review of Novo's experimental haemophilia A drug denecimig because of ongoing remediation work at a facility that will be used to manufacture the Factor VIIIa mimetic.

The Danish group is seeking approval of denecimig (previously known as Mim8) for the treatment of haemophilia A (congenital Factor VIII deficiency) in adults and children, with or without Factor VIII inhibitors.

In a statement, Novo confirmed that the expected decision during the third quarter of this year had been delayed – without any new timeline set by the FDA – but stressed that the review "is still ongoing" and the agency has not identified any deficiencies in its filing related to the clinical efficacy or safety data.

The drug is described by Novo as a "next-generation" Factor VIIIa mimetic that can be used to prevent or reduce the frequency of bleeding episodes, offering once-monthly, once-every-two-weeks, and once-weekly dosing – depending on the patient's body weight and the physician's assessment – via a single-use, prefilled pen.

Already recommended for approval in Europe under the Frehemgo brand name, denecimig is one of a series of new products Novo is relying on to build revenues in the next few years and spearhead a diversification of its business beyond its main obesity and diabetes franchise, which has driven the company's growth in recent years. Analysts have predicted peak sales of denecimig could reach around $2 billion a year.

Novo said it still plans to launch denecimig in the US in the first half of next year, and is "actively working with the FDA, addressing the agency's requests for remediations related to the manufacturing facility."

Denecimig is a bispecific antibody that works by bridging Factor IXa and Factor X on the surface of activated platelets, promoting thrombin generation and enhancing coagulation to help prevent bleeding. It has a similar mechanism to Roche's NXT007, another Factor VIIIa mimetic drug in phase 3 testing, which is derived from its near $6 billion blockbuster haemophilia A drug Hemlibra (emicizumab).

"Haemophilia A patients should expect more from their standard of care," said Mike Doustdar, Novo's chief executive.

"As Novo prepares to bring denecimig to market, our ambition is to help make better care something they can count on," he added. "Novo is already in the process of addressing the FDA's requests as efficiently as possible and continuing to work closely with the agency toward bringing denecimig to patients in the US."