CHMP backs Novo's haemophilia A drug Frehemgo

Novo has moved a step closer to EU approval of its long-acting haemophilia A therapy, Frehemgo, after it was backed by the EMA's human medicines committee.

Frehemgo (denecimig, also known as Mim8) – which picked up its first world approval in Saudi Arabia a few days ago – has been recommended by the CHMP for the treatment of haemophilia A (congenital Factor VIII deficiency) in adults and children, with or without Factor VIII inhibitors.

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.

The bispecific antibody 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 – and a follow-up to – its blockbuster haemophilia A therapy Hemlibra (emicizumab). Hemlibra has transformed the market and made CHF 4.8 billion ($5.81 billion) in sales last year.

In the pivotal FRONTIER 2 trial, Frehemgo reduced annualised bleeding rate compared to prior clotting factor prophylaxis and on-demand treatment in people with haemophilia A, with no safety issues in patients who switched to the drug from Hemlibra.

The positive opinion from the CHMP sets up a possible EU approval in the coming weeks. Novo said it expects to launch Frehemgo in its first European markets before the end of the year, with a wider rollout in 2027. It is also under regulatory review in the US and China.

It's some good news for the Danish group after some high-profile disappointments in its late-stage pipeline – including failed trials for its IL-6 inhibitor ziltivekimab in heart failure and atherosclerosis, and the discontinuation of CB1 inverse agonist monlunabant – as it comes under competitive pressure in its core diabetes and weight-loss markets.

Novo's chief executive, Mike Doustdar, said that Frehemgo's combination of "strong bleed protection, flexible dosing frequency, and a prefilled pen offers a differentiated treatment option that can reduce treatment burden and give people with haemophilia A greater freedom in managing their disease."

Analysts have suggested that peak sales of Frehemgo could approach $2 billion a year at peak.