Novo Nordisk nabs FDA nod for hemophilia A drug Esperoct but can’t launch until 2020

Novo Nordisk just added a new therapy to its declining hemophilia stable, but it won’t be able to reap the benefit until 2020.

The FDA has greenlighted Esperoct (turoctocog alfa pegol, N8-GP) for adults and children with hemophilia A, the Danish company said Tuesday. The approval allows Esperoct to be used to reduce the risk of bleeding but also to control bleeding episodes when they occur.

It’s good news for Novo’s hemophilia franchise, which has been under pressure from sliding NovoSeven sales. But citing third-party IP agreements, the company said it won’t be able to launch Esperoct in the U.S. before 2020.

The company’s other antihemophilic factor, NovoEight, previously experienced a similar delay. Novo launched that drug mid-2015, when a third-party patent expired, about a year and a half after it earned the FDA go-ahead.

A long-acting factor VIII, Esperoct is administered once every four days in adults and adolescents or every 3 to 4 days in children. In clinical trials conducted in 270 previously treated patients with severe hemophilia A, the drug held off bleeding episodes to a low median average annualized rate of 1.18. And the safety profile is similar to that of rival drugs, which ODDO BHF analyst Martial Descoutures called "one of the key points."

Current consensus has put Esperoct’s peak sales at 1.86 billion Danish kroner ($284 million) in 2025, according to Descoutures in a Wednesday note to clients. That would constitute about 10% of Novo’s BioPharma franchise but only 1% of the group’s total sales, as the company is heavily weighted toward diabetes.

Esperoct will be launched into a crowded market, though. There’s Shire (now part of Takeda), which has Adynovate, and Sanofi, which nabbed hemophilia A drug Eloctate through its $11.9 billion buyout of Biogen spinoff Bioverativ. Bayer also has a new long-acting factor VIII therapy, Jivi. But all of them face a formidable force in Roche’s new antibody therapy Hemlibra.

The Roche drug has posted strong data, showing it could cut bleeds by 68% compared to standard factor VIII therapy. And it recently marched into the wider market of noninhibitor patients with an FDA nod last October. For 2018, Hemlibra ginned up 224 million Swiss francs ($224 million), with sales virtually doubling every quarter.

Novo has its own antibody in development, concizumab. The drug recently completed a phase 2 trial in hemophilia A and B patients with inhibitors, and showed a reduced annual bleeding rate on prophylaxis compared with on-demand NovoSeven. On the company’s fourth-quarter earnings call, Chief Scientific Officer Mads Krogsgaard Thomsen said the company will start a phase 3 study in the second half of 2019. The new antibody is also under trial in patients without inhibitors.

But that's not all the hemophilia A competition in sight. BioMarin’s gene therapy, valoctocogene roxaparvovec, has already entered phase 3, and Spark Therapeutics is looking to follow with its gene therapy, SPK-8011. In a recent report, Morningstar named BioMarin as an enticing takeover target due to its "explosive growth potential in hemophilia and ultra-orphan disease."

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