UK haemophilia A patients get once-weekly treatment option
A long-acting Factor VIII replacement therapy for haemophilia A from Sobi and Sanofi has been recommended for use by the NHS and could allow patients to reduce the number of injections they need to have.
Final drug guidance published by NICE backs Altuvoct (efanesoctocog alfa) as an option for treating and preventing bleeding in people aged two and over with severe haemophilia A, as an alternative to other FVIII replacement therapies that have to be dosed two or three times a week.
The new drug was approved by the UK medicines regulator on 14th February and is a successor to Sobi's long-established haemophilia A therapy Eloctate (efmoroctocog alfa), also sold by partner Sanofi, which has to be dosed every four days or sometimes even more frequently.
"Current factor VIII replacements can be difficult to manage due to the need for frequent dosing to prevent potentially life-threatening and debilitating bleeding episodes," said Helen Knight, director of medicines evaluation at NICE.
"Combined with its effective bleeding control, [Altuvoct] has the potential to have a significant positive impact for some people with severe haemophilia A," she added.
In clinical trials, weekly injections of Altuvoct as prophylaxis led to 65% of patients reporting zero overall bleeding episodes over the course of a year, while the remaining 35% had a much-reduced rate.
Around 7,700 people in England have haemophilia A, of whom around 1,900 are considered to have severe disease.
Altuvoct is a key product for Sobi and Sanofi, which have seen sales of Eloctate hit hard by competition from Roche's antibody-based Hemlibra (emicizumab) – already approved for NHS use with a broad label in haemophilia A, including patients of all ages – and had global sales of around $5 billion last year.
Once a blockbuster, Eloctate sales have fallen sharply in the last few years and came in at around $385 million last year, while Altuvoct – sold as Altuviiio in the US and some other markets – rocketed 330% to $712 million.
Most of the sales are still in the US market but Sobi and Sanofi expect European and other markets to ramp up rapidly in the next few years, with analysts at Barclays predicting peak sales could reach around $2.4 billion.
The Haemophilia Society said it expects NICE's new guidance to be confirmed in March with Altuvoct becoming available in July. Availability of the product through the NHS in Wales, Scotland and Northern Ireland should follow, it added.
The patient organisation's chief executive Kate Burt said Altuvoct "has the potential to offer better protection against bleeds with less frequent infusions, which should help people focus on living the lives they choose, rather than managing their bleeding disorder."
