ALTUVOCT (efanesoctocog alfa) - Haemophilia A

Opinions on drugs - Posted on Dec 05 2024

Reason for request

First listing

Summary of opinion

Favourable opinion for reimbursement in the MA indication: “Treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency). ALTUVOCT can be used for all age groups.”

Clinical Benefit

Substantial

The Committee deems that the clinical benefit of ALTUVOCT 250 IU, 500 IU, 750 IU, 1,000 IU, 2,000 IU, 3,000 IU and 4,000 IU (efanesoctocog alfa) powder and solvent for solution for injection is substantial in the MA indication.

Clinical Added Value

no clinical added value

Considering:
efficacy results in the two pivotal studies (XTEND-1 and XTEND-Kids) demonstrating a clinically relevant efficacy of ALTUVOCT (efanesoctocog alfa) as routine weekly prophylaxis, with a mean annualised bleeding rate under treatment of less than 1 in bothstudies, as well as in the treat ment of bleeding episodes and the perioperative management of bleeding,
the favourable safety profile of ALTUVOCT (efanesoctocog alfa) in these studies, particularly in terms of immunogenicity,
the favourable pharmacokinetic profile in view of its longer half-life in comparison with other FVIIIs,

but also considering:

the non-randomised, open-label design of the studies,
the lack of robust data demonstrating a clinical benefit with ALTUVOCT (efanesoctocog alfa) compared to the available alternatives (FVIII concentrates for the prophylaxis and treatment of bleeding, and emicizumab for prophylaxis only), in terms of either efficacy or safety, given:
- the methodological limitations of intra-patient comparison of routine prophylaxis with efanesoctocog alfa versus prophylaxis with other FVIIIs (historic data from a previous pro spective observational study) conducted in the XTEND-1 study, the only comparative analy sis versus the alternatives scheduled in the protocols of the two pivotal studies,
- the methodological weaknesses of the indirect comparisons provided versus FVIII and emi cizumab as prophylaxis,
the lack of evidence of a benefit on quality of life compared to the available alternatives, partic ularly for routine prophylaxis in a context of current availability of emicizumab and a FVIII that can be administered every 5 days according to its RCP.

the Committee deems that ALTUVOCT 250 IU, 500 IU, 750 IU, 1,000 IU, 2,000 IU, 3,000 IU and 4,000 IU (efanesoctocog alfa) powder and solvent for solution for injection provides no clinical added va