VOYDEYA (danicopan) - Paroxysmal nocturnal haemoglobinuria

The Committee deems that the clinical benefit of VOYDEYA 50 mg and 100 mg (danicopan) film-coated tablet is substantial only “as an add-on to ravulizumab or eculizumab in the treatment of adult patients with PNH who have symptomatic haemolytic anaemia after treatment with a C5 inhibitor for at least 6 months”.

Considering:

• evidence of the superiority of VOYDEYA (danicopan) in combination with a C5 inhibitor (ravulizumab or eculizumab) versus continuation of a C5 inhibitor alone, in terms of increase in haemoglobin levels, reduction in transfusion needs and change in the FACIT-fatigue score after 12 weeks, in adults with PNH who have residual haemolytic anaemia after treatment with a C5 inhibitor for at least 6 months,
• the clinical relevance of these assessment criteria,
• the high effect size observed for haemoglobin levels and reduction in transfusion needs,

And despite:

• uncertainties with respect to the clinical relevance of the benefit observed for the FACIT-fatigue score and the absence of a benefit demonstrated on a quality of life score,
• the absence of comparative data beyond 12 weeks in a context of chronic disease with potentially lifelong treatment, in order to be able to assess maintenance of the benefits and the safety profile of danicopan in the long term, along with its impact on the incidence of thrombotic events, the leading cause of death in patients,
• the absence of robust comparative data versus pegcetacoplan, a treatment already available as second-line therapy after a C5 inhibitor, in particular the absence of a direct comparison, which would nonetheless have been justified on the date of the present assessment given their concomitant development; to date, it cannot be assumed that there is a lower risk of serious haemolysis with danicopan in combination with a C5 inhibitor than with pegcetacoplan, in view of their similar mechanisms of action,

the Committee deems that VOYDEYA 50 mg and 100 mg (danicopan) film-coated tablet
provides a moderate clinical added value (CAV III) as an adjunct to ravulizumab or eculizumab in the treatment of adult patients with PNH who have symptomatic haemolytic anaemia after treatment with a C5 inhibitor for at least 6 months, in the same way as ASPAVELI (pegcetacoplan).