The Committee deems that the clinical benefit of CRYSVITA (burosumab) 10 mg, 20 mg, 30 mg, solution for injection, is substantial for the treatment of X-linked hypophosphataemia with radiographic signs of bone involvement in children aged one year and over and adolescents in the bone growth phase.
Clinical Added Value
moderate
In view of:
the evidence of the superiority of burosumab monotherapy in relation to conventional treatments, a clinically relevant comparator, after 40 weeks of treatment on improvement of a radiology outcome measure,
the additional effect size deemed to be clinically relevant in a population of paediatric and adolescent patients with severe forms, failing to respond to the conventional treatment,
the additional follow-up provided by the findings at 64 and 88 weeks which suggest continued efficacy with an acceptable safety profile,
the lack of usable quality-of-life data in this high-impact condition on this outcome measure,
the safety profile of burosumab over a longer follow-up period, deemed to be acceptable, compared to the safety profile of conventional treatments, particularly marked by the onset of complications such as ectopic mineralisation, manifested as nephrocalcinosis,
the administration constraints in respect of the conventional treatment which are particularly impactful for paediatric patients and their close family,
the Committee deems thatCRYSVITA 10 mg, 20 mg, 30 mg, solution for injection, provides moderate clinical added value (CAV III) in relation to the conventional treatment for the treatment of X-linked hypophosphataemia with radiographic signs of bone involvement in children aged one year and over and adolescents in the bone growth phase.
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