Favourable opinion for reimbursement only in “the treatment of adults with chronic hypoparathyroidism in whom parathyroid hormone (PTH) replacement therapy is necessary and optimal control is not achieved with vitamin and calcium treatment”
Unfavourable opinion for reimbursement in the other situations covered by the MA indication.
Clinical Benefit
Substantial
The Committee deems that the clinical benefit of YORVIPATH (palopegteriparatide) is substantial in the treatment of adults with chronic hypoparathyroidism in whom parathyroid hormone (PTH) replacement therapy is necessary and optimal control is not achieved with vitamin and calcium treatment
Insufficient
Clinical Added Value
minor
Considering:
evidence in a phase 3 trial of the superiority of YORVIAPTH (palopegteriparatide) compared to placebo, as an adjunct to vitamin and calcium treatment, on a composite endpoint including albumin-adjusted serum calcium levels within the normal range, independence from hydroxylated vitamin D derivatives, independence from calcium supplementation and no increase in prescribed study treatment, in 78.8% of patients in the palopegteriparatide group versus 4.8% of patients in the placebo group, p<0.0001;
evidence of the superiority of palopegteriparatide also on the ranked secondary endpoints of quality of life, via the HPES score and the SF-36;
the safety profile that appears to be favourable, primarily marked by moderate adverse events and mainly injection site reactions and headaches;
uncertainties with respect to the prevention of renal complications or neuromuscular symptoms, not demonstrated in the study, particularly considering the short follow-up period of patients in the phase 3 study, i.e. 26 weeks;
the medical need partially met by the available alternatives, but inadequately met in patients in whom optimal control is not achieved with standard therapy, i.e. calcium and vitamin D treatment;
the Committee deems that YORVIAPTH (palopegteriparatide) provides a minor clinical added value (CAV IV) in the current care pathway for adults with chronic hypoparathyroidism in whom parathyroid hormone (PTH) replacement therapy is necessary and optimal control is not achieved with vitamin and calcium treatment.
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