NICE does not recommend Crysvita as a treatment for X-linked hypophosphatemia.-Kyowa Hakko Kirin.

The National Institute for Health and Care Excellence (NICE) recommended in a draft evaluation that the the incremental cost-effectiveness ratios of Crysvita are above ranges "that can be considered an effective use of NHS resources for highly specialized technologies." The agency also expressed concerns with uncertainties around the rare disease drug.

Crysvita treats X-linked hypophosphatemia, or XLH, a hereditary skeletal disease triggered by excess production of the hormone fibroblast growth factor 23. There are about 250 patients with the disease in the U.K., according to NICE. Traditional treatment includes managing symptoms, plus phosphate and vitamin D supplements. The drug won U.S. approval in April 2018 and European approval in February 2018. Another evaluation meeting is set for later in July. The agency expects to issue final guidance in October. It is open to Kyowa Hakko Kirin to offer more attractive pricing to NICE.