U.K. cost watchdogs turn away rare disease med Crysvita

U.K.'s NICE rejected Kyowa Kirin's rare disease drug Crysvita due to concerns over cost effectiveness and uncertainties. (Pixabay)

Months after Kyowa Hakko Kirin won a conditional approval for rare disease drug Crysvita in Europe, cost watchdogs in the U.K. have rejected the med due to cost concerns.

While finding the med provides "meaningful clinical benefits," NICE experts ruled in a draft evaluation that the drug's incremental cost-effectiveness ratios 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 and European approval in February.

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The disease can cause skeletal deformities in childhood that continue into adulthood, causing lifelong disability and pain, according to NICE. Patients with the disease also suffer from muscle weakness and fatigue.

The review is only a draft and the findings are open for comment until July 6, according to NICE. Another evaluation meeting is set for later in July. The agency expects to issue final guidance in October. Drugmakers can sometimes win a NICE reversal by offering better pricing.

Kyowa Hakko Kirin, which partnered with U.S. rare disease biotech Ultragenyx for development back in 2013, won conditional approval for the drug in Europe in February. In a statement, a spokesperson said the company welcomes the "open and constructive deliberation undertaken during the first committee meeting, particularly the thoughtful and moving contributions of expert clinicians and patient representatives."

"We hope that following the responses gathered from the existing stakeholders and wider public during the consultation period, and the introduction of a suitable financial arrangement, the committee would be in a position to issue positive guidance for the use of burosumab within the NHS in England," Kyowa Hakko Kirin's representative said via email.

RELATED: Ultragenyx nabs blockbuster FDA nod for $200K-per-year Crysvita

Crysvita costs £2,992 for a 10mg vial, according to NICE. The drug is given every two weeks and doses vary by patient weight.

Ultragenyx won U.S. approval for Crysvita in April and set out to launch a drug that analysts said could bring in blockbuster-level sales. In the U.S., Ultragenyx costs $160,000 per year for children and $200,000 per year for adults after rebates and discounts.

Editor's note: This story was updated with a statement from Kyowa Hakko Kirin.