Drug for X-Linked Hypophosphatemia Turned Down in the UK Because of Cost

According to a story from Fierce Pharma, the National Institute for Health Care and Excellence (NICE) has decided to reject the drug Crysvita for coverage on the National Health Service (NHS). The agency concluded that that drug’s clinical benefit was not justified by its extreme price. The drug is intended to treat X-linked hypophosphatemia. There are around 250 patients in the country with the disease.
X-linked hypophosphatemia, also known as X-linked vitamin d-resistant rickets, is a genetic disease that affects the bones. It is a form of rickets, a disease that causes soft and weakened bones in children. This leads to poor growth and a bow-legged gait. Other symptoms include other skeletal abnormalities, bone pain, hearing loss, dental abnormalities, and osteoarthritis. X-linked hypophosphatemia is caused by a genetic mutation that creates a deficiency in the substances necessary to properly mineralize bones, such as phosphate. Treatments include surgery to correct deformities and supplementation of phosphate, but these approaches are limited in effectiveness. Crysvita, the first approved drug specifically for X-linked hypophosphatemia, was approved by the FDA in April for all patients over a year old and won approval in Europe in February. To learn more about X-linked hypophosphatemia, click here.

NICE’s current review is only a draft statement, and it is expected to issue its final determination in October. In the past, NICE has reversed its decisions if drug companies are willing to offer a more reasonable price. Crysvita was developed in partnership by the U.S. company Ultragenyx and Kyowa Hakko Kirin, which is based in Japan. In a statement from Kyowa Hakko Kirin, the company appears willing to negotiate with NICE in order to come to an agreement that will allow the drug to get approved.

A 10 mg dose of Crysvita costs £2,992 and is expected to be given every two weeks, with the dose varying depending on the weight of the patient. In the US, annual treatment costs $160,000 for a child and $200,000 for an adult, and this is only after discounts and rebates.

Hopefully, NICE and the companies involved will be able to come to a suitable arrangement that will allow UK patients to access this life-changing drug.


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