The U.K.'s National Institute for Health and Care Excellence has backed the use of Kyowa Hakko Kirin Co. Ltd.'s Crysvita within the country's National Health Service.
The reversal comes months after the price watchdog rejected the medicine stating that the results from clinical trials did not show noticeable benefit in children and teenagers. The price watchdog also raised concerns about the treatment's cost effectiveness.
Crysvita, a jointly developed product by Kyowa Kirin and Ultragenyx Pharmaceutical Inc., is intended to treat a rare inherited skeletal disorder known as X-linked hypophosphatemia in children and young people.
The disorder causes low phosphate levels in the blood, leading to soft, weak bones, which can result in life-long physical disabilities and pain.
A final guidance by the regulator is expected to be published on Oct. 24.
This treatment represents a significant improvement for a condition in which there have been no advances in management for 35 years and will be much easier to adhere to compared to current therapy options. It will result in better healing of rickets, linear growth and muscle function among affected individuals," Poonam Dharmaraj, chairperson of the British Paediatric and Adolescent Bone Group, said in a statement.
The drug is already approved in the U.S. and has a conditional approval for the same indication in the EU.