Europe – NICE U-turn on Crystiva for rare bone disease

Reversing its initial decision to reject the drug, NICE has issued a positive recommendation for Kyowa Kirin’s rare disease drug Crystiva, the first treatment approved to target the underlying pathophysiology of X-Linked Hypophosphataemia (XLH).

The U-turn is good news for the Japan company, which negotiated on the price of the treatment behind closed doors after the NICE’s  conclusionthat the drug wasn’t cost effective.

the UK’s cost-effectiveness watchdog is now set to recommend the twice-monthly injection for the treatment of XLH in children and young people with growing bones, with final guidance expected in October.

Tom Stratford, CEO, Kyowa Kirin International said: It is a major development that NICE has recommended Crysvita for routine use among children and young people with XLH in England and Wales.

“This marks a step change in treatment for XLH, emphasised through the emotional testimonies provided by patient groups and clinicians following the first evaluation consultation.”..