NICE set to reject Kyowa Kirin’s Crysvita for XLH

Drug pricing is still a widespread issue for pharma, and strained healthcare systems have seen many drug makers fall at the mercy of ever tightening budget cuts.

Honing in on the United Kingdom however, it seems that the country's cost-effectiveness watchdog has found its next victim in the form of Japanese pharma group Kyowa Kirin, which is set to receive a NICE rejection for its rare disease therapy Crysvita (burosumab).

The twice-monthly injection was given a conditional approval by the European Medicines Agency (EMA) earlier this year to treat children and young people with X-linked hypophosphataemia (XLH), a genetic disorder characterised by bowed or bent legs, short stature, bone pain and delayed walking.

XLH only affects around 250 children and young people in England, however the disease itself is said to have a substantial physical and emotional impact on the patient due to its debilitating nature.

Interestingly, the current standard of care consists of vitamin D supplements and oral phosphate – to boost low levels of phosphate found in the blood of XLH patients – but there is no treatment which targets the underlying pathophysiology of XLH – until now.

Despite this advancement, the National Institute for Health and Care Excellence’s (NICE) Highly Specialised Technology (HST) committee – which evaluates the price of drugs in the rare disease area – has concluded that the cost-effectiveness estimates are much higher than what would normally be considered value for money.

Crysvita’s annual pricing is conditional on the patient’s weight but has an upper estimated price of an eye watering £388,960 a year for a 17-year-old weighing in at 60kg.

Additionally, NICE has said it’s unsure on the drug’s clinical evidence, confirming that the data is “limited and uncertain” while also highlighting the lack of data available from young people with the disease.

This doesn’t mean to say that NICE won’t change its mind in time – of course, providing a reasonable discount offered on Kyowa Kirin’s behalf would help – but the lack of clinical evidence coupled with a steep price tag might not necessarily put the nail in the coffin.

Take GlaxoSmithKline’s (GSK) gene therapy Strimvelis for example. The drug, which treats children with adenosine deaminase deficiency (ADA-SCID), is easily the most costly therapy approved by NICE, yet the HST committee gave the all-clear despite data “uncertainties”.

If Crysvita follows in Strimvelis’ footsteps, then Kyowa Kirin could see its rare disease therapy approved for use on the UK’s National Health Service (NHS), but that decision will be confirmed in October.