Latest news

• SMC enables access to Kyowa Kirin’s Crysvita for rare bone disease

  The trial evaluated Crysvita in comparison to conventional therapy with oral phosphate and active vitamin D. ... An application for the extended use of Crysvita in adults with XLH is also currently under review by the EMA.

• NICE U-turn on Crystiva for rare bone disease

  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. ... Crysvita targets this pathway by blocking the activity of

• Orphan drugs dominate UK Prix Galien shortlist, medtech debuts

  Seven companies have been shortlisted for the Orphan Product Award; Amicus (Galafold), Biogen (Spinraza), Biomarin (Brineura), Eusa (Qarziba), Intercept (Ocaliva), Kyowa Kirin (Crysvita) and Shire (Revestive).

• NICE set to reject Kyowa Kirin’s Crysvita for XLH

  is set to receive a NICE rejection for its rare disease therapy Crysvita (burosumab). ... 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

• Rigel and Ultragenyx claim approvals for rare drugs

  Tavalisse and Crysvita are set to treat patients with chronic ITP and XLH, respectively. ... Ultragenyx’s FDA approval was for Crysvita (burosumab), which has become the first approved drug for adults and children ages one year and older with X-linked