Latest news

• 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

  NICE set to reject Kyowa Kirin’ s Crysvita for XLH. Committee says the rare disease therapy isn’t 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

• Rigel and Ultragenyx claim approvals for rare drugs

  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

• Archive listing

  Tavalisse and Crysvita are set to treat patients with chronic ITP and XLH, respectively. .