Latest news

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

  XLH is usually first observed in infants and is characterised by bowed or bent legs, a short stature, bone pain and delayed walking. ... 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

  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.”. ... Characterised by bowed or bent legs, a short stature, bone pain and

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

  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 ... XLH only affects around 250 children and young

• 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