US FDA approves XLH drug Crysvita

The US Food and Drug Administration has approved Crysvita (burosumab), the first drug cleared to treat adults and children ages 1 year and older with x-linked hypophosphatemia (XLH), a rare, inherited form of rickets.

The FDA granted approval of Crysvita to US ultra-rare diseases focussed biotech firm Ultragenyx Pharmaceutical (Nasdaq: RARE), which has certain rights to the drug from originator Japan’s Kyowa Hakko Kirin (TYO: 4151). In February this year, Crysvita received a positive European Commission decision granting a conditional marketing authorization for the treatment of XLH.

The news was welcomed by investors, as Ultragenyx’ share rose as much as 6.1% to $57.92 in Tuesday trading, though they closed up just 1.6% at $54.68.