Positive European Medicines Agency recommendation for KRN23/burosumab

Excellent news for the European members of our community!

According to a press release today from Ultragenyx and Kyowa Hakko Kirin, "the Committee for Medicinal Products for Human Use (CHMP), the European Medicines Agency's (EMA) scientific committee, has adopted a Positive Opinion recommending the conditional marketing authorization of burosumab, an anti-FGF23 human monoclonal antibody, for the treatment of X-linked hypophosphatemia (XLH) with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons."

There's more work to be done before the treatment will be available to patients in Europe, but this is a huge leap forward! A final decision needs to be made by the European Commission, and is expected in the first quarter of 2018. It will apply to all 28 countries of the European Union, Norway, Iceland and Liechtenstein, but individual countries will still need to do additional reviews, largely with respect to payment issues, rather than safety and efficacy issues.

You can read the entire press release here: http://ir.ultragenyx.com/releasedetail.cfm?ReleaseID=1051923

Filing for KRN23 approval from European Medicines Agency

Ultragenyx has announced that "the European Medicines Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) for KRN23 for the treatment of X-linked hypophosphatemia (XLH). The MAA was filed and accepted in late 2016, and an opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected in the second half of 2017."

You can read the full press release here: http://ir.ultragenyx.com/releasedetail.cfm?ReleaseID=1006547