- Ultragenyx Pharmaceutical (RARE +2.5%) and development partner Kyowa Kirin International PLC announce that the FDA does not plan on holding an advisory committee meeting to review the Biologics License Application (BLA) seeking approval for burosumab for the treatment of pediatric and adult patients with X-linked hypophosphatemia (XLH). Under Priority Review status, the agency's action date is April 17, 2018.
- Burosumab is a recombinant fully human monoclonal IgG1 antibody that binds to FGF23, a hormone that reduces serum phosphorus and active vitamin D by regulating phosphate excretion and active vitamin D production by the kidney.
- XLH is an inherited disorder akin to rickets but differs in that the ingestion of vitamin D is relatively ineffective.
- Previously: FDA accepts burosumab marketing application for inherited low blood phosphate, action date April 17; co-developer Ultragenyx up 1% (Oct. 10)
- Now read: Ultragenyx Pharmaceutical: A Pullback And Upcoming Regulatory Catalysts
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