• News

    FDA Approves Burosumab Therapeutic Treatment for X-linked Hypophosphatemia

    Date: April 19, 2018

    Three decades of research and clinical trials led by ASBMR members, including ASBMR President, Michael Econs, MD, Kenneth E. White, PhD, Erik A. Imel, MD and Thomas Carpenter, MD, and several others, have culminated with FDA approval on April 17th of a new drug for patients with X-linked hypophosphatemia (XLH), an uncommon phosphate wasting disorder that results in rickets and osteomalacia.  The drug, burosumab, is being brought to market by Ultragenyx Pharmaceuticals in collaboration with Kyowa Hakko Kirin under the brand name Crysvita to treat XLH. Patients are typically diagnosed as children and have lower extremity deformities, short stature, and experience bone pain and dental abscesses. Clinical trials have demonstrated that burosumab normalizes blood phosphate thereby healing rickets, improves bowing of the legs in children, reduces pain and stiffness, promotes bone healing and fracture resolution, and improves osteomalacia. “I am delighted to have a new therapy for this disease,” said Dr. Econs, “I have been caring for individuals with XLH for my entire career and I have seen how debilitating it can be. To see our research lead to a new drug for XLH is a dream come true. This therapy will help alleviate suffering and enable patients to live fuller, more active lives.”

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