Drug Clinical Trial Reports Back Positive Results for Rare Bone Disease

Great news for the rare disease community!
Ultragenyx Pharmaceuticals reported back positive results from its phase 3 study of the treatment drug burosumab in adults with X-linked hypophosphatemia (XLH).

XLH is an inherited disorder characterized by low levels of phosphate in the blood, causing a loss of phosphate in the urine (phosphate wasting) and leading to soft, weak bones (rickets). To learn more about it, click here.

“This longer term data on symptom improvement and fracture healing support burosumab’s potential value in treating serious disease symptoms and promoting bone healing in adult patients with XLH,” said Ultragenyx CEO Emil Kakkis, M.D., in a statement. “The continued clinical improvements in patients and the new data demonstrating a significant decrease in pain medication use after treatment with burosumab provide further support for the potential value in the treatment of adults with XLH.” 
In Ultragenyx’s trial – which enrolled 134 patients – treatment with burosumab for 48 weeks resulted in “sustained maintenance of normal serum phosphorus levels and further improvement in stiffness, physical function and pain,” Ultragenyx said.
In addition, patients who switched from the placebo to the therapy after 24 weeks “showed normalization of serum phosphorus and improvement in stiffness, pain and physical functioning,” according to the company.
These results are promising in an effort to treat XLH – and is paving the way for FDA approval!
The FDA approval date is set for April 17th, 2018.
Read the full press release here!

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