Three Decades of Research Culminates in FDA Drug Approval

By IU School of Medicine and CTSI Staff Reports:
Indiana CTSI-supported research led by two Indiana University School of Medicine faculty members has culminated in U.S. Food and Drug Administration approval of a new drug for patients with an uncommon, painful and deforming bone disease.

The drug, burosumab, treats X-linked hypophosphatemia, or XLH. It is being brought to market by Ultragenyx Pharmaceutical Inc. in collaboration with Kyowa Hakko Kirin Co., Ltd. and its European subsidiary, Kyowa Kirin International PLC, under the brand name Crysvita.
XLH is a phosphate-wasting disease that causes rickets and osteomalacia, or softening of the bones. It is typically inherited and affects more than 12,000 patients in the United States and one out of every 20,000 people worldwide. Patients are typically diagnosed as children and have bowed legs, short stature, and experience bone pain and dental abscesses.

The first ever human dose of burosumab was administered by an Indiana CTSI Clinical Research Center (CRC) nurse on January 27, 2009. CRC staff conducted study procedures for all clinic study visits associated with the Phase I – III studies (eight studies total), which included 46 patients (aged 1 year to 70 years) and 822 total study visits, and the CRC continues to support this important work with an upcoming Phase IV study.

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