There’s a new treatment for X-Linked Hypophosphatemia (XLH) that may be available in the not-so-distant future.
XLH occurs when the kidneys process phosphate abnormally, leading to excess phosphate loss in urine. This is known as phosphate wasting. This process reduces phosphate levels in the blood, which leads to soft bones, short stature, and altered skull growth. To read more about this rare genetic condition, click here.
As of now, treatment includes supplements, growth hormones, and corrective surgery. However, while patients can take greater amounts of dietary phosphate, there are currently no available treatments that work directly on the underlying problem of phosphate wasting.
This may change when burosamab is released. Burosamab is a drug sponsored by Ultragenyx Pharmaceutical Inc and Kyowa Hakko Kirin International PLC. Ultragenyx Pharmaceutical Inc is a relatively new biopharmaceutical company, focused on finding treatments for rare and ultra conditions. Kyowa Hakko Kirin International PLC is a life science company that focuses in biotechnologies.
The week before last, the U.S. Food and Drug Administration (FDA) approved the Biologics License Application. Because burosamab treats a rare pediatric disease, it qualifies for Priority Review. This means that it’ll be reviewed in six months rather than the usual ten. The Prescription Drug User Fee Act action will take place in late April, 2018.
Heres how it works: burosamab is an antibody that acts directly against the phosphaturic hormone fibroblast growth factor 23 (FGF23). FGF23 is the hormone responsible for lowering levels of vitamin D and phosphorus. By counteracting the hormone at the root of the problem, burosamab could provide a deeper solution. The drug would work both for adults and children.
Right now, the outlook seems pretty good. There’s impressive data supporting this drug. Karl Insogna, M.D., of the Yale School of Medicine showed data at a recent convention of the effects of burosamab in a phase 3 study. 94% of the group of patients who took the medicine had phosphorus levels above a 2.5 mg.dL following 24 weeks of treatment. This is a pretty dramatic difference when you compare it to 7.6% the placebo group who met the same standard. Twice as many patients who took burosamab showed healed fractures after treatment when compared to the patients who received a placebo.
Will there be a new, more effective treatment on the market for patients suffering from XLH soon? Only time and clinical trials will tell for sure, but as of right now, the signs are looking up.
