FDA Approves First Drug for Rare Form of Rickets

Share this content:
FDA Approves First Drug for Rare Form of Rickets
FDA Approves First Drug for Rare Form of Rickets

FRIDAY, April 20, 2018 (HealthDay News) -- Ultragenyx Pharmaceutical Inc.'s Crysvita (burosumab-twza) has been approved by the U.S. Food and Drug Administration to treat adults and children ages 1 year and older with x-linked hypophosphatemia (XLH).

A clinical trial found that 94 percent of adults who took Crysvita once a month achieved normal phosphorus levels, compared with 8 percent of those who took a placebo, according to the FDA. In children, 94 to 100 percent of those treated with Crysvita every two weeks achieved normal phosphorus levels.

The most common negative side effects of Crysvita in adults were back pain, headache, restless leg syndrome, decreased vitamin D, dizziness, and constipation. The most common negative side effects in children were headache, injection site reaction, vomiting, decreased vitamin D, and fever, the FDA said.

"XLH differs from other forms of rickets in that vitamin D therapy is not effective," Julie Beitz, M.D., director of the Office of Drug Evaluation III in the FDA's Center for Drug Evaluation and Research, said in an agency news release. "This is the first FDA-approved medication for the treatment of XLH and a real breakthrough for those living with this serious disease."

More Information

Share this content:

is free, fast, and customized just for you!




Already a member?

Sign In Now »

Trending Activities

All Professions

Drug Lookup

Browse drugs by: BrandGenericDisease

More in Home

FDA Permits Marketing of Brain Stimulation Device for OCD

FDA Permits Marketing of Brain Stimulation Device for ...

FDA previously approved transcranial magnetic stimulation for major depression, certain migraines

Comments Open on End of NIH Review for Gene Therapy Studies

Comments Open on End of NIH Review for ...

NIH oversight panel no longer plans to review all applications for gene therapy experiments

U.S. Measles Outbreak Hits 107 Cases in 21 States, D.C.

U.S. Measles Outbreak Hits 107 Cases in 21 ...

Outbreak on track to exceed last year's; most of the people who got measles weren't vaccinated

is free, fast, and customized just for you!




Already a member?

Sign In Now »