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FDA Approves Record Number of Rare Disease Drugs

FDA Approves Record Number of Rare Disease Drugs
(Copyright DPC)

By    |   Tuesday, 05 January 2016 02:36 PM

The U.S. Food and Drug Administration set a record last year for approvals of new drugs to treat rare diseases, an advocacy group said.

The National Organization for Rare Disorders (NORD), an independent nonprofit organization representing patients and families affected by rare diseases, reports that the FDA approved 21 new “orphan” drugs to treat rare diseases in 2015 — 47 percent of all novel new drugs approved for the year. 

This is the second consecutive year in which the FDA approved more orphan drugs for rare diseases than any previous year in FDA history, according to Dr. John K. Jenkins, director of the FDA’s Office of New Drugs, Center for Drug Evaluation and Research.

“The remarkable number of orphan drugs approved by FDA in 2015 underscores the progress we are making in identifying and studying new therapies, the willingness of the investment community to invest in orphan drugs, and the continued flexibility shown by the FDA in reviewing applications that often include small studies due to the small number of patients with the disease,” said Peter L. Saltonstall, president and CEO of NORD.

 “At the same time, we must recognize that our patient community faces new challenges in accessing many new therapies, and that the vast majority of the 7,000 identified rare diseases still have no treatment options.”

Among the newly approved drugs:
  • Kanuma – To treat lysosomal acid lipase deficiency, a rare inherited genetic disorder that does not allow the body to produce an enzyme responsible for breaking down fats, and can lead to liver disease, cardiovascular disease, and life-threatening organ damage.
  • Orkambi – A therapy for the lung disease, cystic fibrosis.
  • Strensiq - Long-term enzyme replacement therapy in patients with infantile- and juvenile - onset hypophosphatasia, a serious and sometimes fatal bone disease.
  • Unituxin - To treat pediatric patients with high-risk neuroblastoma (brain tumors).
  • Xuriden – To treat patients with hereditary orotic aciduria, a condition that can result in blood abnormalities (anemia, decreased white blood cell count, decreased neutrophil count), urinary tract obstruction, failure to thrive, and developmental delays.

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The FDA set a record last year for approvals of new drugs to treat rare diseases, an advocacy group said.
fda, rare, disease, drug, approvals
Tuesday, 05 January 2016 02:36 PM
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