FDA Grants to Stimulate Rare Disease Research

iStock_000016297240XSmallOn September 30, 2014, the U.S. Food and Drug Administration (FDA) announced the awarding of 15 grants totaling more than $19 million that will boost the development of treatments and products for patients with rare diseases.

Through its Orphan Products Grants Program, the FDA supports clinical studies on safety and/or effectiveness of products for rare diseases. The program was created in 1983 as part of the Orphan Drug Act and has, to date, funded more than 530 new clinical studies devoted to the development of treatments for rare diseases. Through the program, more than 50 products have been approved for marketing.

LGDA is pleased that among the 15 grantees for 2014 are Dr. Denise Adams, Cincinnati Children’s Hospital Medical Center (Cincinnati, Ohio), for the Phase 2 Study of Vincristine vs. Sirolimus for the Treatment of High Risk Kaposiform Hemangioendothelioma, and Dr. Alfred Lane, Stanford University (Stanford, Calif.), for a Phase 2 Study of Sildenafil for the Treatment of Lymphatic Malformations. Each study will receive approximately $1.6 million over four years.

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