FDA awards more than a dozen grants for rare disease research

SILVER SPRING, Md. — The Food and Drug Administration is offering more than $14 million in grants for the development of products for patients with rare diseases, the agency said Monday.

The FDA announced that it had granted 15 grants for orphan drug research. As defined by the agency for drugs, orphan diseases affect fewer than 200,000 people in the United States. For devices, a rare disease occurs so infrequently in the country that there is no reasonable explanation that a medical device for such a disease will be developed without assistance. According to the National Institutes of Health, there are about 6,800 rare diseases and conditions, affecting nearly 30 million Americans.

Among the grants, Alkeus Pharmaceuticals received $167,000 for a drug for Stargardt disease currently in phase-1 development; Children's Hospital Medical Center in Cincinnati received $600,000 for a phase-1 study of a drug to treat Fanconi anemia; Children's Hospital received $600,000 for a phase-1 study of a drug for Wiskott-Aldrich syndrome; and Vanderbilt University received $1.59 million for a phase-2 study of montelukast, which Merck markets as the asthma and allergy drug Singulair, for the treatment of sickle-cell anemia.

"The FDA is committed to fostering and encoring the development of products for rare diseases, most of which have no available or adequate treatments," FDA Office of Orphan Product Development director Gayatri Rao said. "The grants awarded this year support studies in very vulnerable, difficult-to-treat populations who have no available options."