The announcement of the new funding comes as approximately 30 million Americans are affected by 7,000 known rare diseases, but only a few hundred treatments exist for these rare diseases.
Two-thirds of the 2019 grants will go to studies affiliated with universities, two-thirds will also go for studies of rare cancers and three-quarters of the new awards fund studies enrolling children, including children as young as one month, according to FDA.
For instance, Cincinnati Children’s Hospital Medical Center won a three-year, $750,000 grant for a phase 1 study of a treatment for a type of pediatric brain cancer. Similarly, New Jersey-based Targeted Therapy Technologies won a $660,000 grant over three years for a phase 1 study of a treatment for retinoblastoma, which FDA said is a rare cancer in the eye most commonly affecting young children.
Researchers at the University of Alabama at Birmingham also won a $750,000 grant over three years for a phase 1 study of an oncolytic engineered herpes simplex virus therapy for the treatment of pediatric brain tumors.
The largest award — $2 million over four years — went to Columbia University Health Sciences for a phase 2 study of daily vitamin D for the treatment of sickle-cell respiratory complications.
“To date, the Orphan Products Clinical Trials Grants Program’s grants have supported research that led to the marketing approval of more than 60 treatments for rare diseases,”
said FDA Principal Deputy Commissioner Amy Abernethy.
Janet Maynard, director of FDA’s Office of Orphan Products Development, also noted that since the grant program was created in 1983, FDA has provided more than $400 million for more than 600 new clinical studies.