Rare Diseases

FDA Awards $10M in Grants for Rare Disease Natural History Studies

  • The FDA said today it has awarded six research grants totaling more than $10 million for natural history studies in rare diseases.

    The $10.3 million includes $6.3 million over five years from the FDA’s Orphan Products Grants Program—the first time the FDA is providing funding through the program—and $3.5 million received through a partnership with the NIH’s National Center for Advancing Translational Sciences (NCATS).

    The $3.5 million accounted for most of the $4 million awarded to two of the six grant awardees, with the rest coming from the FDA:

    • Children’s Hospital Corporation, Wen-Hann Tan, MBBS, prospective study in Angelman syndrome, approximately $2 million over 5 years;
    • University of Utah, Nicholas Johnson, M.D., prospective study in myotonic dystrophy type 1 to determine biomarkers and clinical endpoints, approximately $2 million over 5 years.

    The remaining grant awardees, who received the combined $6.3 million from the FDA Orphan Products Grants Program, included:

    • Children’s Hospital of Philadelphia, David Lynch, M.D., Ph.D., prospective study in Friedreich’s ataxia, approximately $2 million over 5 years;
    • Columbia University Medical Center, Adi Cohen, M.D., prospective study in pregnancy and lactation-associated osteoporosis, approximately $2 million over 5 years;
    • University of Iowa, Alicia Gerke, M.D., MBA, retrospective study in sarcoidosis, approximately $300,000 over 2 years;
    • University of North Carolina at Chapel Hill, Kenneth Ataga, MBBS, prospective study in sickle cell…