Rare Disease Report

FDA Grants More than $30 Million to Rare Disease Community

OCTOBER 06, 2017
Mathew Shanley
This morning, the U.S. Food and Drug Administration announced the awarding of grants to fund 15 clinical trials and 6 natural history studies, all pertaining to the rare disease community.

Over the next 4 years the FDA will provide more than $22 million toward the clinical trials and over the next 5 years, another $6.3 million will fund natural history studies in rare diseases.

Recipients of the clinical trial research grants include principal investigators from academia and industry across the rare disease landscape. The grants were awarded through the Orphan Products Clinical Trials Grant Program and funded by Congressional appropriations. They are intended for clinical studies, to evaluate the safety and efficacy of products that could result in or contribute to future FDA approvals.

Since 1983, the program has provided more than $390 million to fund more than 600 clinical studies.

This year’s grant recipients include:
  • Sickle Cell Disease: Caterina Minniti, Albert Einstein College of Medicine (Bronx, New York) – Phase 2 Study of Topical Sodium Nitrite for the Treatment of patients with Sickle Cell Disease & Leg Ulcers ($2 million/4 years)
  • Prader-Willi Syndrome: Eric Hollander, Albert Einstein College of Medicine (Bronx, New York) – Phase 2 Study of Oxytocin for the Treatment of Hyperphagia in Prader-Willi Syndrome ($1.5 million /3 years)
  • Neuroblastoma: Steven Dubois, Dana-Farber Cancer Institute (Boston) – Phase 1 Study of dual PI3K/BRD4 Inhibitor SF1126 for the Treatment of Neuroblastoma ($750,000/3 years)
  • Allan Kirk, Duke University (Durham, North Carolina) – Phase 2 Study of Belatacept, Alemtuzumab, and Sirolimus in Renal Transplantation ($1 million/3 years)
  • Immune Thrombocytopenia: Richard Francovitch, Protalex, Inc (Florham Park, New Jersey) – Phase 1/2 Study of PRTX-100 for the Treatment of Immune Thrombocytopenia ($500,000/2 years)
  • And 10 others. For the full list, see the FDA press release.
“Given the often-small number of patients facing certain rare diseases, there can be limited resources devoted to researching new drugs and unique challenges with recruiting and conducting the clinical trials needed to develop medicines targeted to rare conditions,” said FDA Commissioner Scott Gottlieb, M.D.

“By helping to support the cost of development of these potential new drugs, and reduce some of the financial risk, we also hope that these grants will lower the cost of the capital needed to develop these products, boost competition and translate into lower prices for successful medicines. This can help increase access to resulting therapies.”

While the FDA has been investing in trials for more than 30 years, this is the first time that the administration is providing funding toward rare disease natural history studies. It is widely-assumed that these models can reduce the current necessity for placebo arms in studies of drugs for ultra-rare diseases where recruitment presents a challenge.

More than 80 grant applications were reviewed and evaluated for scientific and technical merit, and 2 will be partially funded by the National Institute of Health (NIH)/National Center for Advancing Translational Scientists (NCATS) the list of the award recipients include:
  • Friedreich’s Ataxia: David Lynch, Children's Hospital of Philadelphia – Prospective study in Friedreich’s ataxia ($2 million/5 years)
  • Pregnancy and Lactation-Associated Osteoporosis: Adi Cohen, Columbia University Medical Center – Prospective study in pregnancy and lactation-associated osteoporosis ($2 million/5 years)
  • Sarcoidosis: Alicia Gerke, University of Iowa, Alicia Gerke – Retrospective study in sarcoidosis, ($300,000/2 years)
  • Chronic Kidney Disease: Kenneth Ataga, University of North Carolina at Chapel Hill – Prospective study in sickle cell anemia to determine biomarkers of endothelial function changes in chronic kidney disease ($2 million/5 years)
  • Angelman Syndrome: Wen-Hann Tan, Children’s Hospital Corporation – Prospective study in Angelman syndrome ($2 million/5 years) (Partially funded by NIH/NCATS)
  • Myotonic Dystrophy Type 1: Nicholas Johnson, University of Utah – Prospective study in Myotonic Dystrophy Type 1 to Determine Biomarkers and Clinical Endpoints, ($2 million/5 years) (Partially funded by NIH/NCATS)
“We believe these important studies will provide key information about how these rare diseases develop and progress and can ultimately help in the development of models of disease behavior that can make the development process more efficient,” said FDA Commissioner Scott Gottlieb, M.D. in an official press release.

“We’ve been working overtime to develop models that can simulate the behavior of placebo arms in the setting of very rare diseases, where recruiting for clinical trials can be especially hard. We’re grateful to NCATS for its support of this important program, which enabled us to extend our support to two additional studies.”

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