FDA Awards Grants for Clinical Trials to Stimulate Product Development for Rare Diseases


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THE U.S. FOOD and Drug Administration (FDA) has awarded numerous new clinical trial research grants, totaling more than $22 million over the next 4 years, to boost the development of products for patients with rare diseases. These new grants were awarded to principal investigators from academia and industry across the country. 

“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, MD. “For more than 30 years, the FDA has been committed to investing in trials of potentially life-changing treatments for patients with rare diseases, especially in situations where commercial incentives may not be enough to foster the collection of quality data that can ultimately support efficient development and FDA approval of treatments for patients who lack effective alternatives. By helping to support the cost of development of these potential new drugs, and reduce some of the financial risk, we also hope 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.” 

The FDA awarded the grants through the Orphan Products Clinical Trials Grants Program, funded by Congressional appropriations, to encourage clinical development of drugs, biologics, medical devices, or medical foods for use in rare diseases. The grants are intended for clinical studies evaluating the safety and effectiveness of products that could either result in, or substantially contribute to, the FDA approval of products targeted to rare diseases. 

Oncology-Specific Grants 

APPROXIMATELY 33% of the new grant awards fund studies to accelerate cancer research by enrolling patients with rare forms of cancer. Sixty percent of these studies target devastating forms of brain and peripheral nervous system cancers, including glioblastoma and anaplastic astrocytoma. One study is recruiting children as young as a year old with a particularly aggressive form of neuroblastoma. 

The oncology-specific grant recipients for fiscal year 2017 follow: 

  • AADi, LLC: Phase II Study of ABI-009 for the Treatment of Advanced Perivascular Epithelioid Cell Tumors 
  • Columbia University Medical Center: Phase II Study of PLX3397 and Sirolimus for the Treatment of Malignant Peripheral Nerve Sheath Tumors 
  • Dana-Farber Cancer Institute: Phase I Study of Dual PI3K/BRD4 Inhibitor SF1126 for the Treatment of Neuroblastoma 
  • Protalex, Inc: Phase I/II Study of PRTX-100 for the Treatment of Immune Thrombocytopenia 
  • Sloan Kettering Institute for Cancer Research: Phase 2 Study of MEK162 and Imatinib for the Treatment of Gastrointestinal Stromal Tumors 
  • Tocagen Inc: Phase II/III Study of Toca 511 and Toca FC vs Standard of Care in Recurrent Glioblastoma and Anaplastic Astrocytoma. ■


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