FDA Awards 12 Grants to Fund Clinical Trials to Advance Rare Disease Treatments

Rare Daily Staff

The U.S. Food and Drug Administration said that it has awarded 12 new clinical trial research grants totaling more than $18 million over the next four years to enhance the development of medical products for patients with rare diseases.

“Developing a treatment for a rare disease can be especially challenging,” said FDA Commissioner Scott Gottlieb. “Given the often small number of patients affected by certain very rare diseases, there can be limited markets for new treatments, and as a result fewer resources devoted to researching these opportunities.”

Gottlieb said the funding will help support early-stage development activities targeting rare diseases that don’t have effective treatments. The grants are intended to allow researchers to prove out important concepts and provide recognition to promising development programs that can help researchers attract additional funding.

The FDA awarded the grants through its Orphan Products Clinical Trials Grants Program. 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 the treatment of rare diseases. The program covers the clinical development of drugs, biologics, medical devices, and medical foods for use in rare diseases.

Grant applications were reviewed and evaluated for scientific and technical merit by more than 100 rare disease experts, which included representatives from academia, the National Institutes of Health, and the FDA.

The grant recipients include:

• Alkeus Pharmaceuticals, Inc. (Cambridge, Massachusetts), Leonide Saad, phase 2 study of ALK-001 for the treatment of Stargardt disease – $1.75 million over four years
• Arizona State University-Tempe Campus (Tempe, Arizona), Keith Lindor, phase 2 study of oral vancomycin for the treatment of primary sclerosing cholangitis – $2 million over four years
• Cedars-Sinai Medical Center (Los Angeles), Shlomo Melmed, phase 2 study of seliciclib for the treatment of Cushing disease – $2 million over four years
• Columbia University of New York (New York), Yvonne Saenger, phase 1 study of talimogene laherparepvec for the treatment for advanced pancreatic cancer –  $750,000 over three years
• Emory University (Atlanta), Eric Sorscher, phase 1/ 2 study of Ad/PNP fludarabine for the treatment of head and neck squamous cell carcinoma – $1.5 million over three years
• Fibrocell Technologies, Inc. (Exton, Pennsylvania), John Maslowski, phase 1/2 study of gene-modified ex-vivo autologous fibroblasts for the treatment of dystrophic epidermolysis bullosa – $1.5 million over four years
• Johns Hopkins University (Baltimore), Amy Dezern, phase 1/2 study of CD8-reduced T cells for the treatment of myelodysplastic syndrome or acute myeloid leukemia – $750,000 over three years
• Oncolmmune, Inc. (Rockville, Maryland) Yang Liu, phase 2b study of CD24Fc for the prevention of graft versus host disease – $2 million over four years
• Patagonia Pharmaceuticals, LLC (Woodcliff Lake, New Jersey), Zachary Rome, phase 2 study of PAT-001 (isotretinoin) for the treatment of congenital ichthyosis – $1.5 million over three years
• The General Hospital Corporation (Boston), Stephanie Seminara, phase 2 study of kisspeptin for the treatment of dopamine agonist intolerant hyperprolactinemia – $1.4 million over four years
• University of Minnesota (Minneapolis), Kyriakie Sarafoglou, phase 2a study of subcutaneous hydrocortisone infusion pump for the treatment of congenital adrenal hyperplasia – $1.4 million over three years
• University of North Carolina at Chapel Hill (Chapel Hill, North Carolina), Matthew Laughon, phase 2 study of sildenafil for the prevention of bronchopulmonary dysplasia – $2 million over four years

Since its creation in 1983, the Orphan Products Grants Program has provided more than $400 million to fund more than 600 new clinical studies. To date, the program’s grants have supported research that led to the marketing approval of more than 60 orphan products.

September 24, 2018
Photo: Scott Gottlieb, commissioner of the FDA