FDA awards $4.8 million for study of three rare endocrine conditions

The FDA awarded 12 clinical trial research grants to enhance the development of medical products for patients with rare diseases, including treatments for Cushing’s disease, hyperprolactinemia and congenital adrenal hyperplasia, according to a press release from the agency.

Awards issued include a grant to Cedars-Sinai Medical Center, Los Angeles, for a phase 2 study of seliciclib for the treatment of Cushing disease ($2 million over 4 years), a grant to the University of Minnesota in Minneapolis for a phase 2a study of a subcutaneous hydrocortisone infusion pump for the treatment of congenital adrenal hyperplasia ($1.4 million over 3 years) and an award to the General Hospital Corporation in Boston for a phase 2 study of kisspeptin for the treatment of dopamine agonist intolerant hyperprolactinemia ($1.4 million over 4 years).

“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,” Scott Gottlieb, MD, commissioner of the FDA, said in a statement. “The FDA is committed to doing its part to facilitate continued progress toward more treatments, and even potential cures, for patients with rare diseases. New scientific advances offer more opportunities to develop these potential cures. With efficient regulation, proper incentives for product development and the continued support of patients, providers and researchers, we have more opportunities to pursue these advances than ever before.”

The FDA awarded the grants through the Orphan Products Clinical Trials Grants Program. This program is funded by Congressional appropriations and encourages 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 the treatment of 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 NIH and the FDA.

Other grant recipients include the following:

Alkeus Pharmaceuticals, Inc., Cambridge, Massachusetts, for a phase 2 study of ALK-001 for the treatment of Stargardt disease ($1.75 million over 4 years)
Arizona State University-Tempe Campus, Tempe, Arizona, for a phase 2 study of oral vancomycin for the treatment of primary sclerosing cholangitis ($2 million over 4 years)
Columbia University of New York for a phase 1 study of talimogene laherparepvec for the treatment for advanced pancreatic cancer ($750,000 over 3 years)
Emory University, Atlanta, Georgia, for a phase 1/ 2 study of Ad/PNP fludarabine for the treatment of head and neck squamous cell carcinoma ($1.5 million over 3 years)
Fibrocell Technologies, Inc., Exton, Pennsylvania, for a phase 1/2 study of gene-modified ex-vivo autologous fibroblasts for the treatment of dystrophic epidermolysis bullosa ($1.5 million over 4 years)
Johns Hopkins University, Baltimore, Maryland, for a phase 1/2 study of CD8-reduced T cells for the treatment of myelodysplastic syndrome or acute myeloid leukemia ($750,000 over 3 years)
Oncolmmune, Inc., Rockville, Maryland, for a phase 2b study of CD24Fc for the prevention of graft versus host disease ($2 million over 4 years)
Patagonia Pharmaceuticals, LLC, Woodcliff Lake, New Jersey, for a phase 2 study of PAT-001 (isotretinoin) for the treatment of congenital ichthyosis ($1.5 million over 3 years)
University of North Carolina at Chapel Hill, North Carolina, for a phase 2 study of sildenafil for the prevention of bronchopulmonary dysplasia ($2 million over 4 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,” Debra Lewis, OD, acting director of the FDA’s Office of Orphan Products Development, said in a statement. “We are encouraged to see so much interest in our grants program and are pleased to support research for a variety of rare diseases that have little, or no, treatment options for patients.”

The program’s grants have supported research that led to the marketing approval of more than 60 orphan products. The FDA is also supporting six natural history studies for rare diseases to further advance the mission of bringing new therapies to market. – by Regina Schaffer