FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases- Drug Information Update

The U.S. Food and Drug Administration today announced 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. These new grants were awarded to principal investigators from academia and industry across the country. 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 The grant recipients, principal investigators and approximate funding amounts, listed alphabetically, are: 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 For more information, please visit: FDA awards 12 research grants for the development of medical products for rare diseases. The goal of the Orphan Products Clinical Trials Grants Program (formerly known as Orphan Products Grants) is to encourage clinical development of products for use in rare diseases or conditions. The products studied can be drugs, biologics, medical devices, or medical foods. For more detailed information on the OOPD Grant Program, please review the following: General Information: Information on the Orphan Products Clinical Trials  Grants Program Searchable Database of Orphan Product Funded Grants Instructions for Searchable Database of Funded Grants Frequently Asked Questions (FAQ) 2015 through 2018 Submission Dates: Request for Applications (RFA) for Orphan Grants: For Receipt Dates 2015 through 2018 (Abbreviated RFA) NIH Guide for Receipt Dates 2015 through 2018 (RFA-FD-15-001) (Detailed RFA)  2017-2018 Application Instructions and Helpful Hints Other Useful Links: Sponsor Investigator Roles and Responsibilities in Clinical Investigations for Drugs and Biologics ClinicalTrials.Gov FDA's Role: ClinicalTrials.gov Information Choose one the following Center links for information for Sponsor-Investigators submitting Investigational New Drug applications (INDs) or Investigational Device Exemptions (IDEs) CDER CBER CDRH Guidance: Guidance for Industry E6 Good Clinical Practice: Consolidated Guidance