FDA News

FDA awards $18 million to fund trials of medical products for rare diseases

Scott Gottlieb
Scott Gottlieb

The FDA awarded more than $18 million in grants over the next 4 years to fund clinical trials of medical products intended to treat patients with rare diseases.

“Developing a treatment for a rare disease can be especially challenging. 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,” FDA Commissioner Scott Gottlieb, MD, said in a press release. “The FDA is committed to doing its part to facilitate continued progress toward more treatments, and even potential cures, for patients with rare diseases.”

The FDA issued the grants through the Orphan Products Clinical Trials Grants Program. The program has provided more than $400 million to fund more than 600 clinical studies since 1983.

The grants are intended to fund studies that will evaluate the effectiveness and safety or products that could lead to or contribute to FDA approval of products intended to treat rare diseases.

“This funding helps support early-stage development activities targeting rare diseases that don’t have effective treatments,” Gottlieb said. “By providing seed capital, these FDA-administered grants enable researchers to prove out important concepts. The FDA grants also provide some important recognition to promising development programs that ultimately can help researchers attract additional funding.”

More than 100 rare disease experts reviewed grant applications for scientific and technical merit.

Grant recipients, as well as principal investigators and approximate funding amounts, are as follows:

  • Alkeus Pharmaceuticals Inc. will receive $1.75 million over 4 years. Leonide Saad, PhD, will serve as principal investigator of a phase 2 study of ALK-001 (Alkeus Pharmaceuticals) for treatment of Stargardt disease. The condition causes degeneration of the macula, a small area in the center of the retina.
  • Arizona State University will receive $2 million over 4 years. Keith D. Lindor, MD, will serve as principal investigator of a phase 2 study of oral vancomycin for treatment of primary sclerosing cholangitis, a progressive disease of the liver and gallbladder.
  • Cedars-Sinai Medical Center will receive $2 million over 4 years. Shlomo Melmed, MB, ChB, will serve as principal investigator of a phase 2 study of seliciclib (Cyclacel Pharmaceuticals) for treatment of Cushing disease, a benign tumor of the pituitary gland.
  • Columbia University will receive $750,000 over 3 years. Yvonne Saenger, MD, will serve as principal investigator of a phase 1 study of talimogene laherparepvec (Imlygic, Amgen) for treatment of advanced pancreatic cancer.
  • Emory University will receive $1.5 million over 3 years. Eric J. Sorscher, MD, will serve as principal investigator of a phase 1/phase 2 study of Ad/PNP fludarabine for the treatment of head and neck squamous cell carcinoma.
  • Fibrocell Technologies Inc. will receive $1.5 million over 4 years. Josh Maslowski will serve as principal investigator of a phase 1/phase 2 study of gene-modified ex-vivo autologous fibroblasts for treatment of dystrophic epidermolysis bullosa, an inherited disease that affects the skin or other organs.
  • Johns Hopkins University will receive $750,000 over 3 years. Amy Dezern, MD, MHS, will serve as principal investigator of a phase 1/phase 2 study of CD8-reduced T cells for treatment of acute myeloid leukemia or myelodysplastic syndrome.
  • OncoImmune Inc. will receive $2 million over 4 years. Yang Liu, PhD, will serve as principal investigator of a phase 2b study of CD24Fc for prevention of graft-versus-host disease.
  • Patagonia Pharmaceuticals LLC will receive $1.5 million over 3 years. Zachary Rome will serve as principal investigator of a phase 2 study of isotretinoin (PAT-001, Patagonia Pharmaceuticals) for treatment of congenital ichthyosis, a family of rare genetic skin disorders.
  • The General Hospital Corporation will receive $1.4 million over 4 years. Stephanie Seminara, MD, will serve as principal investigator of a phase 2 study of kisspeptin for the treatment of dopamine agonist intolerant hyperprolactinemia, a condition characterized by higher-than-normal levels of prolactin in the blood.
  • The University of North Carolina at Chapel Hill will receive $2 million over 4 years. Matthew Laughon, MD, MPH, will serve as principal investigator of a phase 2 study of sildenafil for the prevention of bronchopulmonary dysplasia, a form of chronic lung disease that affects newborns and infants.
  • University of Minnesota will receive $1.4 million over 3 years. Kyriakie Sarafoglou, MD, will serve as principal investigator of a phase 2a study of subcutaneous hydrocortisone infusion pump for the treatment of congenital adrenal hyperplasia, a group of genetic conditions that limit hormone production in the adrenal glands.

Scott Gottlieb
Scott Gottlieb

The FDA awarded more than $18 million in grants over the next 4 years to fund clinical trials of medical products intended to treat patients with rare diseases.

“Developing a treatment for a rare disease can be especially challenging. 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,” FDA Commissioner Scott Gottlieb, MD, said in a press release. “The FDA is committed to doing its part to facilitate continued progress toward more treatments, and even potential cures, for patients with rare diseases.”

The FDA issued the grants through the Orphan Products Clinical Trials Grants Program. The program has provided more than $400 million to fund more than 600 clinical studies since 1983.

The grants are intended to fund studies that will evaluate the effectiveness and safety or products that could lead to or contribute to FDA approval of products intended to treat rare diseases.

“This funding helps support early-stage development activities targeting rare diseases that don’t have effective treatments,” Gottlieb said. “By providing seed capital, these FDA-administered grants enable researchers to prove out important concepts. The FDA grants also provide some important recognition to promising development programs that ultimately can help researchers attract additional funding.”

More than 100 rare disease experts reviewed grant applications for scientific and technical merit.

Grant recipients, as well as principal investigators and approximate funding amounts, are as follows:

  • Alkeus Pharmaceuticals Inc. will receive $1.75 million over 4 years. Leonide Saad, PhD, will serve as principal investigator of a phase 2 study of ALK-001 (Alkeus Pharmaceuticals) for treatment of Stargardt disease. The condition causes degeneration of the macula, a small area in the center of the retina.
  • Arizona State University will receive $2 million over 4 years. Keith D. Lindor, MD, will serve as principal investigator of a phase 2 study of oral vancomycin for treatment of primary sclerosing cholangitis, a progressive disease of the liver and gallbladder.
  • Cedars-Sinai Medical Center will receive $2 million over 4 years. Shlomo Melmed, MB, ChB, will serve as principal investigator of a phase 2 study of seliciclib (Cyclacel Pharmaceuticals) for treatment of Cushing disease, a benign tumor of the pituitary gland.
  • Columbia University will receive $750,000 over 3 years. Yvonne Saenger, MD, will serve as principal investigator of a phase 1 study of talimogene laherparepvec (Imlygic, Amgen) for treatment of advanced pancreatic cancer.
  • Emory University will receive $1.5 million over 3 years. Eric J. Sorscher, MD, will serve as principal investigator of a phase 1/phase 2 study of Ad/PNP fludarabine for the treatment of head and neck squamous cell carcinoma.
  • Fibrocell Technologies Inc. will receive $1.5 million over 4 years. Josh Maslowski will serve as principal investigator of a phase 1/phase 2 study of gene-modified ex-vivo autologous fibroblasts for treatment of dystrophic epidermolysis bullosa, an inherited disease that affects the skin or other organs.
  • Johns Hopkins University will receive $750,000 over 3 years. Amy Dezern, MD, MHS, will serve as principal investigator of a phase 1/phase 2 study of CD8-reduced T cells for treatment of acute myeloid leukemia or myelodysplastic syndrome.
  • OncoImmune Inc. will receive $2 million over 4 years. Yang Liu, PhD, will serve as principal investigator of a phase 2b study of CD24Fc for prevention of graft-versus-host disease.
  • Patagonia Pharmaceuticals LLC will receive $1.5 million over 3 years. Zachary Rome will serve as principal investigator of a phase 2 study of isotretinoin (PAT-001, Patagonia Pharmaceuticals) for treatment of congenital ichthyosis, a family of rare genetic skin disorders.
  • The General Hospital Corporation will receive $1.4 million over 4 years. Stephanie Seminara, MD, will serve as principal investigator of a phase 2 study of kisspeptin for the treatment of dopamine agonist intolerant hyperprolactinemia, a condition characterized by higher-than-normal levels of prolactin in the blood.
  • The University of North Carolina at Chapel Hill will receive $2 million over 4 years. Matthew Laughon, MD, MPH, will serve as principal investigator of a phase 2 study of sildenafil for the prevention of bronchopulmonary dysplasia, a form of chronic lung disease that affects newborns and infants.
  • University of Minnesota will receive $1.4 million over 3 years. Kyriakie Sarafoglou, MD, will serve as principal investigator of a phase 2a study of subcutaneous hydrocortisone infusion pump for the treatment of congenital adrenal hyperplasia, a group of genetic conditions that limit hormone production in the adrenal glands.