Rare Daily Staff

The U.S. Food and Drug Administration awarded 12 new clinical trial research grants totaling more than $15 million over the next four years to enhance the development of medical products for patients with rare diseases.

The FDA awarded the grants through the Orphan Products Clinical Trials Grants Program to encourage clinical development of drugs, biologics, medical devices, and medical foods for the treatment of rare diseases. The grants are intended to contribute to marketing approval of products to treat rare diseases or provide essential data needed for development of such products. The FDA received 89 clinical trial grant applications that were reviewed and evaluated for scientific and technical merit by more than 100 rare disease experts, including members of academia.

Three-quarters of the new awards fund studies enrolling children, including children as young as one month. These studies target a variety of rare diseases affecting children and have the potential to advance treatments for these diseases. Two-thirds of the new awards fund clinical studies of products for use in various rare cancers.

The recipients, principal investigators, and approximate funding amounts are:

•             Chemocentryx, Inc. (Mountain View, California), Peter Staehr, phase 2 study of avacopan for the treatment of complement 3 glomerulopathy – $1 million over two years

•             Cincinnati Children’s Hospital Medical Center (Cincinnati, Ohio), Maryam Fouladi, phase 1 study of PTC596 for the treatment of diffuse intrinsic pontine glioma and high-grade gliomas – $750,000 over three years

•             Cincinnati Children’s Hospital Medical Center (Cincinnati, Ohio), Parinda Mehta, phase 2 study of quercetin chemoprevention for the treatment of squamous cell carcinoma in patients with Fanconi Anemia – $1.7 million over four years

•             Columbia University Health Sciences (New York, New York), Gary Brittenham, phase 2 study of daily vitamin D for the treatment of sickle-cell respiratory complications – $2 million over four years

•             Cumberland Pharmaceuticals, Inc. (Nashville, Tennessee), Ines Macias-Perez, phase 2 study of oral ifetroban for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy – $1 million over three years

•             Massachusetts General Hospital (Boston, Massachusetts), Sara Pai, phase 2 study of anti-PD1 therapy for the treatment of HPV-associated recurrent respiratory papillomatosis – $1 million over three years

•             New York Medical College (Valhalla, New York), Mitchell Cairo, phase 2 study of viral specific cytotoxic T-lymphocytes for the treatment of refractory viral infections and T-cell immunodeficiency – $1.7 million over four years

•             Privo Technologies, LLC. (Peabody, Massachusetts), Manijeh Goldberg, phase 1/2 study of cisplatin patch (PRV111) for the treatment of oral cancer – $2 million over four years

•             Targeted Therapy Technologies, LLC (Somerset, New Jersey), Ricardo Carvalho, phase 1 study of episcleral topotecan for the treatment of retinoblastoma – $660,000 over three years

•             University of Alabama at Birmingham (Birmingham, Alabama), Gregory Friedman, phase 1 study of oncolytic engineered herpes simplex virus therapy for the treatment of pediatric malignant cerebellar brain tumors – $750,000 over three years

•             University of California San Diego (La Jolla, California), Jason Sicklick, phase 2 study of temozolomide for the treatment of gastrointestinal stromal tumor – $1.5 million over three years

•             University of Texas MD Anderson Cancer Center (Houston, Texas), Michael Andreeff, phase 1/2 study of the imipridone (ONC201) for treatment of acute myeloid leukemia – $1 million over four years

“The majority of rare diseases still do not have approved therapies and the FDA is committed to fostering product development in areas of unmet need,” said Janet Maynard, director of the FDA’s Office of Orphan Products Development. “The Orphan Products Grants Program is one of several ways that the FDA supports the development of products for rare diseases.”

Since its creation in 1983, the program has provided more than $400 million to fund more than 600 new clinical studies. By helping to spark research, the agency said it hopes to speed the development of products for rare diseases, and ultimately, make needed treatments available to those patients who need them most.

Photo: Janet Maynard, director of the FDA’s Office of Orphan Products Development

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