The U.S. Food and Drug Administration (FDA) announced it has awarded over $15 million in new clinical trial research grants to enhance the development of medical products for patients with rare diseases. The grants, part of the Orphan Products Clinical Trials Grants Program, encourages clinical development of drugs, biologics, medical devices and medical foods for the treatment of rare diseases.
A total of 89 grant applications were submitted and of those, the 12 awardees were:

Chemocentryx, Inc. (Mountain View, California); Peter Staehr, MD
– 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, MD
– phase 1 study of PTC596 for the treatment of diffuse intrinsic pontine glioma & high-grade gliomas
– $750,000 over three years

Cincinnati Children’s Hospital Medical Center (Cincinnati, Ohio); Parinda Mehta, MD
– 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, MD
– 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, PhD
– phase 2 study for 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, MD
– 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, PhD
– 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, MD, PhD
-vphase 1 study of episcleral topotecan for the treatment of retinoblastoma
-v$660,000 over three years

University of Alabama at Birmingham (Birmingham, Alabama); Gregory Friedman, MD
-vphase 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, MD
– 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, MD, PhD
– phase 1/2 study of the imipridone (ONC201) for treatment of acute myeloid leukemia
– $1 million over four years

In a press release, FDA Principal Deputy Commissioner Amy Abernethy, M.D., Ph.D. said, “For more than 35 years, the FDA has been providing much-needed financial support for clinical trials of potentially life-changing treatments for patients with rare diseases. To date, the Orphan Products Clinical Trials Grants Program’s grants have supported research that led to the marketing approval of more than 60 treatments for rare diseases.”
Janet Maynard, M.D., director of the FDA’s Office of Orphan Products Development added, “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. 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.”

Dr. Maynard continued, “We are pleased to continue to support research for a variety of rare diseases that have little, or no, treatment options for patients. By helping to spark research, we hope to speed the development of products for rare diseases, and ultimately, make needed treatments available to those patients who need them most.”

The FDA also noted that 75%)of the awards will fund studies enrolling children and 67% will be for rare cancers.

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