FDA News

FDA awards $15 million to fund trials of treatments for rare diseases

Amy Abernethy
Amy P. Abernethy

The FDA awarded 12 grants worth more than $15 million to fund clinical trials of medical products intended to treat patients with rare diseases.

The grants were awarded through the Orphan Products Clinical Trials Grants Program, a Congress-funded initiative that promotes clinical development of drugs, biologics, medical devices and medical foods for the treatment of rare diseases.

The program — created in 1983 — has provided more than $400 million to fund 600 new clinical studies, and its grants have supported research that led to marketing approval of more than 60 treatments for rare diseases.

“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,” FDA Principal Deputy Commissioner Amy P. Abernethy, MD, PhD, said in a press release. “We are encouraged by the amount of interest we continue to have in the grants program and are committed to working with researchers and industry to facilitate and support the study and development of treatments for patients with rare diseases.”

This year, more than 100 rare disease experts evaluated 89 clinical trial grant applications for their scientific and technical merit.

The recipients are as follows:

•  Chemocentryx Inc. will receive $1 million over 2 years for a phase 2 study of avacopan (CCX168) for the treatment of complement 3 glomerulopathy.

Cincinnati Children’s Hospital Medical Center will receive $750,000 over 3 years for a phase 1 study of PTC596 (PTC Therapeutics) for the treatment of diffuse intrinsic pontine glioma and high-grade gliomas.

Cincinnati Children’s Hospital Medical Center also will receive $1.7 million over 4 years for a phase 2 study of quercetin chemoprevention for the treatment of squamous cell carcinoma among patients with Fanconi anemia.

Columbia University Health Sciences will receive $2 million over 4 years for a phase 2 study of daily vitamin D for the treatment of respiratory complications from sickle cell anemia.

Cumberland Pharmaceuticals Inc. will receive $1 million over 3 years for a phase 2 study of oral ifetroban for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy.

Massachusetts General Hospital will receive $1 million over 3 years for a phase 2 study of anti-PD-1 therapy for the treatment of HPV-associated recurrent respiratory papillomatosis.

New York Medical College will receive $1.7 million over 4 years for a phase 2 study of viral specific cytotoxic T lymphocytes for the treatment of refractory viral infections and T-cell immunodeficiency.

Privo Technologies LLC will receive $2 million over 4 years for a phase 1/phase 2 study of cisplatin patch (PRV111) for the treatment of oral cancer.

Targeted Therapy Technologies LLC will receive $660,000 over 3 years for a phase 1 study of episcleral topotecan for the treatment of retinoblastoma.

The University of Alabama at Birmingham will receive $750,000 over 3 years for a phase 1 study of oncolytic engineered herpes simplex virus therapy for the treatment of pediatric malignant cerebellar brain tumors.

The University of Texas MD Anderson Cancer Center will receive $1 million over 4 years for a phase 1/phase 2 study of the imipridone (ONC201, Oncoceutics) for treatment of acute myeloid leukemia.

University of California, San Diego, will receive $1.5 million over 3 years for a phase 2 study of temozolomide for the treatment of gastrointestinal stromal tumor.

“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,” Janet Maynard, MD, director of the FDA’s Office of Orphan Products Development, said in the release. “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.”

Amy Abernethy
Amy P. Abernethy

The FDA awarded 12 grants worth more than $15 million to fund clinical trials of medical products intended to treat patients with rare diseases.

The grants were awarded through the Orphan Products Clinical Trials Grants Program, a Congress-funded initiative that promotes clinical development of drugs, biologics, medical devices and medical foods for the treatment of rare diseases.

The program — created in 1983 — has provided more than $400 million to fund 600 new clinical studies, and its grants have supported research that led to marketing approval of more than 60 treatments for rare diseases.

“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,” FDA Principal Deputy Commissioner Amy P. Abernethy, MD, PhD, said in a press release. “We are encouraged by the amount of interest we continue to have in the grants program and are committed to working with researchers and industry to facilitate and support the study and development of treatments for patients with rare diseases.”

This year, more than 100 rare disease experts evaluated 89 clinical trial grant applications for their scientific and technical merit.

The recipients are as follows:

•  Chemocentryx Inc. will receive $1 million over 2 years for a phase 2 study of avacopan (CCX168) for the treatment of complement 3 glomerulopathy.

Cincinnati Children’s Hospital Medical Center will receive $750,000 over 3 years for a phase 1 study of PTC596 (PTC Therapeutics) for the treatment of diffuse intrinsic pontine glioma and high-grade gliomas.

Cincinnati Children’s Hospital Medical Center also will receive $1.7 million over 4 years for a phase 2 study of quercetin chemoprevention for the treatment of squamous cell carcinoma among patients with Fanconi anemia.

Columbia University Health Sciences will receive $2 million over 4 years for a phase 2 study of daily vitamin D for the treatment of respiratory complications from sickle cell anemia.

Cumberland Pharmaceuticals Inc. will receive $1 million over 3 years for a phase 2 study of oral ifetroban for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy.

Massachusetts General Hospital will receive $1 million over 3 years for a phase 2 study of anti-PD-1 therapy for the treatment of HPV-associated recurrent respiratory papillomatosis.

New York Medical College will receive $1.7 million over 4 years for a phase 2 study of viral specific cytotoxic T lymphocytes for the treatment of refractory viral infections and T-cell immunodeficiency.

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Privo Technologies LLC will receive $2 million over 4 years for a phase 1/phase 2 study of cisplatin patch (PRV111) for the treatment of oral cancer.

Targeted Therapy Technologies LLC will receive $660,000 over 3 years for a phase 1 study of episcleral topotecan for the treatment of retinoblastoma.

The University of Alabama at Birmingham will receive $750,000 over 3 years for a phase 1 study of oncolytic engineered herpes simplex virus therapy for the treatment of pediatric malignant cerebellar brain tumors.

The University of Texas MD Anderson Cancer Center will receive $1 million over 4 years for a phase 1/phase 2 study of the imipridone (ONC201, Oncoceutics) for treatment of acute myeloid leukemia.

University of California, San Diego, will receive $1.5 million over 3 years for a phase 2 study of temozolomide for the treatment of gastrointestinal stromal tumor.

“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,” Janet Maynard, MD, director of the FDA’s Office of Orphan Products Development, said in the release. “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.”