Rare Diseases: FDA Awards Grants for 21 Clinical, Natural History Studies

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Rare Diseases: FDA Awards Grants for 21 Clinical, Natural History StudiesPosted 06 October 2017By Michael Mezher

The US Food and Drug Administration (FDA) on Friday awarded $32 million in research grants to fund 15 clinical studies and six natural history studies for rare diseases.

The grants are being funded through FDA's orphan products grants program, with $22 million going to the clinical studies and $9.8 million in combined funding from FDA and the National Center for Advancing Translational Sciences' (NCATS) Therapeutics for Rare and Neglected Diseases program going to fund the natural history studies.

About a third of the clinical trials being funded by the grants are aimed at rare forms of cancer, particularly cancers that affect the brain and peripheral nervous system. Other studies set to receive funding include trials to address unmet needs for patients with diseases such as sickle cell disease, Prader-Willi syndrome and cystine nephrolithiasis.

In total, FDA says it received 76 grant applications for clinical trials in FY 2017 and 80 grant applications for natural history studies under the grants program.

According to FDA, the orphan products grants program has paid out more than $390 million to more than 600 clinical studies since its creation under the 1983 Orphan Drug Act.  The agency also says that studies funded by the program have been used to support the approval of 55 orphan drugs.

Natural History Studies

This marks the first time FDA is funding natural history studies through its grants program after the agency announced the availability of the grants in February 2016.

Natural history studies are used to gain a greater understanding of a disease and its progression. For rare diseases, natural history studies can be an important tool for establishing baseline information about a disease and identifying potential outcomes or biomarkers to use in clinical trials.

In some cases, natural history data can be used as a historical control in settings where patient recruitment is especially challenging or where a placebo arm would be unethical.

"One of the challenges we encounter developing therapies for rare diseases is the lack of natural history data to guide the design of successful clinical trials," said Nora Yang, director of portfolio management and strategic operations in NCATS' Division of Pre-Clinical Innovation.

Speaking at RAPS' 2017 Convergence in September, FDA Commissioner Scott Gottlieb said the agency is working with researchers to develop natural history models to simulate placebo arms for studies in diseases such as Alzheimer's and muscular dystrophy.

"If we're able to make better use of rigorous, reliable natural history models, especially for rare diseases, it can help us make the clinical trial process more efficient," Gottlieb said.

FDA Rare Disease Study Grants
Grantee	Study	Amount (Approximate)
Clinical Trials
AADi, LLC	Phase 2 Study of ABI-009 for the Treatment of Advanced Perivascular Epithelioid Cell Tumors	$2 million over four years
Albert Einstein College of Medicine	Phase 2 Study of Topical Sodium Nitrite for the Treatment of patients with Sickle Cell Disease & Leg Ulcers	$2 million over four years
Albert Einstein College of Medicine	Phase 2 Study of Oxytocin for the Treatment of Hyperphagia in Prader-Willi Syndrome	$1.5 million over three years
Alkeus Pharmaceuticals, Inc	Phase 2 Study of ALK-001 for the Treatment of Stargardt Disease	$250,000 over one year
CereNova, LLC	Phase 2A Study of CN-105 for the Treatment of Intracerebral Hemorrhage	$1 million over two years
Columbia University Medical Center	Phase 2 Study of Teriparatide for the Treatment of Idiopathic Osteoporosis in Premenopausal Women	$1.9 million over four years
Columbia University Medical Center	Phase 2 Study of PLX3397 + Sirolimus for the Treatment of Malignant Peripheral Nerve Sheath Tumors	$2 million over four years
Dana-Farber Cancer Institute	Phase 1 Study of dual PI3K/BRD4 Inhibitor SF1126 for the Treatment of Neuroblastoma	$750,000 over three years
Duke University	Phase 2 Study of Belatacept, Alemtuzumab, and Sirolimus in Renal Transplantation	$1 million over three years
Johns Hopkins University	Phase 2a Study of Rifampin, Merrem and Augmentin for the Treatment of Pulmonary Tuberculosis	$2 million over four years
New York Medical College	Phase 2 Defibrotide for the Prevention of Complications in High-Risk Sickle Cell Disease Patients Following Allogeneic Stem Cell Transplantation	$1.75 million over four years
Protalex, Inc	Phase 1/2 Study of PRTX-100 for the Treatment of Immune Thrombocytopenia	$500,000 over two years
Sloan-Kettering Institute for Cancer Research	Phase 2 Study of MEK162 & Imatinib for the Treatment of Gastrointestinal Stromal Tumors	$2 million over four years
Tocagen Inc	Phase 2/3 Study of Toca 511 +Toca FC versus SOC in Recurrent Glioblastoma and Anaplastic Astrocytoma	$2 million over four years
University of California, San Francisco	Phase 2 Study of Lipoic Acid for the Treatment of Cystine Nephrolithiasis	$2 million over four years
Natural History Studies
Children's Hospital of Philadelphia	Prospective study in Friedreich's ataxia	$2 million over 5 years
Columbia University Medical Center	Prospective study in pregnancy and lactation-associated osteoporosis	$2 million over 5 years
University of Iowa	Retrospective study in sarcoidosis	$300,000 over 2 years
University of North Carolina at Chapel Hill	Prospective study in sickle cell anemia to determine biomarkers of endothelial function changes in chronic kidney disease	$2 million over 5 years
Children's Hospital Corporation	Prospective study in Angelman syndrome	$2 million over 5 years
University of Utah	Prospective study in Myotonic Dystrophy Type 1 to Determine Biomarkers and Clinical Endpoints	$2 million over 5 years

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