Orphan Products Grants Awarded
FDA Office of Orphan Products Development (OOPD)
On this page: Clinical Trials Grants Program Awards | Natural History Studies Grants Program Awards | Historical Information | Related Links
Clinical Trials Grants Program Awards
Clinical trial grants for orphan products are a proven method of successfully fostering and encouraging the development of new safe and effective medical products for rare diseases and conditions. The FDA’s Office of Orphan Products Development (OOPD) awards new clinical trial grants annually. For more information, see Clinical Trials Grants Program.
FDA has awarded hundreds of grants under this program since 1983. The program has funded clinical trials that have facilitated the approval of more than 85 products including drugs, biologics, and devices for rare diseases.
The most recent awards are listed below. For a full list of grants awarded since the program's inception, please visit our searchable list of funded grants.
Instructions below refer to the Orphan Product Grants Program database.
During searches, if more than one field is populated, results of the search will display as if the word “and” was put between the search criteria. For example, searching 'interferon' as a product and 'Smith' as an investigator will return only matches where both criteria are met. If you want all records that include either one or both criteria (i.e., an 'or' search), the searches must be run separately. A search with no criteria will return all grants, current and/or previous, depending on the button selected.
Disease Indication, Product Name, or Grant Title
Entering a search term in the Disease Indication field or Grant Title field will find any occurrence of the term in either of these fields, i.e., both fields are searched automatically. The database does not use a standard terminology, and searching both fields will increase the possibility of finding the appropriate records. For example, some records may have a disease name in the grant title that is listed differently in the indication. Searches on product name search only the Product Name field, therefore, if the search does not return expected records, the search should be repeated with the product name searched under the Grant Title field.
Principal Investigator, Institution, or Location
Grants can be searched by the name of the Principal Investigator, Institution, or Location. For Location, enter a city, state, or country. States should be entered as two letter abbreviations, e.g., 'NJ'. Entering only a location (e.g., 'NJ') will return all grants for investigators in New Jersey. For 'country', only countries outside the United States need to be entered.
Output Format
Results can be displayed as a list or downloaded as an Excel file. If displayed as a list, the number of records specifies how many records are returned on each page displayed. The default is 100 records per page. Output can also be sorted by several options, e.g., investigator or date.
Note: If you need help with accessing information in different file formats, see Instructions for Downloading Viewers and Players.
FY 2024 Clinical Trials Program Grants Awarded
In FY 2024, FDA received 51 grant applications, and awarded 7 new clinical trials that will help to accelerate the development of safe and effective treatments to address the significant and often unmet needs of those affected by rare diseases. The new awards will provide more than $17.2 million to clinical researchers over the next four years to advance the development of medical products for rare diseases. This includes additional funding to support innovative and efficient trial designs that can be used to advance treatments through product development and as models for future drug development in rare diseases. Reference: Request for Applications (RFA FD-23-001)
| Awardee | Principal Investigator | Description of Project | Amount (approximate) | Duration |
|---|---|---|---|---|
| Palvella Therapeutics, Inc. (Wayne, Pennsylvania) | Jeff Martini | Phase 3 study of PTX-022 (Sirolimus) for the treatment of microcystic lymphatic malformations | $2.6 million | 4 years |
| Sloan-Kettering Institute Cancer Research (New York, New York) | Alan Ho | Phase 2 study of avutometinib and defactinib for the treatment of RAF dimer-driven thyroid cancer | $1 million | 4 years |
| Sloan-Kettering Institute Cancer Research (New York, New York) | Joseph Oved | Phase 2a/2b Study of emapalumab for the treatment of pediatric aplastic anemia | $2.6 million | 4 years |
| Targeted Therapy Technologies, LLC (Somerset, New Jersey) | Ricardo Carvalho | Phase 2 expansion study of topotecan (chemoplaque) for the treatment of retinoblastoma | $2.6 million | 4 years |
| University of California Los Angeles (Los Angeles, California) | Anthony Heaney | Phase 2 study of fimepinostat for the treatment of Cushing disease | $1.7 million | 4 years |
| University of Pennsylvania (Philadelphia, Pennsylvania) | Tomas Aleman | Phase 1/2 study of recombinant adeno-associated virus serotype 8 (AAV8) vector LCA5 gene therapy for the treatment of Leber congenital amaurosis 5 (LCA5) | $3.4 million | 4 years |
| University of Pittsburgh at Pittsburgh (Pittsburgh, Pennsylvania) | Sawa Ito | Phase 2 study of interferon-gamma and donor lymphocyte infusion for the treatment of relapsed myeloid malignancies after allogeneic stem cell transplantation | $3.4 million | 4 years |
Natural History Studies Grants Program Awards
FDA has funded natural history studies since 2016 to help address significant unmet medical needs for patients with rare diseases. For more information, see: Natural History Studies Grants Program.
FY 2024 Natural History Studies Grants Awarded
In FY 2024, FDA received 53 natural history grant applications and funded three new grants totaling more than $4.7 million spread over four years to continue to advance the understanding of rare diseases. This includes one award made with additional support from the FDA Center for Drug Evaluation and Research (CDER) Accelerating Rare disease Cures (ARC) program. These studies will address knowledge gaps in support of future clinical trials.
Specifically, one study will utilize a collaborative network and novel machine learning techniques to assess patient-reported outcomes and stratify patients with pediatric intestinal failure (PIF) (the inability of the gastrointestinal tract to sustain life without supplemental nutrition or intestinal transplant). This has the potential to advance clinical trial design as there is currently no consensus on disease stratification or specific outcome measures in PIF. Another study will use machine learning to create a patient network and identify clinical outcomes and prognostic risk factors in children with autosomal recessive polycystic kidney disease (ARPKD), which will have the potential to advance the clinical study of promising candidate therapies identified in animal models. Another study will bring together international centers of excellence, patient advocacy groups, and industry partners currently developing interventions for congenital central hypoventilation syndrome (CCHS). The study aims to define clinical, functional, and quality of life assessments to develop effective treatment strategies in this complex disease that includes a life-threatening inability to regulate breathing necessitating life-long ventilatory support. Reference: Request for Applications (RFA-FD-22-001)
| Awardee | Principal Investigator | Description of Project | Amount (approximate) | Duration |
|---|---|---|---|---|
| Children's Hospital of Philadelphia (Philadelphia, Pennsylvania) | Erum Hartung | Retrospective and prospective study in autosomal recessive polycystic kidney disease (ARPKD) to create PKDnet to inform future clinical trial design | $1.6 million | 4 years |
| Lurie Children's Hospital of Chicago (Chicago, Illinois) | Debra Weese-Mayer | Prospective study in congenital central hypoventilation syndrome to establish measures that correlate with disease severity and inform therapeutic development | $1.6 million | 4 years |
| University of Pittsburgh At Pittsburgh (Pittsburgh, Pennsylvania) | Simon Horslen | Prospective study in pediatric intestinal failure to inform natural history and develop outcome measures | $1.5 million | 4 years |
Other Past and Ongoing OOPD-Supported Natural History Grants
| Awardee | Principal Investigator (Last Name, First Name) | Description of Project | Location (State, City) | Start Date (YYYY-MM-DD) | End Date (YYYY-MM-DD) |
|---|---|---|---|---|---|
| Children's Hospital Corp. | Tan, Wen Hann | Prospective Study in Angelman Syndrome | Massachusetts, Boston | 2017-09-15 | 2022-08-31 |
| Children's Hospital of Philadelphia | Lynch, David | Prospective Study in Friedreich Ataxia | Pennsylvania, Philadelphia | 2017-09-01 | 2023-02-28 |
| Cincinnati Children’s Hospital Medical Center | Trapnell, Bruce | Retrospective Study in Autoimmune Pulmonary Alveolar Proteinosis | Ohio, Cincinnati | 2022-09-01 | 2025-08-31 |
| Columbia University Health Sciences | Rubin, Mishaela | Prospective Study in Hypoparathyroidism | New York, New York | 2022-09-01 | 2026-08-31 |
| Columbia University Medical Center | Cohen, Adi | Prospective Study in Pregnancy & Lactation Associated with Osteoporosis | New York, New York | 2017-09-01 | 2023-08-31 |
| Jaeb Center for Health Research, Inc. | Ayala, Allison | Prospective Study in Gyrate Atrophy | Florida, Tampa | 2022-09-15 | 2027-08-31 |
| Johns Hopkins University | Lederman, Howard | Prospective Study in Ataxia-Telangiectasia | Maryland, Baltimore | 2022-09-01 | 2026-08-31 |
| University of Iowa | Gerke, Alicia | Retrospective Study in Sarcoidosis | Iowa, Iowa City | 2017-09-01 | 2020-08-31 |
| University of Minnesota | Walk, David | Retrospective and Prospective Study in Amyotrophic Lateral Sclerosis | Minnesota, Minneapolis | 2022-09-20 | 2026-08-31 |
| University of Pennsylvania | Fajgenbaum, David | Prospective Study in Castleman disease | Pennsylvania, Philadelphia | 2022-09-01 | 2026-08-31 |
| University of Tennessee Health Science Center | Ataga, Kenneth | Prospective Study in Sickle Cell Anemia | Tennessee, Memphis | 2017-09-01 | 2023-08-31 |
| University of Texas MD Anderson Cancer Center | Grubbs, Elizabeth | Prospective Study in Medullary Thyroid Carcinoma | Texas, Houston | 2019-09-01 | 2024-07-31 |
| Vanderbilt University Medical Center | Brittain, Evan | Prospective Study in Pulmonary Arterial Hypertension | Tennessee, Nashville | 2022-09-01 | 2026-08-31 |
| Vanderbilt University Medical Center | Soslow, Jonathan | Prospective Study in Cardiac Disease in Duchenne Muscular Dystrophy (DMD) | Tennessee, Nashville | 2019-09-01 | 2025-02-28 |
| Virginia Commonwealth University | Johnson, Nicholas | Prospective Study in Myotonic Dystrophy Type 1 to Determine Biomarkers in Clinical Endpoints | Virginia, Richmond | 2017-09-15 | 2027-02-28 |