The U.S. Food and Drug Administration today announced that it has 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 grants were awarded to principal investigators from academia and industry across the country.
The FDA awarded the grants through the Orphan Products Clinical Trials Grants Program, funded by Congress to encourage clinical development of drugs, biologics, medical devices and medical foods for the treatment of rare diseases. The grants are intended to substantially contribute to marketing approval of products to treat rare diseases or provide essential data needed for development of such products.
"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," said FDA Principal Deputy Commissioner Amy Abernethy, M.D., Ph.D. "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."
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.
The grants awarded are focused on supporting product development to meet the needs of patients impacted by a variety of rare diseases, mainly those affecting children and cancers. The recipients, principal investigators and approximate funding amounts, listed alphabetically, are:
"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," said Janet Maynard, M.D., director of the FDA's Office of Orphan Products Development. "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."
Three-quarters (75%) 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. Some of these diseases include Duchenne Muscular Dystrophy, a genetic disorder characterized by progressive muscle loss and weakness; sickle cell disease, a group of inherited red blood disorders which can cause anemia, infections and stroke; and Fanconi Anemia, a rare inherited condition that can result in bone marrow failure and has a high risk for squamous cell cancers.
Two-thirds (67%) of the new awards fund clinical studies of products for use in various rare cancers. For example, one of the new awards aims to advance research in brain cancers affecting children. This study enrolls affected children as young as 36 months and tests an innovative treatment approach to treat brain cancers using an engineered virus therapy designed to target and kill tumor cells while sparing normal brain cells. Another newly funded study evaluates a novel drug delivery system that delivers chemotherapy on a sustained basis directly to the eye to treat retinoblastoma, a rare cancer in the eye most commonly affecting young children. This delivery system may help address current barriers to drug delivery that patients face.
Past studies funded by this program that have resulted in or contributed to recent product approvals, include: fish oil triglycerides, a first FDA-approved lipid emulsion made from fish oil, approved as a source of calories and fatty acids in children with parenteral nutrition-associated cholestasis; and the first treatment (tafamidis meglumine and tafamidis) for heart disease caused by transthyretin mediated amyloidosis.
In addition to the FDA Orphan Products Clinical Trials Grants Program, the agency also supports natural history studies for rare diseases to further advance the FDA's mission of bringing new therapies to approval and patients.
For more information:
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
Media Inquiries: Sandy Walsh, 301-796-4669, firstname.lastname@example.org Consumer Inquiries: 888-INFO-FDA
SOURCE U.S. Food and Drug Administration
美国食品药品监督管理局（Food and Drug Administration）今天宣布，在未来四年内，它已经授予12项新的临床试验研究补助金，总额超过1500万美元，以加强为罕见疾病患者开发医疗产品。这些资助被授予全国学术界和工业界的主要研究者。
“35年多来， FDA 一直在为可能改变生命的罕见疾病患者的临床试验提供急需的资金支持。到目前为止，孤儿产品临床试验补助金计划的赠款已经支持研究，导致60多种治疗罕见疾病的治疗方法获得市场批准医学博士（M.D.）博士说：「我们对拨款计划的关注程度感到鼓舞，并致力与研究人员及业界合作，以促进及支持罕见病患者的治疗研究及发展。」
“大多数罕见疾病仍然没有批准的治疗方法， FDA 致力于促进产品开发领域的未满足需求。孤儿产品补助计划是 FDA 支持罕见疾病产品开发的几种方式之一。FDA 孤儿产品开发办公室主任 Janet Maynard 医学博士说：“自1983年创建以来，该项目已经提供了4亿多美元，用于资助600多项新的临床研究。“我们很高兴继续支持对各种罕见疾病的研究，这些疾病对患者几乎没有或根本没有治疗选择。通过帮助推动研究，我们希望加快罕见疾病产品的开发，并最终为最需要治疗的患者提供所需的治疗。”
四分之三（75%）的新奖励基金资助儿童入学，包括一个月以下的儿童。这些研究针对影响儿童的各种罕见疾病，并有可能促进这些疾病的治疗。其中一些疾病包括 Duchenne Music Dystrophy ，一种遗传性疾病，其特征是进行性肌肉丧失和虚弱；镰状细胞疾病，一组遗传性红细胞疾病，可引起贫血、感染和中风； Fanconi 贫血，一种罕见的遗传性疾病，可能导致骨髓衰竭，并有鳞状细胞癌的高风险。
过去由该计划资助的研究已经导致或促成了最近的产品批准，包括：鱼油甘油三酯，第一个 FDA 批准的由鱼油制成的脂乳，被批准为儿童的热量和脂肪酸来源与肠外营养相关的胆汁淤积；和首次治疗心脏病（ tafamidoglamine 和 tafadis ）引起的转甲状腺素介导的淀粉样变性。
除了 FDA 孤儿产品临床试验补助金计划外， FDA 还支持对罕见疾病的自然历史研究，以进一步推进 FDA 为批准和患者提供新疗法的使命。
媒体提问：桑迪·沃尔什，301-796-4669，桑迪。walsh @ fda.hhs.gov 消费者查询：888-INFO-FDA
美国食品药品监督管理局（Food and Drug Administration）