The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting a marketing authorisation for the gene therapy Luxturna (voretigene neparvovec), for the treatment of adults and children suffering from inherited retinal dystrophy caused by RPE65 gene mutations, a rare genetic disorder which causes vision loss and usually leads to blindness.
Americans depend on the U.S. Food and Drug Administration to help ensure that the foods they buy and consume are safe.
Our teams routinely work with food producers on voluntary recalls, and when necessary and where applicable, mandate recalls in order to keep people from getting sick or being harmed. To promote these goals, FDA is advancing an important new policy.
The U.S. Food and Drug Administration announced that it has awarded 12 new clinical trial research grants totaling more than USD18 million over the next four years to enhance the development of medical products for patients with rare diseases. These new 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.
This program is funded by Congressional appropriations and encourages clinical development of drugs, biologics, medical devices or medical foods for use in rare diseases. The grants are intended for clinical studies evaluating the safety and effectiveness of products that could either result in, or substantially contribute to, the FDA approval of products targeted to the treatment of rare diseases. Grant applications were reviewed and evaluated for scientific and technical merit by more than 100 rare disease experts, which included representatives from academia, the National Institutes of Health and the FDA.
The grant recipients, principal investigators and approximate funding amounts, listed alphabetically, are: Alkeus Pharmaceuticals, Inc. (Cambridge, Massachusetts), Leonide Saad, phase 2 study of ALK-001 for the treatment of Stargardt disease – USD1.75 million over four years; Arizona State University-Tempe Campus (Tempe, Arizona), Keith Lindor, phase 2 study of oral vancomycin for the treatment of primary sclerosing cholangitis – USD2 million over four years; Cedars-Sinai Medical Center (Los Angeles), Shlomo Melmed, phase 2 study of seliciclib for the treatment of Cushing disease – USD2 million over four years; Columbia University of New York (New York), Yvonne Saenger, phase 1 study of talimogene laherparepvec for the treatment for advanced pancreatic cancer – USD750,000 over three years; Emory University (Atlanta), Eric Sorscher, phase 1/ 2 study of Ad/PNP fludarabine for the treatment of head and neck squamous cell carcinoma – USD1.5 million over three years.
Fibrocell Technologies, Inc. (Exton, Pennsylvania), John Maslowski, phase 1/2 study of gene-modified ex-vivo autologous fibroblasts for the treatment of dystrophic epidermolysis bullosa – USD1.5 million over four years; Johns Hopkins University (Baltimore), Amy Dezern, phase 1/2 study of CD8-reduced T cells for the treatment of myelodysplastic syndrome or acute myeloid leukemia – USD750,000 over three years; Oncolmmune, Inc. (Rockville, Maryland) Yang Liu, phase 2b study of CD24Fc for the prevention of graft versus host disease – USD2 million over four years; Patagonia Pharmaceuticals, LLC (Woodcliff Lake, New Jersey), Zachary Rome, phase 2 study of PAT-001 (isotretinoin) for the treatment of congenital ichthyosis – USD1.5 million over three years; The General Hospital Corporation (Boston), Stephanie Seminara, phase 2 study of kisspeptin for the treatment of dopamine agonist intolerant hyperprolactinemia – USD1.4 million over four years; University of Minnesota (Minneapolis), Kyriakie Sarafoglou, phase 2a study of subcutaneous hydrocortisone infusion pump for the treatment of congenital adrenal hyperplasia – USD1.4 million over three years; University of North Carolina at Chapel Hill (Chapel Hill, North Carolina), Matthew Laughon, phase 2 study of sildenafil for the prevention of bronchopulmonary dysplasia – USD2 million over four years
One-third (33 percent) of the new awards aim to accelerate cancer research by enrolling patients with rare forms of cancer, including advanced pancreatic cancer, head and neck squamous cell carcinoma, myelodysplastic syndrome and acute myeloid leukemia. Another 25 percent of the new awards fund studies evaluating drug products for rare endocrine disorders, including Cushing disease, dopamine agonist intolerant hyperprolactinemia and congenital adrenal hyperplasia. Another study addresses an unmet need in primary sclerosing cholangitis, a rare, chronic and potentially serious bile duct disease.
About 42 percent of the grants fund studies which enroll children and adolescents, targeting a variety of rare diseases in children such as Stargardt disease, a juvenile genetic eye disorder that causes progressive vision loss; dystrophic epidermolysis bullosa, a genetic condition that causes the skin to be fragile resulting in painful blisters; and bronchopulmonary dysplasia, a serious lung condition that affects infants.
To date, the program’s grants have supported research that led to the marketing approval of more than 60 orphan products. Among the recent product approvals which were supported by studies funded by this grants program are a marketing approval for a much-needed treatment of human immunodeficiency virus type 1 (HIV-1) infection in adults with multidrug resistant HIV-1 infection and another approval to reduce the acute complications of sickle cell disease in adult and pediatric patients.
The FDA is also currently supporting six natural history studies for rare diseases to further advance the mission of bringing new therapies to market.
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