FDA Awards 12 Grants for Development of Orphan Drugs

Agency To Provide $18 Million Over the Next Four Years

About 42 percent of the money allocated this week by the FDA’s Orphan Products Clinical Trials Grants Program will go to the development of medications for rare diseases that affect children and adolescents, the FDA announced. A few of those include Stargardt disease, a juvenile genetic eye disorder that causes loss of vision; dystrophic epidermolysis bullosa, which causes painful blisters; and bronchopulmonary dysplasia, a lung condition that affects infants. Money allocated toward research and development for these diseases are:

  • $1.75 million to Leonide Saad of Alkeus Pharmaceuticals for a phase 2 study of ALK-001 for the treatment of Stargardt disease
  • $1.5 million to John Maslowski of Fibrocell Technologies for a phase 1/2 study of gene-modified ex-vivo autologous fibroblasts for the treatment of dystrophic epidermolysis bullosa
  • $2 million to Matthew Laughon of the University of North Carolina at Chapel Hill for a phase 2 study of sildenafil for the prevention of bronchopulmonary dysplasia

“Developing a treatment for a rare disease can be especially challenging,” said FDA Commissioner Scott Gottlieb, MD, in a statement. “Given the often small number of patients affected by certain very rare diseases, there can be limited markets for new treatments, and as a result fewer resources devoted to researching these opportunities.”

Grant money allocated by the Orphan Products Clinical Trials Grants Program, which was launched in 1983, has led to FDA approval of more than 60 orphan products.

Of the $18 million in grant money the FDA unveiled on Monday, 33 percent will go toward accelerating cancer research by enrolling patients with rare forms of cancer in clinical trials. Those rare cancers include advanced pancreatic cancer, head and neck squamous cell carcinoma, myelodysplastic syndrome and acute myeloid leukemia.

Another 25 percent will help fund studies evaluating products for rare endocrine disorders, including Cushing disease, dopamine agonist intolerant hyperprolactinemia, and congenital adrenal hyperplasia.

“New scientific advances offer more opportunities to develop these potential cures, Gottlieb said. “With efficient regulation, proper incentives for product development and the continued support of patients, providers and researchers, we have more opportunities to pursue these advances than ever before.”

Source: FDA.gov, September 24, 2018