FDA Grants Orphan Drug Status To Immtech's Pafuramidine
FDA has granted Orphan Drug Designation to Immtech Pharmaceuticals's pafuramidine (DB289) for treating Human African Trypanosomiasis (HAT), also known as African sleeping sickness.
Orphan drug designation provides Immtech with numerous financial and regulatory benefits during pafuramidine's development, including government grants for conducting clinical trials, waiver of New Drug Application submission fees, tax credits, and a seven-year market exclusivity upon final FDA approval.
Pafuramidine is currently in phase III clinical trials for African sleeping sickness at six trial sites in Africa. The infectious disease, spread by tsetse flies, threatens approximately 60 million people in over 30 countries in sub-Saharan Africa. Current treatments for the disease are associated with high levels of toxicity and are difficult to administer. Safety data for pafuramidine has been positive to date, and the FDA and Ethics Committees responsible for the study oversight currently allow adolescents, pregnant women and nursing mothers to participate in these clinical trials. These groups are particularly vulnerable to the disease.
"The FDA's decision reflects the significant need for new treatments for African sleeping sickness," stated Carol Olson, MD, PhD, Immtech's Sr. Vice President and Chief Medical Officer. "Pafuramidine has the potential to be the first oral treatment for this fatal and neglected disease. Patients often put off screening and treatment because current drugs must be given by injection. A safe and effective pill that is readily available should facilitate cure for more patients in the early stage of the disease. Because pafuramidine is more user-friendly than existing therapies, we anticipate that it would be available in community clinics as well as at current African sleeping sickness treatment centers. Patients could be treated close to home, rather than traveling long distances to specialized centers."
Pafuramidine has previously been granted Orphan Drug Designation for treatment of pneumocystis pneumonia (PCP) and malaria. In addition to trials for African sleeping sickness, pafuramidine is currently in Phase III clinical trials for PCP, and in Phase II trials targeting malaria treatment and malaria prophylaxis.
"This is another positive milestone for Immtech as pafuramidine advances towards commercialization," commented Eric L. Sorkin, Immtech's Chairman and Chief Executive Officer. "We will continue to work closely with our consortium of scientists, and global collaborators to develop safer and more effective therapies for this and other global health threats."