Immtech Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for Pafuramidine (DB289) to treat Pneumocystis Jiroveci Pneumonia (PCP). PCP is a common life-threatening opportunistic infection in HIV/AIDS and other immunosuppressed patients.
This FDA orphan drug designation provides Immtech with financial and regulatory benefits during the development course of Pafuramidine, including government grants for conducting clinical trials, waiver of the Prescription Drug User's Fee for submission of the new drug application for Pafuramidine maleate for PCP, tax credits, and a seven-year market exclusivity upon final FDA approval.
Carol Olson, MD, PhD, Immtech's Sr. vice president and chief medical officer stated, "We appreciate the FDA's recognition of Pafuramidine as an orphan drug in treatment of PCP. We believe Pafuramidine will be an important new agent for the treatment of this life threatening disease because many patients experience significant adverse effects related to current treatments. In addition, scientists and clinicians are concerned about potential loss of the standard treatments' effectiveness due to emerging resistance to these drugs in the pneumocystis organism. Thus, a new effective and more tolerable drug for treating PCP would be a significant benefit to these patients."
Pafuramidine, the active component of Immtech's oral drug candidate Pafuramidine maleate, is currently in phase III clinical trials in HIV/AIDS patients with PCP. Previous clinical research has indicated that Pafuramidine could have similar efficacy to trimethoprim-sulfamethoxazole, the current standard therapy for PCP. We also expect Pafuramidine to have fewer and less severe adverse effects, including the significant allergic reactions that can occur with TMP-SMX.
PCP affects patients with HIV/AIDS, many different types of cancer, and patients with solid organ transplants and autoimmune disorders. In addition to the PCP phase III clinical trial, Immtech is initiating studies to evaluate Pafuramidine for the PCP prophylaxis market, which is projected to be USD $1 billion. "Immtech is committed to meeting the worldwide demand for safer, more effective options for managing PCP, a life threatening disease for many patients." commented Eric L. Sorkin, chairman and chief executive officer, "Our goals is to make this product available to the patients as soon as possible. We are focused on advancing toward a strong, cost effective, and timely market launch for Pafuramidine."