FDA News

FDA grants orphan drug designation to PCM-075 for acute myeloid leukemia

The FDA granted orphan drug designation to PCM-075 for the treatment of patients with acute myeloid leukemia, according to the drug’s manufacturer.

PCM-075 (Trovagene) is an oral, highly selective adenosine triphosphate competitive inhibitor of the serine/threonine polo-like kinase 1 enzyme, which is overexpressed in many cancer types.

“AML is an aggressive cancer of the blood and bone marrow with approximately 20,000 new cases in the U.S. each year,” Bill Welch, CEO of Trovagene, said in a company-issued press release. “We see the FDA's granting of orphan drug designation for PCM-075 as underscoring the medical need for new therapies for patients with AML and an important step forward in our clinical development program.”

Studies have shown that inhibition of polo-like-kinases can induce tumor cell death. The addition of polo-like kinase inhibition to standard low-dose cytarabine improved response rates in a phase 2 trial of patients with AML (31% vs. 13.3%).

Following the completion of a phase 1 dose-escalation study, Trovagene initiated a phase 1b/phase 2 open-label trial, designed to evaluate the safety and antileukemic activity of PCM-075 in combination with standard-of-care therapy in patients with AML. The study — led by Jorge Cortes, MD, deputy department chair of leukemia at The University of Texas MD Anderson Cancer Center — will be conducted at 10 research centers across the United States.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

The FDA granted orphan drug designation to PCM-075 for the treatment of patients with acute myeloid leukemia, according to the drug’s manufacturer.

PCM-075 (Trovagene) is an oral, highly selective adenosine triphosphate competitive inhibitor of the serine/threonine polo-like kinase 1 enzyme, which is overexpressed in many cancer types.

“AML is an aggressive cancer of the blood and bone marrow with approximately 20,000 new cases in the U.S. each year,” Bill Welch, CEO of Trovagene, said in a company-issued press release. “We see the FDA's granting of orphan drug designation for PCM-075 as underscoring the medical need for new therapies for patients with AML and an important step forward in our clinical development program.”

Studies have shown that inhibition of polo-like-kinases can induce tumor cell death. The addition of polo-like kinase inhibition to standard low-dose cytarabine improved response rates in a phase 2 trial of patients with AML (31% vs. 13.3%).

Following the completion of a phase 1 dose-escalation study, Trovagene initiated a phase 1b/phase 2 open-label trial, designed to evaluate the safety and antileukemic activity of PCM-075 in combination with standard-of-care therapy in patients with AML. The study — led by Jorge Cortes, MD, deputy department chair of leukemia at The University of Texas MD Anderson Cancer Center — will be conducted at 10 research centers across the United States.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.