Novel leukaemia drug receives FDA Orphan Drug Designation

Blood cancer

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to Pleco Therapeutics’s lead compound PTX-252 for the treatment of acute myeloid leukaemia (AML).

PTX-252 is a novel molecular entity developed to increase the sensitivity of cancer cells to chemotherapy.

The FDA’s designation acknowledges the potential of PTX-252 in addressing AML, a rare blood cancer with a poor prognosis.

Orphan Drug Designation is granted to medicines that treat or prevent life-threatening or chronically debilitating rare disease, and with either no currently approved method of prevention or treatment or with significant clinical benefit when compared with existing treatments.

Orphan Drug Designation qualifies companies for incentives, including tax credits for clinical trials, exemption of user fees, and seven years of market exclusivity after approval.

“We are very excited to receive this designation for PTX-252 from the FDA,” stated Ivo Timmermans, Chief Executive Officer of Pleco Therapeutics. “This milestone underscores our commitment to innovative therapies for rare diseases and it brings hope to AML patients who have limited treatment options. Our team is dedicated to advancing this therapy through clinical development as swiftly as possible.”

In the development of the lead compound PTX-252, Pleco collaborated with Belgian company Hyloris Pharmaceuticals. Stijn Van Rompay, Chief Executive Officer of Hyloris, comments: “Securing orphan drug designation for PTX-252 stands as a testament to our unwavering commitment to advancing the frontiers of scientific discovery within the repurposing space.”

Worldwide the incidence of AML is estimated to be 350,000 cases per year (4.7 cases per 100,000 population, Globocan). In the US, there were an estimated 20,050 new cases of AML in 2022 and 11,540 deaths.

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