FDA grants orphan drug designation to eFT508 for DLBCL

The FDA granted orphan drug designation to eFT508 for the treatment of diffuse large B-cell lymphoma, according to the drug’s manufacturer.

An open-label phase 1/phase 2 study of eFT508 (eFFECTOR Therapeutics) — a highly selective inhibitor of MNK1 and MNK2 — has been initiated to evaluate the safety, pharmacokinetics, pharmacodynamics and antitumor activity of the agent in patients with B-cell hematologic malignancies.

The sequential-group, dose-escalation and cohort-expansion trial will establish a maximum tolerated dose and recommended dose for further evaluation. Investigators also will assess preliminary efficacy of the compound.

“Diffuse large B-cell lymphoma is an aggressive disease for which patients badly need additional treatment options,” Steve Worland, PhD, president and CEO of eFFECTOR, said in a company-issued press release. “We hope that eFT508 — a selective translation regulator that blocks multiple oncogenic growth and proliferation pathways, as well as activates antitumor immunity — will prove effective in treating patients afflicted with this devastating disease.”

The company also is conducting a phase 1/phase 2 dose-escalation trial of eFT508 in patients with solid tumors.

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.