FDA News

FDA grants orphan drug designation to multi-antigen T-cell therapy for AML

The FDA granted orphan drug designation to MT-401, an allogeneic T-cell therapy for the treatment of acute myeloid leukemia, according to the agent’s manufacturer.

MT-401 (Marker Therapeutics) — an investigational, multi-tumor-associated antigen (MultiTAA)-specific T-cell therapy — will be tested in a phase 2 clinical trial of patients with AML after allogeneic stem cell transplantation.

T cells are taken from healthy donors and put through a two-step cell culture process. The result is a CD4/CD8 cell mixture that can recognize multiple tumor-specific antigens.

“We are pleased that the FDA has granted orphan designation to MT-401 and believe it is supportive of its potential to treat post-allogeneic stem cell transplant patients with AML — a devastating and pervasive blood disease with a high medical need for a treatment,” Peter L. Hoang, president and CEO of Marker Therapeutics, said in a company-issued press release.

“In investigator-sponsored trials, our MultiTAA-specific T-cell product candidate was well-tolerated, and we have observed clinical benefit across various liquid and solid tumors, suggesting the product candidate’s ability to induce a patient’s own T cells to expand for a more durable antitumor effect. We look forward to initiating our company-sponsored phase 2 study,” he added.

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 MT-401, an allogeneic T-cell therapy for the treatment of acute myeloid leukemia, according to the agent’s manufacturer.

MT-401 (Marker Therapeutics) — an investigational, multi-tumor-associated antigen (MultiTAA)-specific T-cell therapy — will be tested in a phase 2 clinical trial of patients with AML after allogeneic stem cell transplantation.

T cells are taken from healthy donors and put through a two-step cell culture process. The result is a CD4/CD8 cell mixture that can recognize multiple tumor-specific antigens.

“We are pleased that the FDA has granted orphan designation to MT-401 and believe it is supportive of its potential to treat post-allogeneic stem cell transplant patients with AML — a devastating and pervasive blood disease with a high medical need for a treatment,” Peter L. Hoang, president and CEO of Marker Therapeutics, said in a company-issued press release.

“In investigator-sponsored trials, our MultiTAA-specific T-cell product candidate was well-tolerated, and we have observed clinical benefit across various liquid and solid tumors, suggesting the product candidate’s ability to induce a patient’s own T cells to expand for a more durable antitumor effect. We look forward to initiating our company-sponsored phase 2 study,” he added.

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.

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