Immuno-Oncology Resource Center

Immuno-Oncology Resource Center

April 14, 2021
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CYNK-001 receives orphan drug designation for malignant gliomas

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The FDA granted orphan drug designation to CYNK-001 for the treatment of malignant gliomas, according to the agent’s manufacturer.

CYNK-001 (Celularity) is an allogeneic natural killer cell therapy.

The nongenetically modified, cryopreserved human placental hematopoietic stem cell-derived therapy is being developed as a treatment for hematologic malignancies, solid tumors and infectious diseases.

A phase 1 trial is underway to evaluate CYNK-001 for treatment of glioblastoma multiforme.

“We are very pleased the FDA has granted orphan designation in malignant gliomas to continue to develop off-the-shelf therapies for serious unmet clinical needs,” Robert J. Hariri, MD, PhD, founder, chairman and CEO of Celularity, said in a company-issued press release. “Building on the FDA’s recent decision to grant fast track status to CYNK-001, we view the orphan drug designation as yet another milestone on our journey to deliver patients a potentially novel treatment.

“To date, we have observed the potential of CYNK-001 in multiple preclinical models, as well as early evidence of activity in the clinic, and believe this approach may shift the paradigm in augmenting the body’s natural immune response to diseases such as glioblastoma, other cancer indications and infectious diseases,” Hariri added. “We are very excited to continue working with the FDA on the development of this exciting therapy.”

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.