February 22, 2022
1 min learn
The FDA granted orphan drug designation to CYNK-101, an investigational pure killer cell-based remedy for remedy of superior HER2/neu-positive gastric or gastroesophageal junction adenocarcinoma.
CYNK-101 (Celularity) is a gene-edited, allogeneic pure killer (NK) cell remedy composed of human placental hematopoietic stem cells engineered to precise a high-affinity and cleavage-resistant CD16 (FCGRIIIA) variant.
The FDA beforehand granted quick observe designation to CYNK-101 for a similar indication.
A nonrandomized part 1/part 2A scientific trial is evaluating CYNK-101 together with customary chemotherapy, trastuzumab (Herceptin, Genentech) and pembrolizumab (Keytruda, Merck) as first-line remedy for sufferers with domestically superior unresectable or metastatic HER2/neu-positive gastric or gastroesophageal junction adenocarcinoma.
“This [orphan drug] designation underscores the numerous unmet want for these sufferers and CYNK-101’s potential in a brand new first-line remedy technique,” Robert J. Hariri, MD, PhD, chairman and CEO of Celularity, stated in a company-issued press launch. “We’re dedicated to forging new remedy methods that leverage the distinctive properties of placental-derived mobile therapies to enhance the lives of sufferers with this difficult-to-treat most cancers.”
The FDA Workplace of Orphan Merchandise Growth grants orphan drug designation to novel medicine and biologics which are supposed for the secure and efficient remedy, analysis or prevention of uncommon illnesses or issues that have an effect on fewer than 200,000 individuals in the USA. The designation permits producers to qualify for numerous incentives, together with tax credit for certified scientific trials and — upon regulatory approval — 7 years of market exclusivity.