BeyondSpring Pharmaceuticals (NASDAQ:BYSI) received a complete response letter (CRL) from the FDA for its NDA seeking approval of plinabulin in combination with granulocyte colony-stimulating factor (G-CSF) for the prevention of chemotherapy-induced neutropenia (CIN).
The FDA issued the CRL to indicate it has completed a review of the application and determined that it cannot be approved in its present form.
The FDA’s CRL indicated that the results of the single 106 registrational trial was not sufficiently robust to demonstrate benefit and that a second well controlled trial would be required to satisfy the substantial evidence requirement to support the CIN indication.
“BeyondSpring strongly believes that plinabulin in combination with G-CSF has significant potential to raise the standard of care in CIN, a devastating side effect of chemotherapy,” Dr. Lan Huang, BeyondSpring’s co-founder, chairwoman and CEO, said in a statement.
The company plans to request a meeting with the FDA and remains committed to its goal of bringing plinabulin to cancer patients in need globally, she added.
BeyondSpring said it remains “confident in the efficacy and safety data for plinabulin in combination with G-CSF for the prevention of CIN.” The company expects to work closely with the FDA to consider a possible future clinical pathway for CIN, which may include a second study.
Plinabulin is the first drug candidate submitted for FDA approval that has the potential to work in the critical first week of chemotherapy treatment before G-CSF is effective, to prevent the onset and improve clinical outcomes of CIN.