FDA Grants Full Approval to ONTAK for Use in Patients with Cutaneous T-Cell Lymphoma

The US Food and Drug Administration (FDA) has approved an efficacy supplemental biologics license application (sBLA) for Eisai Corporation of North America’s (Woodcliff Lake, NJ) ONTAK (denileukin diftitox).

The FDA’s action marks the conversion of an accelerated approval indication to full approval and is based on data from a Phase 3 clinical trial that evaluated the overall efficacy and safety of ONTAK in certain patients with recurrent cutaneous T-cell lymphoma (CTCL). The accelerated approval program is intended to make promising products for life threatening diseases available on the market on the basis of preliminary evidence prior to formal demonstration of patient benefit.

The drug is now fully approved for the treatment of patients with persistent or recurrent CTCL whose malignant cells express the CD25 component of the interleukin (IL)-2 receptor (CD25+). A separate efficacy supplement that included data from patients with CTCL whose malignant cells did not test positive for the CD25 component of the IL-2 receptor received a complete response letter.

CTCL is a term for a group of rare malignant lymphomas with primary manifestation in the skin. This trial was the largest Phase 3, randomized, double blind, placebo-controlled trial ever conducted in CTCL.

The study met its primary endpoint of overall response rate (ORR). ORR is the sum of complete and partial responses seen in a study, divided by the number of evaluable patients.

Eisai release