Amgen’s T-Vec Cancer Therapy Gets FDA Panel OK
Published: May 04, 2015
April 30, 2015
By Mark Terry, BioSpace.com Breaking News Staff
A panel for the U.S. Food and Drug Administration (FDA) voted yesterday to approve the Biologics License Application (BLA) for talimogene laherparepvec monotherapy for Thousand Oaks, Calif.-based Amgen . Twenty-two of the 23 members of the FDA panel voted in approval of T-Vec.
The drug, T-Vec, is a cancer therapy built out of a herpes simplex virus that has been engineered to produce granulocyte macrophage colony-stimulating factor (GM-CSF), a growth factor that stimulates stem cells to produce immune cells. As an immunotherapy, T-Vec is injected directly into tumors. There, it replicates inside the tumor cells, causing them to rupture and die. The cancer cell rupture releases tumor-derived particles along with GM-CSF, which stimulates the immune system to attack the cancer cells.
Earlier this week the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee and the Oncologic Drugs Advisory Committee published a preliminary review that expressed concerns over study design and results, with a particular concern over study bias. “These concerns include,” the report stated, “appropriateness of the study control; differential outcome assessments in the two arms of the study; the reliability of response assessments; the meaningfulness of the primary endpoint of durable response rate; the absence of a clear effect on overall survival; and limited evidence that the product has a systemic effect.”
There was concern that 278 participants had dropped out of the study, which brought into question whether investigators biased their decisions in favor of positive outcomes. However, despite these issues, the committee concluded that, “The study results for the primary endpoint are statistically robust. Therefore, FDA believes that any bias that might have occurred in the study conduct would not change the study results sufficiently to alter the overall interpretation that talimogene laherparepvec had an effect on durable response rate.”
Many analysts believe the approval of T-Vec could be the first of an entirely new class of virus-based cancer drugs. Amgen acquired the rights to T-Vec in 2011 from its inventor, Biovex, for $425 million and an additional $575 million in milestone payments.
Despite the new approach, T-Vec enters a market that already has a number of new and effective treatments for melanoma. This includes Yervoy (ipilimumab) and Opdivo (nivolumab) from Bristol-Myers Squibb Company , and Keytruda (pembrolizumab) from Merck & Co. .
Amgen also announced yesterday that the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) will review data regarding its BLA for Repatha (evolocumab) for the treatment of high cholesterol at a meeting on June 10, 2015.
“There is a critical need for additional treatment options for high-risk patients who are unable to control their high cholesterol with currently available therapies,” said Sean Harper, executive vice president of research and development at Amgen in a statement. “We look forward to discussing the efficacy and safety data from our clinical program with the members of the Committee.”
The T-Vec data is the result of Study 005/05, also called OPTiM, a Phase III, multicenter, open-label, randomized trail that compared T-Vec to GM-CSF in patients with advanced melanoma that was not surgically resectable. The study involved 436 patients and T-Vec significantly improved durable response rate (DDR) with 16.3 percent of T-Vec patients achieving a complete response (CR) or partial response (PR) within the first 12 months of treatment.
“It is clear from today’s discussion that the committee recognized the importance of the need for new therapeutic options for patients with metastatic melanoma,” Amgen said in a statement after the FDA meeting. “We look forward to talking with the FDA about how to best make talimogene laherparepvec monotherapy available to patients as they complete their review of the Biologics License Application.”