Interestingly, this extremely positive vote follows an obscure briefing document, in which the FDA expressed uncertainty about whether the product offers an acceptable benefit-risk profile.
On Wednesday, during a joint meeting between the Oncologic Drugs Advisory Committee (ODAC) and the Cellular, Tissue and Gene Therapies Advisory Committee (ETGTAC), panelists voted 22 to 1 in favor of approval for talimogene laherparepavec (T-VEC, Amgen Inc). T-VEC is an experimental treatment for melanoma that is injected directly into lesions (i.e. tumors).
Interestingly, this extremely positive vote follows an obscure briefing document, in which the FDA expressed uncertainty about whether the product offers an acceptable benefit-risk profile in a therapeutic field where many new therapies have recently been launched. The briefing materials for this meeting can be found HERE.
T-VEC is a version of herpes simplex virus that has been genetically modified so it only replicates in cancer cells, destroying tumors while sparing healthy tissues. It also includes a gene that encodes a type of cytokine, or protein, called granulocyte-macrophage colony-stimulating factor (GM-CSF), which recruits immune-boosting cells to the tumor. The combination of the virus along with GM-CSF is expected to both speed up the drug’s cancer-killing effect while also stimulating the immune system to continue killing melanoma cells.
Amgen describes T-VEC as the first oncolytic immunotherapy, destined for use in patients with “injectable regionally or distantly metastatic melanoma lesions.“Oncolytic viruses,” or “cancer-killing viruses,” have been of great interest in cancer research for decades. Oncolytic viruses are designed to have both direct and indirect responses. In other words, the viruses themselves destroy tumor cells, then they prompt the immune system to continue killing the cancer long after the drug has cleared the body. Oncolytic viruses are unlike other cancer drugs, which indirectly kill cancer cells by stimulating the immune system to launch anti-tumor attacks.
The clinical trial data reviewed during this meeting came primarily from a phase 3 pivotal trial known as OPTiM (or study 005/05).This study involved 436 patients with malignant melanoma that was not surgically resectable who were randomly assigned in a 2:1 ratio to receive talimogene laherparepavec or control (GM-CSF). The treatment was injected directly into all reasonably injectable skin lesions.
Results of the study demonstrated a significant extension in durable response rates (DRR) with the immunotherapy compared with GM-CSF. DRR was the primary endpoint of the OPTiM study, with overall survival (OS) as a secondary endpoint. In the final analysis for OS, a 4.4-month extension was noted; however, this was not deemed statistically significant (P = .051).
ODAC panel member Brian Rini, MD, associate professor of Medicine at the Cleveland Clinic Lerner College of Medicine of Case Western Reserve University voted, “Yes,” and supported his vote by stating, “I thought the totality of the evidence was that there was a benefit, and it seems to be beneficial even [given] the evolving current landscape [in melanoma].”
The FDA is expected to make a final decision on approval of talimogene laherparepavec before the end of October 2015. The FDA is not bound by the Advisory Committee’s recommendation but often follows their advice.
ISS has over a decade of experience in developing regulatory strategies including support for FDA Advisory Committee meetings. We are involved in more FDA AdComs per year than even the largest pharmaceutical companies. For more information on how ISS can help you prepare for your next meeting, contact email@example.com.
Do you have an upcoming Advisory Committee Meeting?
Over the past few years, the number of ex-US manufacturers submitting a Class II exempt 510k device registration has increased substantially. For companies with medical devices that are approved and successful in a foreign market, the logical next step is to introduce their product into the US, which has one of the most lucrative healthcare markets in the world. Read more
FDA advisory committee meetings are important regulatory events for many manufacturers on the path to marketing approval. These meetings can quite literally result in the approval or rejection of a product based on the panel’s vote. Read more
One of the main challenges our clients face is determining which regulatory pathway is best for their products. For example, what if an OTC product has a Monograph active ingredient but that ingredient is present at a different amount than the permitted amount? Or what if the label claims a novel indication not specified in a Monograph? If you are debating pursuit of either regulatory pathway for your OTC drug, it is critical to consider cost, timeline, and label claims to ensure your products has a unique stance in today’s market. Read more
US distributors and agents trying to import rapid antibody tests for coronavirus face a number of obstacles. The good news is that FDA has opened up the Emergency Use Authorization (EUA) program to include SARS-CoV-2 (the virus) and COVID-19 (the disease), expediting time to market. Read more
The journey to scientific and commercial success is often complex and always critical, if you are looking for an expert partner to help steer you to confident solutions, contact us todayContact us