Vemurafenib/Cobimetinib Combo for Melanoma Approved by FDA

“The FDA has approved a combination of vemurafenib (Zelboraf) and cobimetinib (Cotellic) to treat patients with metastatic or unresectable BRAF V600E/K mutation-positive melanoma. The approval was based on based on an extension in progression-free survival (PFS) in the phase III coBRIM study.

“In the data submitted to the FDA, the median PFS with the combination was 12.3 versus 7.2 months with vemurafenib plus placebo (HR, 0.58; 95% CI, 0.46-0.72). PFS was the primary endpoint of the study with secondary outcome measures including overall survival (OS), objective response rate (ORR), duration of response, and safety.”


Bristol-Myers Squibb Receives Approval from the U.S. Food and Drug Administration for Yervoy (ipilimumab) as Adjuvant Treatment for Fully Resected Stage III Melanoma

Bristol-Myers Squibb Company BMY, -0.27% today announced that the U.S. Food and Drug Administration (FDA) has approved Yervoy (ipilimumab) 10 mg/kg for the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm who have undergone complete resection including total lymphadenectomy. This approval is based on clinical data from a pivotal Phase 3 trial, CA184-029 (EORTC 18071), which demonstrated Yervoy 10 mg/kg significantly improved recurrence-free survival (RFS) vs. placebo in this setting, with a 25 percent reduction in the risk of recurrence or death. The median RFS was 26 months (95% ci:19)(95% ci:39) for Yervoy vs. 17 months (95% ci:13)(95% ci:22) for placebo (hazard ratio [HR]=0.75; 95% CI: 0.64, 0.90; p<0.002). Yervoy is the first and only FDA-approved immune checkpoint inhibitor in the adjuvant treatment for fully resected Stage III melanoma (lymph node >1 mm).”


Bristol-Myers Squibb’s Opdivo (nivolumab) Receives Expanded FDA Approval in Previously-Treated Metastatic Non-Small Cell Lung Cancer (NSCLC), Offering Improved Survival to More Patients

Bristol-Myers Squibb Company (NYSE:BMY) today announced that the U.S. Food and Drug Administration (FDA) has approved Opdivo (nivolumab) injection, for intravenous use, for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR mutation or ALK translocation should have disease progression on appropriate targeted therapy prior to receiving Opdivo. In a Phase 3 trial, CheckMate -057, Opdivodemonstrated superior overall survival (OS) in previously treated metastatic non-squamous NSCLC compared to chemotherapy, with a 27% reduction in the risk of death (hazard ratio: 0.73 [95% CI: 0.60, 0.89; p=0.0015]), based on a prespecified interim analysis. The median OS was 12.2 months in the Opdivo arm (95% CI: 9.7, 15.0) and 9.4 months in the docetaxel arm (95% CI: 8.0, 10.7). This approval expands Opdivo’s indication for previously treated metastatic squamous NSCLC to include the non-squamous patient population. Squamous and non-squamous NSCLC together represent approximately 85% to 90% of lung cancer cases.”


FDA Approves Pembrolizumab for PD-L1-Positive Lung Cancer

“The FDA granted an accelerated approval to pembrolizumab (Keytruda) as a treatment for patients with pretreated advanced non­–small cell lung cancer (NSCLC) across all histologies whose tumors express PD-L1. The PD-1 inhibitor was approved along with a companion diagnostic, the PD-L1 IHC 22C3 pharmDx test, and is indicated for patients who progressed on or after platinum-containing chemotherapy or EGFR-or ALK-targeted agents in patients harboring those mutations.

“The approval was based on data from the phase I KEYNOTE-001 trial, in which the overall response rate (ORR) with the drug was 41% among a subgroup of 61 patients with pretreated PD-L1­–positive advanced NSCLC as determined by the 22C3 pharmDx diagnostic test. Response duration ranged from 2.1 to 9.1 months. A survival improvement has yet to be demonstrated in a clinical trial, and the accelerated approval is contingent upon the eventual outcomes of confirmatory studies.”


Nivolumab/Ipilimumab Combination FDA Approved for BRAF Wild-Type Melanoma

“The combination of nivolumab and ipilimumab has received accelerated FDA approval as a treatment for patients with BRAF V600 wild-type (WT) unresectable or metastatic melanoma, based on findings from the phase II CheckMate-069 study.

” ‘Historically, metastatic melanoma has been a difficult disease to treat. Now, a new treatment option based on the combination of two valued immuno-oncology agents demonstrates significant efficacy versus ipilimumab in metastatic melanoma,’ said Jedd D. Wolchok, MD, PhD, chief, Melanoma and Immunotherapeutics Service, Department of Medicine and Ludwig Center at Memorial Sloan Kettering Cancer Center, in a statement. ‘Today’s approval represents a step forward for the melanoma community, providing hope for patients with metastatic melanoma.’ ”


FDA Grants Breakthrough Therapy Designation to Azedra for Pheochromocytoma, Paraganglioma

“The FDA granted breakthrough therapy designation to Ultratrace iobenguane I-131 for the treatment of patients with iobenguane-avid metastatic or recurrent pheochromocytoma and paraganglioma, according to a press release from the drug’s manufacturer.

“Ultratrace iobenguane I-131 (Azedra, Progenics Pharma), a late-state radiotherapeutic drug candidate, is being evaluated in a phase 2b trial of patients with malignant pheochromocytoma or paraganglioma. Pheochromocytomas — rare tumors usually found within one or both adrenal glands — are referred to as paragangliomas when they arise in other areas of sympathetic nerve cells.

“Ultratrace iobenguane I-131 — a very high specific activity form of iobenguane I-131 — is produced using Progenics’ Ultratrace platform, a technology designed to prevent unlabeled iobenguane from being carried through the manufacturing process to the final formulation, according to information listed on Progenics’ website. Ultratrace iobenguane I-131 is designed for use as an imaging agent in order to determine a therapeutic dose that is safe for normal organs, as well as for therapy.”


FDA Approves Iressa for EGFR Metastatic Lung Cancer

“Iressa (gefitinib) has been approved by the U.S. Food and Drug Administration to treat patients with metastatic non-small-cell lung cancer (NSCLC) with a specific genetic mutation (epidermal growth factor receptor [EGFR]). A just-approved companion diagnostic test can identify patients who could benefit from this new use.

“Iressa is a kinase inhibitor, a class of drugs designed to block proteins that spur development of cancer cells. The therascreen EGFR RGQ PCR Kit is a newly approved diagnostic that can help doctors detect patients with the genetic mutation who are candidates for treatment with Iressa.

“Iressa was evaluated for this use in clinical trials involving 106 people with previously untreated EGFR mutation-positive metastatic NSCLC. Tumors shrank in about 50 percent of people treated with Iressa 250 mg once daily. This effect lasted an average of six months, the FDA said. Severe side effects of Iressa may include interstitial lung disease, liver damage, gastrointestinal perforation, severe diarrhea, and ocular disorders. More common side effects are diarrhea and skin reactions.”


Cuba Has a Lung Cancer Vaccine—And America Wants It

“Cuba has for several years had a promising therapeutic vaccine against lung cancer. The 55-year trade embargo led by the US made sure that Cuba was mostly where it stayed. Until—maybe—now.

“The Obama administration has, of course, been trying to normalize relations with the island nation. And last month, during New York Gov. Andrew Cuomo’s visit to Havana, Roswell Park Cancer Institute finalized an agreement with Cuba’s Center for Molecular Immunology to develop a lung cancer vaccine and begin clinical trials in the US. Essentially, US researchers will bring the Cimavax vaccine stateside and get on track for approval by the Food and Drug Administration.

“ ‘The chance to evaluate a vaccine like this is a very exciting prospect,’ says Candace Johnson, CEO of Roswell Park. She’s excited, most likely, because research on the vaccine so far shows that it has low toxicity, and it’s relatively cheap to produce and store. The Center for Molecular Immunology will give Roswell Park all of the documentation (how it’s produced, toxicity data, results from past trials) for an FDA drug application; Johnson says she hopes to get approval for testing Cimavax within six to eight months, and to start clinical trials in a year.”


FDA Panel Supports Approval of T-VEC

“Two days after the U.S. Food & Drug Administration signaled it might quash Amgen Inc.’s attempt to usher in a whole new class of virus-based cancer drugs, an advisory panel for the agency voted ‘yes’ on the question of whether the company’s experimental melanoma treatment, talimogene laherparepvec (T-VEC), has a favorable enough risk-benefit profile for approval. T-VEC is made from a modified herpes bug and would likely be the first among a number of virus-based cancer treatments in the pharma pipeline to be approved. Of the 23 members on the panel, all but one voted in favor of approval. The FDA doesn’t have to follow the direction of its advisory panels but it usually does.

“T-VEC, which is injected directly into melanoma tumors, 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 hope is that the combination of the virus with GM-CSF will not only speed up the drug’s cancer-killing effect, but also stimulate the immune system to continue killing melanoma cells—even those that have traveled away from the treated tumor.

“In 2011, when T-VEC was in the early stages of development, Amgen paid a remarkable $425 million plus $575 million in milestone commitments to acquire its inventor, Biovex. At the time, there were a few hopeful signs: The FDA had granted the drug “fast-track” designation, indicating the potential to speed up its path to market. Then the agency added orphan designation for T-VEC, which could offer benefits such as tax credits and a period of market exclusivity if the drug is approved.”