Drug Approvals Highlight Oncology News Roundup

Recent developments in cancer treatment and research.

Cabozantinib Nabs RCC Breakthrough Designation

Exelixis recently announced that cabozantinib received a breakthrough therapy designation from the FDA for the treatment of patients with advanced renal cell carcinoma following one prior therapy. The designation is based on findings from the open-label, phase III METEOR study, in which cabozantinib reduced the risk of progression or death by 42% versus everolimus (HR, 0.58; P <.0001).

Overall survival with cabozantinib was improved by 33% versus everolimus, with data continuing to mature (HR, 0.67; P = .005). Statistical significance for the OS endpoint was represented by a P value of .0019. In addition to efficacy data, the company noted that serious adverse events were balanced between the cabozantinib and everolimus arms in the phase III study. In both arms, treatment discontinuation due to an AE was approximately 10%.

Exelixis reported that complete results from METEOR are scheduled for presentation as a late-breaking abstract on September 26 at the 2015 European Cancer Congress. These data will likely be submitted to the FDA for regulatory approval. See more at: http://www.specialtypharmacytimes.com/news/fda-grants-breakthrough-designation-to-renal-cell-carcinoma-drug

Eltrombopag Approved for Pediatric Chronic ITP Patients

The FDA recently approved eltrombopag as a treatment for pediatric patients with chronic immune thrombocytopenic purpura following a lack of response to other therapies or splenectomy. The decision was based on improvements in platelet counts seen with eltrombopag in two trials.

In the first trial, the phase II PETIT study, 62% of patients treated with eltrombopag experienced an improvement in platelet counts compared with 32% with placebo. In the second study, the phase III PETIT2 trial, platelet counts were improved in 40% of patients receiving eltrombopag versus just 3% with placebo.

For those aged 12—17, the response was 39% with eltrombopag versus 10% with placebo. In the 6–11 age bracket, platelet counts were improved in 42% of patients treated with eltrombopag versus 0% with placebo. An oral suspension formulation of eltrombopag was used for those aged 5–1, which was approved with the new indication. With formulation, the response rate was 36% versus 0% for those aged 5–1.

The treatment is now indicated for those age 1—17 and adults with chronic ITP. See more at: http://www.specialtypharmacytimes.com/news/fda-expands-use-for-rare-blood-disorder-drug

Frontline Pembrolizumab Accelerated, While Second Application Is Delayed

The FDA recently granted a priority review to pembrolizumab as a frontline treatment for patients with advanced melanoma, according to Merck, the manufacturer of the anti—PD-1 therapy. A final decision is scheduled from the FDA by December 19, 2015.

In a separate action, the FDA delayed its decision date on an application for pembrolizumab in ipilimumab-refractory advanced melanoma to December 24, 2015, based on the need to review additional data submitted by Merck from the KEYNOTE-002 study. In this study, at the recommended 2-mg/kg dose, pembrolizumab demonstrated a PFS of 34% at 6 months and 24% at 9 months. In the chemotherapy arm, the PFS was 16% and 8%, at the 6- and 9-month analyses, respectively.

In the frontline setting data that received a priority review, at a 6-month assessment the PFS was 47% and 46% with pembrolizumab every 2- and 3-weeks, respectively. For ipilimumab, the PFS rate was 27%. At 9 months, PFS rates were 40% and 42% compared with 16%, in the 2-week, 3-week, and ipilimumab arms, respectively.

In the 3-week pembrolizumab arm, the 1-year OS rate was 68% compared with 58% for ipilimumab (HR, 0.69). In the 2-week arm, the 1-year OS rate was 74% (HR, 0.63). See more at: http://www.onclive.com/web-exclusives/frontline-pembrolizumab-receives-priority-review-in-melanoma

Sonidegib Approved for Basal Cell Carcinoma in Europe

Last week, the European Commission approved the hedgehog inhibitor sonidegib for the treatment of patients with locally advanced basal cell carcinoma who are not amenable to curative surgery or radiation therapy. The decision was based on the phase II BOLT study in which sonidegib had an objective response rate of 56% and a median progression-free survival of 22 months among patients with laBCC who received the hedgehog inhibitor at a 200-mg dose.

The FDA approved the treatment on July 27, 2015. The 56% an independent panel in a cohort of 66 patients observed ORR on which the EC based its approval with laBCC treated at the 200-mg dose who were followed for at least 18 months. The ORR comprised three complete responses (5%) and 34 partial responses (52%).

The CR rate was 23% in a prespecified sensitivity analysis, which used a varying definition of CR—PR based on MRI and/or photography and no evidence of tumor on biopsy of the residual lesion. The median duration of response has not been reached. ORR per investigator review was 71% and included 6 CRs (9%) and 41 PRs (62%).

PFS rates per independent and investigator review were 22 and 19 months, respectively. Treatment with the higher dose did not significantly improve clinical outcomes. The ORR was 44% among 128 patients with laBCC who received 800 mg of sonidegib. See more at: http://www.onclive.com/web-exclusives/sonidegib-approved-in-europe-for-basal-cell-carcinoma

New Patient Video Portal Launches

A new video platform designed specifically for patients with cancer, labeled CURE Connections, was launched last week. The platform host videos on being diagnosed with cancer, talking about the diagnosis, myths about cancer and treatment, nutrition in cancer care, financial assistance, chemotherapy-induced nausea and vomiting, and becoming a caregiver for a loved one with cancer.

Dr. Philippa J. Cheetham, a board-certified Urological Surgeon, practicing in New York City, hosts the series. According to Dr. Cheetham, CURE Connections can help educate patients and physicians on the more personal factors of cancer, with the goal of standardizing best practices nationwide.

The video portal now contains information on breast, colorectal, pancreatic, and prostate cancers. Additionally, there is a series on cooking and nutrition with oncology dieticians that highlights easy to prepare nutritional recipes that are recommended for cancer patients to help manage their health before, during, and after treatment. CURE connection is part of CURE Media Group, which also hosts CURE and Heal Magazines and CUREtoday.com.

The web site was acquired by Michael J Hennessy Associates, Inc. approximately 1 year ago. Here's more on the video website http://cureconnections.curetoday.com