Cancer Drugs Approved by the FDA in Early 2016

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The following drugs received either a new indication or were newly approved by the FDA in the first quarter of 2016; these new approvals extend the therapeutic options available for patients with different types of oncologic and hematologic cancers.

Venclexta First BCL-2 Targeted Drug Approved for Patients with CLL plus 17p Deletion

On April 11, 2016, the FDA approved venetoclax (Venclexta; AbbVie/Genentech) for the treatment of patients with chronic lymphocytic leukemia (CLL) associated with chromosome 17p deletion who have received at least 1 previous therapy.

Venetoclax is the first FDA-approved drug that targets the B-cell lymphoma (BCL)-2 protein. The overexpression of BCL-2 in CLL cells mediates tumor-cell survival and has been linked to resistance to chemotherapy. Venetoclax helps restore tumor-cell apoptosis by binding directly to the BCL-2 protein and triggering the activation of caspases.

Patients with the 17p deletion lack a portion of the chromosome that helps to suppress cancer cell growth; this abnormality occurs in 10% of patients with untreated CLL and in 20% of patients with relapsed CLL.

“These patients now have a new, targeted therapy that inhibits a protein involved in keeping tumor cells alive,” said Richard Pazdur, FDA Director of the Office of Hematology and Oncology Products. “For certain patients with CLL who have not had favorable outcomes with other therapies, Venclexta may provide a new option for their specific condition.”

Overall, 80% of patients who received venetoclax experienced a complete or partial remission of their cancer. The median time to first response was 0.8 months, and the duration of response ranged from 2.9 months to >19 months.

The most common adverse reactions associated with venetoclax therapy included neutropenia, diarrhea, nausea, anemia, upper respiratory tract infection, thrombocytopenia, and fatigue.

Venetoclax is indicated for daily use in patients with CLL whose 17p deletion status was confirmed by a companion diagnostic Vysis CLL FISH Probe Kit (manufactured by Abbott).

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Xalkori Receives New Indication for Metastatic NSCLC with the ROS-1 Mutation

On March 11, 2016, the FDA approved a new indication for crizotinib (Xalkori; Pfizer) for the treatment of patients with metastatic non–small-cell lung cancer (NSCLC) that is associated with the ROS-1 genetic mutation. Crizotinib is the first and only FDA-approved treatment for patients with ROS-1–positive NSCLC. Oral crizotinib blocks the activity of the ROS-1 protein in tumors that harbor the ROS-1 mutation. This effect on ROS-1 may prevent NSCLC from growing and spreading.

The ROS-1 mutation has been identified in different types of cancers. Approximately 1% of patients with NSCLC have this mutation.

Overall, 66% of the patients had a complete or partial tumor reduction lasting a median of 18.3 months with crizotinib. The safety profile of crizotinib in this patient population was consistent with the safety profile of crizotinib that had been reported in 1669 patients with ALK-positive metastatic NSCLC (for which it was originally approved).

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Imbruvica Now Approved for First-Line Treatment of Patients with CLL

On March 4, 2016, the FDA approved a new indication for ibrutinib (Imbruvica; Pharmacyclics) as a first-line treatment for patients with CLL. This approval is based on the results from RESONATE-2, a randomized, multicenter, open-label phase 3 clinical trial, which compared ibrutinib with chlorambucil in 269 treatment-naïve patients with CLL or with small lymphocytic lymphoma.

Ibrutinib significantly prolonged progression-free survival (PFS) and reduced the risk for disease progression or death by 84% versus chlor­ambucil (hazard ratio, 0.161; 95% confidence interval [CI], 0.091-0.283; P <.001). The median PFS was not reached with ibrutinib and was 18.9 months with chlorambucil (95% CI, 14.1-22.0).

With this new approval, ibrutinib is now approved for all patients with CLL, regardless of their treatment history. Ibrutinib was also previously approved for the treatment of patients with mantle-cell lymphoma and is the first and only drug approved by the FDA for patients with Waldenström’s macroglobulinemia.

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Gazyva New Option for Relapsed/Refractory Follicular Lymphoma

On February 26, 2016, the FDA approved a new indication for obinutuzumab (Gazyva Injection; Genentech) for use in combination with bendamustine, followed by obinutuz­umab monotherapy, for the treatment of patients with follicular lymphoma whose disease relapsed after, or is refractory to, a regimen containing rituximab. Obinutuzumab was previously approved in combination with chlorambucil for patients with treatment-naive CLL.

The approval was based on the phase 3 GADOLIN clinical trial. The median PFS was 13.8 months in the bendamustine arm and was not yet reached in the combination arm of bendamustine plus obinutuzumab followed by obinutuzumab alone (hazard ratio, 0.48; 95% CI, 0.34-0.68; P <.001). The median duration of response was not reached with the obinutuzumab regimen compared with 11.6 months with bendamustine alone.

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Afinitor Indicated for Unresectable Advanced/Metastatic Neuroendocrine Tumors

On February 26, 2016, the FDA approved a new indication for everolimus (Afinitor; Novartis) for the treatment of adults with progressive, well-differentiated, nonfunctional neuroendocrine tumors (NETs) of gastrointestinal or lung origin with unresectable, locally advanced or metastatic disease.

The FDA approval was based on a multicenter, placebo-controlled, randomized clinical trial of 302 patients with unresectable, locally advanced or metastatic, well-differentiated, nonfunctional (ie, no current or a history of carcinoid symptoms) NETs of gastrointestinal or lung origin. The patients were randomized (in a 2:1 ratio) to oral everolimus 10 mg once daily plus best supportive care or to placebo plus best supportive care.

The median PFS was 11 months in the everolimus arm versus 3.9 months in the placebo arm (hazard ratio, 0.48; 95% CI, 0.35-0.67;P <.001). The overall response rate was 2% in the everolimus arm versus 1% in the placebo arm. At a planned interim analysis, no significant difference in overall survival was found between the 2 arms.

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Ibrance Receives an Expanded Indication in Breast Cancer

On February 25, 2016, the FDA approved an expanded indication for the first-in-class cyclin-dependent kinase (CDK)4/CDK6 inhibitor palbociclib (Ibrance; Pfizer) for use in combination with fulvestrant (Faslodex) in women with hormone receptor (HR)-positive, HER2-negative advanced or metastatic breast cancer that has progressed after endocrine therapy. The drug was first approved by the FDA in February 2015 for the treatment of women with estrogen receptor–positive, HER2-negative metastatic breast cancer who had not received endocrine therapy. The expanded indication was based on the results of the PALOMA-3 clinical trial, which was stopped early after an interim analysis showed superior clinical benefit with the use of palbociclib in combination with fulvestrant versus fulvestrant plus placebo.

“There currently is no cure for metastatic breast cancer, so ongoing treatment is usually needed to control the spread of the disease,” said Marisa Weiss, MD, Founder and Chief Medical Officer of Breastcan, in a press release. “That’s why the availability of a first-of-its-kind treatment option like Ibrance for women dealing with HR-positive, HER2-negative metastatic disease represents a very important advance.”

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Kyprolis Gets New Indication for Relapsed/Refractory Multiple Myeloma

On January 21, 2016, the FDA approved a new indication for carfilz­omib (Kyprolis; Onyx Pharmaceuticals, a subsidiary of Amgen) for injection, for use in combination with dexamethasone or with lenalidomide plus dexamethasone in the treatment of patients with relapsed or refractory multiple myeloma who have received 1 to 3 previous therapies. The FDA also approved carfilzomib as a single agent for the treatment of patients with relapsed or refractory multiple myeloma who have received at least 1 previous therapy.

“Multiple myeloma remains an incurable disease, where relapse inevitably occurs and over time patients become resistant to treatments,” said Ruben Niesvizky, MD, Director of the Multiple Myeloma Center at Weill Cornell Medicine. This FDA approval is important because it provides physicians with flexible options for Kyprolis use in helping to manage this challenging disease.”

In July 2015, the FDA approved another expanded indication for carfilzomib for use in combination with lenalidomide and dexamethasone for the treatment of patients with multiple myeloma who had received 1 to 3 previous therapies.

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