Recent Drugs and Diagnostics Approved by the FDA

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This section provides updates on recently FDA-approved new diagnostic tests, as well as new drugs or new indications. These approvals extend the diagnostic and therapeutic options available for patients with different types of oncologic and hematologic conditions.

First Liquid Biopsy Approved for Diagnosing EGFR Mutations in NSCLC

On June 1, 2016, the FDA approved the cobas EGFR Mutation Test v2 (Roche Molecular Systems), the first blood-based companion diagnostic for erlotinib (Tarceva) in patients with non–small-cell lung cancer (NSCLC).

Erlotinib received its latest indication in 2013 for the first-line treatment of patients with metastatic NSCLC whose tumors have EGFR exon 19 deletions or exon 21 (L858R) substitution mutations as detected by an FDA-approved test. At that time, the cobas EGFR Mutation Test v1 was the approved companion diagnostic test for erlotinib.

Unlike the cobas EGFR Mutation Test v1, which uses patients’ tumor specimens to identify EGFR mutations, the cobas EGFR Mutation Test v2 is a liquid biopsy, and will therefore help physicians to noninvasively identify patients who may benefit from treatment with erlotinib. However, a tumor biopsy should be performed to confirm the absence or presence of EGFR mutations if they are not detected in the blood. This liquid biopsy will be particularly valuable to patients who may be physically unfit or unable to provide a tumor specimen.

“Approvals of liquid biopsy tests make it possible to deliver highly individualized health care for patients. Liquid biopsies also have the potential to allow physicians to identify patients whose tumors have specific mutations in the least invasive way possible,” said Alberto Gutierrez, PhD, Director of the FDA’s Office of In Vitro Diagnostics and Radiological Health.

The cobas EGFR Mutation Test v2 demonstrated its efficacy when it identified EGFR mutation status in patients whose status was previously determined using the cobas EGFR Mutation Test v1.

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FDA Approves Axumin Imaging Agent for Recurrent Prostate Cancer

On May 27, 2016, the FDA approved fluciclovine F18 (Axumin; Blue Earth Diagnostics) injection, a radioactive imaging agent used to detect recurrent prostate cancer.

Fluciclovine F18 injection is indicated for patients with suspected prostate cancer recurrence based on elevated prostate-specific antigen (PSA) levels, and is used with positron emission tomography (PET) imaging.

“Imaging tests are not able to determine the location of the recurrent prostate cancer when the PSA is at very low levels,” said Libero Marzella, MD, PhD, Director of the FDA’s Center for Drug Evaluation and Research Division of Medical Imaging Products. “Axumin is shown to provide another accurate imaging approach for these patients.”

The FDA approval was based on 2 studies evaluating the safety and efficacy of fluciclovine F18 injection in men with suspected recurrence of prostate cancer based on rising PSA levels after radical prostatectomy and/or radiotherapy. The first study compared 105 scans using fluciclovine F18 injection with histopathology obtained by prostate biopsy and by biopsies of suspicious lesions in patients with suspected prostate cancer recurrence. The Axumin images were first read by on-site readers and then by 3 blinded independent readers.

Overall, the results of the independent read were consistent with one another, and confirmed the results of the on-site reads. The detection rate for patients with a PSA value ≤1.78 ng/mL was 15/25, of which 11 were histologically confirmed as positive; there were 4 false positives and 1 false negative. In the remaining 3 PSA quartiles, the detection rate was 71/74, of which 58 were histologically confirmed; there were 13 false positives and no false negatives.

The second study compared the results from 96 scans using fluciclovine F18 injection with C11 choline scans, an approved PET scan imaging test, in men with a median PSA value of 1.44 ng/mL. The agreement values between fluciclovine F18 injection and C11 choline reads ranged from 61% to 77%.

The most frequently reported adverse events were injection-site pain, redness, and a metallic taste in the mouth.

Because fluciclovine F18 is a radioactive drug, the FDA recommends appropriate safety measures to limit exposure to patients and providers during administration.

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Tecentriq Approved for Metastatic Bladder Cancer

On May 18, 2016, the FDA granted accelerated approval to atezolizumab (Tecentriq; Genentech) for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or after platinum-containing chemotherapy, or for those who have had disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

Atezolizumab received a breakthrough therapy designation in May 2014 for metastatic bladder cancer that expresses PD ligand 1 (PD-L1); it was approved for the treatment of locally advanced or metastatic urothelial carcinoma using the FDA’s priority review process.

“Tecentriq provides these patients with a new therapy targeting the PD-L1 pathway,” said Richard Pazdur, MD, Director of the FDA’s Office of Hematology and Oncology Products. “Products that block PD-1/PD-L1 interactions are part of an evolving story about the relationship between the body’s immune system and its interaction with cancer cells.”

The accelerated approval of atezolizumab was based on the results of the IMvigor 210 study, an open-label, multicenter, single-arm, phase 2 study of 310 patients with locally advanced or metastatic urothelial carcinoma who received 1200 mg of atezolizumab intravenously. The study’s primary end point was objective response rate (ORR), and the secondary end point was the duration of response.

Atezolizumab conferred at least partial shrinkage of the tumor in 9.4% of patients. The ORR in the study was 14.8%, and the median duration of response ranged from 2.1 months to 13.8 months. The ORRs were 9.5% in patients with PD-L1 expression of <5% and 26% in patients with PD-L1 expression ≥5%, suggesting that the level of PD-L1 expression may help identify the patients who are more likely to respond to therapy with atezolizumab; the Ventana PD-L1 assay was approved by the FDA on May 18, 2016, to detect PD-L1 levels and to help clinicians identify the patients who would most benefit from treatment with atezolizumab.

The most common (≥20%) adverse events (all grades) associated with atezolizumab included fatigue (52%), decreased appetite (26%), nausea (25%), urinary tract infection (22%), pyrexia (21%), and constipation (21%). The most common (≥2%) grade 3 or 4 adverse events included urinary tract infection (9%), fatigue (6%), abdominal pain (4%), and dyspnea (4%).

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Afatinib Receives New Indication for Advanced Squamous-Cell NSCLC

On April 15, 2016, the FDA approved afatinib (Gilotrif; Boehringer Ingelheim) tablets for the treatment of patients with advanced squamous-cell carcinoma of the lung that has progressed after platinum-containing chemotherapy treatment.

Afatinib was already approved for the first-line treatment of patients with certain EGFR mutation–positive NSCLC.

Advanced squamous-cell carcinoma of the lung has a median overall survival of approximately 1 year after diagnosis and is characterized by a poor prognosis.

The approval was based on the LUX-Lung 8 head-to-head clinical trial comparing afatinib with erlotinib (Tarceva) in patients with squamous-cell carcinoma of the lung whose tumors progressed after first-line chemotherapy. Compared with erlotinib, afatinib demonstrated an 18% reduction in the risk for cancer progression and a 19% reduction in the risk for death. There was also a significant improvement in the disease control rate with afatinib versus with erlotinib (51% vs 40%, respectively; P = .002).

The most common (in ≥20% of patients) adverse events observed with afatinib were diarrhea (75%), rash or acne (70%), stomatitis (30%), decreased appetite (25%), and nausea (21%).

The LUX-Lung 8 trial is part of the LUX-Lung program that includes 8 clinical studies with a total of >3760 patients.

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