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FDA Actions & Updates
The latest approvals, designations, and new indications from the U.S. Food and Drug Administration for oncology drugs.
Wednesday, January 11, 2017

The FDA has accepted the supplemental Biologics License Application (sBLA) and granted Priority Review for atezolizumab (Tecentriq) for the treatment of people with locally advanced or metastatic urothelial carcinoma (mUC) who are ineligible for cisplatin chemotherapy, and are either previously untreated or have disease progression at least 12 months after receiving neoadjuvant or adjuvant chemotherapy.

Atezolizumab is a monoclonal antibody designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, atezolizumab may enable the activation of T cells; it may also affect normal cells.

This sBLA submission for atezolizumab is based on results from the phase II IMvigor210 study, and the FDA will make a decision on approval by April 30, 2017. A Priority Review designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the safety and effectiveness of the treatment, prevention, or diagnosis of a serious disease.

Atezolizumab is currently approved by the FDA to treat people with locally advanced or mUC who have disease progression during or following platinum-based chemotherapy or whose disease has worsened within 12 months of neoadjuvant or adjuvant platinum-based chemotherapy. Atezolizumab is approved under accelerated approval for this indication based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Atezolizumab is also approved for the treatment of people with metastatic non-small cell lung cancer who have disease progression during or following platinum-containing chemotherapy, and have progressed on an appropriate FDA-approved targeted therapy if their tumor has EGFR or ALK gene abnormalities. 

Monday, January 9, 2017

Priority review status has been granted by the FDA for the supplemental New Drug Application (sNDA) for regorafenib (Stivarga) tablets for the second-line systemic treatment of patients with hepatocellular carcinoma (HCC) in the U.S.

HCC is the most common form of liver cancer, a disease that affects nearly 40,000 patients in the United States and is the second deadliest form of cancer worldwide.

The FDA grants priority review to medicines that, if approved, would provide significant improvements in the safety or effectiveness of the treatment for serious conditions. Under the Prescription Drug User Fee Act (PDUFA), the FDA aims to complete its review within 6 months (compared to 10 months under standard review).

The sNDA is based on data from the international, multicenter, placebo-controlled phase III RESORCE trial which investigated regorafenib in patients with HCC whose disease had progressed during treatment with Nexavar.

Regorafenib is currently approved for the treatment of patients with metastatic colorectal cancer (mCRC) who have previously been treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an anti-VEGF therapy, and, if RAS wild type, an anti-EGFR therapy, as well as the treatment of patients with locally advanced, unresectable, or metastatic gastrointestinal stromal tumor (GIST), who were previously treated with imatinib mesylate and sutinib maleate.

Monday, January 2, 2017

The FDA has granted Orphan Drug Designation to YS-ON-001 for the treatment of hepatocellular carcinoma. YS-ON-001 is a promising biological product with immunomodulating effects, including induction of anti-tumor cytokines, activation of NK cells, regulation of macrophage polarization, and suppression of regulatory T cells.

YS-ON-001demonstrates efficacy in animal studies against multiple solid tumors, such as breast, lung, liver, and other cancers. Toxicology studies in animals also demonstrated good safety of the product. A multi-component complex with broad immunomodulating properties, YS-ON-001 promotes Th1-biased immunity; induces the activation and proliferation of dendritic cell, B, and natural killer cells; and promotes macrophage M1 polarization and downregulating regulatory T cells.

Orphan Drug Designation is granted to novel drugs and biologics that are defined as those intended for the safe and effective treatment, diagnosis, or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S.

Monday, January 2, 2017

Napabucasin has received Orphan Drug Designation from the FDA for the treatment of pancreatic cancer. This is the second Orphan Drug Designation for napabucasin, an orally administered agent designed to inhibit cancer stemness pathways by targeting STAT3; the first designation was for gastric cancer including gastroesophageal junction (GEJ) cancer.

Phase Ib data for napabucasin in metastatic pancreatic cancer (NCT02231723) were previously presented at the ASCO 2016 annual meeting (Abstract 284). These data showed napabucasin may be combined with gemcitabine and nab-PTX and showed signs of anti-tumor activity in patients with metastatic pancreatic cancer.

Of the 37 patients enrolled in the study, 57 percent of patients (17 of 30 evaluable patients) had prolonged disease control (≥24 weeks). Common adverse events identified in this clinical trial were grade 1 diarrhea, nausea, fatigue, and neuropathy, which were reversible and manageable with symptom medications.

Napabucasin is currently being investigated in three phase III studies in advanced gastric and GEJ (NCT02178956), colorectal (NCT02753127), and lung cancer (NCT02826161). It is also being investigated in earlier phases in multiple solid and hematologic malignancies, including tumors of the liver, pancreas, and brain.


Monday, January 2, 2017

​​The FDA has granted Breakthrough Therapy Designation to brentuximab vedotin (Adcetris) for the treatment of patients with CD30-expressing mycosis fungoides (MF) and primary cutaneous anaplastic large cell lymphoma (pcALCL) who require systemic therapy and have received one prior systemic therapy.

MF and pcALCL are the most common subtypes of cutaneous T-cell lymphoma (CTCL), accounting for more than 75 percent of the disease. Brentuximab vedotin is an antibody-drug conjugate directed to CD30, which is expressed on skin lesions in approximately 50 percent of patients with CTCL. Brentuximab vedotin is currently not approved for the treatment of CTCL.

This designation was based on data from the phase III ALCANZA clinical trial, which evaluated brentuximab vedotin in CD30-expressing CTCL and met its primary endpoint, demonstrating a highly statistically significant improvement in the rate of objective response lasting at least 4 months. This randomized trial, which received a Special Protocol Assessment agreement from the FDA and scientific advice from the European Medicines Agency, compared the use of single-agent brentuximab vedotin to a control arm of investigator's choice of standard therapies, methotrexate or bexarotene, in 131 patients with CD30-expressing CTCL who received prior systemic or radiation therapy.

About the Author

Sarah DiGiulio