FDA NEWS: February 5, 2021 to February 22, 2021

TOP - March 2021 Vol 14, No 2 - FDA Updates

This section provides a brief overview of new cancer drugs and indications approved by the FDA between February 5, 2021, and February 22, 2021.

FDA Approves Libtayo as Monotherapy for Advanced NSCLC

On February 22, 2021, the FDA approved cemiplimab-rwlc (Libtayo; Regeneron/Sanofi US) for the first-line treatment of patients with advanced non−small-cell lung cancer (NSCLC) whose tumors have high PD-L1 expression (tumor proportion score ≥50%), as determined by an FDA-approved test. Patients must also have metastatic or locally advanced tumors that are not candidates for surgical resection or definitive chemoradiation, and the tumors must not have EGFR, ALK, or ROS1 aberrations. The FDA granted cemiplimab priority review for this indication. Cemiplimab was previously approved for patients with basal-cell carcinoma (BCC) and cutaneous squamous-cell carcinoma.

This new indication was based on data from an analysis of 710 patients in the phase 3, open-label EMPOWER-Lung 1 clinical trial. Patients were randomized in a 1:1 ratio to receive cemiplimab or chemotherapy. A prespecified analysis was performed on a cohort of 563 patients with confirmed PD-L1–high tumors proven by the PD-L1 IHC 22C3 pharmDx kit. Results showed that these patients had a 43% lower risk for death compared with those treated with chemotherapy.

Safety was assessed in 355 patients in the cemiplimab treatment arm and 342 patients in the chemotherapy arm. In the cemiplimab arm, rash and cough occurred more frequently than in the chemotherapy arm (15% vs 6% and 11% vs 8%, respectively).

The most common serious adverse reactions were pneumonia and pneumonitis. Adverse reactions that resulted in permanent discontinuation occurred in 6% of patients treated with cemiplimab.

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Libtayo Approved for Advanced Basal-Cell Carcinoma

On February 9, 2021, the FDA approved cemiplimab-rwlc (Libtayo; Regeneron/Sanofi US) for patients with locally advanced BCC previously treated with a hedgehog pathway inhibitor (HHI) or for whom an HHI is not appropriate and granted accelerated approval to cemiplimab for patients with metastatic BCC (mBCC) previously treated with an HHI or for whom an HHI is not appropriate.

The FDA approved this indication for cemiplimab based on data from an ongoing multicenter, nonrandomized clinical trial in patients with advanced BCC who had progressed on HHI therapy, had not had an objective response after 9 months on HHI therapy, or were intolerant of previous HHI therapy. Patients in this trial received cemiplimab 350 mg every 3 weeks for up to 93 weeks until disease progression, unacceptable toxicity, or completion of planned treatment. The main efficacy outcome measures were confirmed objective response rate (ORR) and duration of response (DOR).

The confirmed ORR among 84 patients with locally advanced BCC was 29% with a median DOR not reached (range, 2.1-21.4+ months) with 79% of responders maintaining their response for ≥6 months. The confirmed ORR among 28 patients with mBCC was 21% with a median DOR not reached (range, 9-23.0+ months), with all responders maintaining their responses for ≥6 months.

Among 132 patients who were evaluable for safety, the most common (≥15%) adverse reactions were fatigue (49%), musculoskeletal pain (33%), diarrhea (25%), rash (22%), pruritus (20%), and upper respiratory tract infection (15%). Serious adverse reactions occurred in 32% of patients; those occurring in ≥2 patients included urinary tract infection, colitis, acute kidney injury, adrenal insufficiency, anemia, infected neoplasm, and somnolence. Adverse reactions resulting in permanent discontinuation occurred in 13% of patients, with the most common reactions (occurring in ≥2 patients) being colitis and general physical health deterioration.

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FDA Approves Breyanzi, Novel CAR T-Cell Therapy, for Advanced Large B-Cell Lymphoma

On February 5, 2021, the FDA approved lisocabtagene maraleucel (Breyanzi; Bristol Myers Squibb), a new chimeric antigen receptor (CAR) T-cell therapy, for the treatment of adult

patients with relapsed or refractory large B-cell lymphoma after ≥2 lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma), high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and grade 3B follicular lymphoma. The FDA granted this application priority review, breakthrough therapy, and orphan drug designation. Lisocabtagene maraleucel is not indicated for patients with primary central nervous system lymphoma.

The approval of lisocabtagene maraleucel was based on data from the multicenter, clinical trial, TRANSCEND NHL 001, which included 269 patients with relapsed or refractory large B-cell lymphoma. The efficacy analysis, which included 256 patients, showed an ORR of 73% and a complete response rate of 53% after 1 infusion.

According to the safety analysis, any grade cytokine release syndrome (CRS) occurred in 46% of patients, with 4% being grade ≥3. One patient had fatal CRS and 2 patients had ongoing CRS at the time of death.

Neurologic toxicities of any grade occurred in 35% of patients, with grade ≥3 neurologic toxicities occurring in 12% of patients. Three patients had fatal neurologic toxicity and 7 had ongoing neurologic toxicity at the time of death.

The most common (≥20%) nonlaboratory adverse reactions of any grade were fatigue, CRS, musculoskeletal pain, nausea, headache, encephalopathy, infections, decreased appetite, diarrhea, hypotension, tachycardia, dizziness, cough, constipation, abdominal pain, vomiting, and edema.

Lisocabtagene maraleucel will include a boxed warning that outlines the risks for CRS and neurologic toxicities associated with CAR T-cell therapy. The FDA also is requiring the manufacturer to conduct a postmarketing observational study of patients treated with lisocabtagene maraleucel to comply with its Risk Evaluation and Mitigation Strategy monitoring program.

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Ukoniq Approved for 2 Types of Lymphoma

On February 5, 2021, the FDA granted accelerated approval to umbralisib (Ukoniq; TG Therapeutics) for adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received ≥1 previous anti-CD20–based regimens, and adult patients with relapsed or refractory follicular lymphoma who have received ≥3 previous lines of systemic therapy.

The FDA previously granted orphan drug designation to umbralisib for both indications, and priority review for the MZL indication.

The FDA approval of umbralisib was based on data from 2 single-arm cohorts of an open-label, multicenter, clinical trial, UTX-TGR-205, which included 69 patients with MZL who had received ≥1 previous therapies and 117 patients with follicular lymphoma who had received ≥2 previous systemic therapies. The main efficacy outcomes measures included ORR and DOR.

In the MZL subgroup, the ORR was 49%, with 16% achieving complete response. Median DOR was not reached. In the follicular lymphoma subgroup, the ORR was 43%, with 3% achieving complete response. Median DOR was 11.1 months.

The most common (>15%) adverse reactions, including laboratory abnormalities, were increased creatinine, diarrhea-colitis, fatigue, nausea, neutropenia, transaminase elevation, musculoskeletal pain, anemia, thrombocytopenia, upper respiratory tract infection, vomiting, abdominal pain, decreased appetite, and rash. Serious adverse reactions occurred in 18% of patients, most often from diarrhea-colitis and infection.

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Last modified: July 22, 2021