FDA denies poziotinib approval for advanced non-small cell lung cancer
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The FDA issued a complete response letter to Spectrum Pharmaceuticals indicating it could not approve the company’s new drug application that sought approval of poziotinib as treatment for certain patients with lung cancer.
The agency determined additional data — including a randomized controlled study — would be necessary.
Following the FDA’s action, Spectrum Pharmaceuticals announced its intention to immediately de-prioritize its poziotinib program activities and reduce its research and development workforce by 75%.
Poziotinib is an oral, irreversible tyrosine kinase inhibitor.
The FDA granted fast track designation to the agent last year for use by previously treated patients with NSCLC who harbor HER2 exon 20 mutations
Most of the data that supported the fast track designation came from cohort 2 of a single-arm phase 2 study.
The cohort included 90 patients with NSCLC and HER2 exon 20 insertion mutations who progressed after standard platinum-based chemotherapy with or without a checkpoint inhibitor. Study participants received poziotinib at a starting dose of 16 mg once daily.
Researchers reported an objective response rate of 27.8% (95% CI, 18.9-38.2), a disease control rate of 70% (95% CI, 59.4-79.2), median duration of response of 5.1 months (95% CI, 4.2-5.5) and median PFS of 5.5 months (95% CI, 3.9-5.8).
Earlier this fall, however, the FDA Oncologic Drugs Advisory Committee (ODAC) voted 9-4 against approval of poziotinib for this indication, concluding safety results from the trial showed the benefits did not outweigh the risks.
The majority (85.6%) of trial participants experienced grade 3 adverse events, and 11.1% experienced grade 4 adverse events. The most common grade 3 and grade 4 events included rash (30%), diarrhea (26.7%) and stomatitis (23.3%).
Forty percent of patients experienced serious adverse events, and 10 patients experienced fatal adverse events.
More than three-quarters of patients (77%) required a dose reduction and 87% required a dose interruption.
The FDA panel also concluded that poziotinib did not improve efficacy compared with current therapies.
“[Although] we are not surprised by the [complete response letter] given the ODAC recommendation in September, we are disappointed. After multiple interactions with the FDA since [the ODAC meeting], and following careful consideration, we have made the strategic decision to immediately de-prioritize the poziotinib program,” Tom Riga, president and CEO of Spectrum Pharmaceuticals, said in a press release. “We continue to believe that poziotinib could present a meaningful treatment option for patients with this rare form of lung cancer, for whom other therapies have failed.”
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