FDA grants breakthrough therapy status to two cancer therapies
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The FDA granted breakthrough therapy status to two cancer therapies.
One designation applies to use of mobocertinib (TAK-788, Takeda) by patients with metastatic non-small cell lung cancer who have EGFR exon 20 insertion mutations and whose disease progressed on or after platinum-based chemotherapy.
There are no FDA-approved therapies for this patient population.
Mobocertinib is a small-molecule tyrosine kinase inhibitor designed to selectively target EGFR and human EGFR 2 (HER2) exon 20 insertion mutations.
The FDA based the breakthrough therapy designation on results of a phase 1/phase 2 study that evaluated the efficacy and safety of mobocertinib for patients with locally advanced or metastatic NSCLC whose tumors harbor EGFR exon 20 insertion mutations and who previously received systemic chemotherapy.
NSCLC accounts for about 85% of lung cancer cases worldwide; however, only 1% to 2% of patients have EGFR exon 20 insertion mutations.
“Although most EGFR mutations can be targeted by currently available TKIs, people with exon 20 insertion mutations often suffer and feel forgotten [because] available EGFR inhibitors don’t work well in their cancer,” Jill Feldman, co-founder of EGFR Resisters, a patient-driven advocacy group, said in a press release. “We are excited by the potential this treatment has to extend the lives of people who have had no approved treatment options to target their disease.”
The FDA also granted breakthrough therapy designation to avelumab (Bavencio; EMD Serono, Pfizer) for first-line maintenance treatment of locally advanced or metastatic urothelial carcinoma.
The agency will review the supplemental biologics license application through its Real-Time Oncology Review pilot program.
The FDA based the breakthrough therapy designation on results of an interim analysis of the randomized phase 3 JAVELIN Bladder 100 trial, which included 700 patients with previously untreated locally advanced or metastatic urothelial carcinoma whose disease did not progress on induction chemotherapy.
Researchers randomly assigned patients to best supportive care with or without first-line maintenance therapy with avelumab, a human anti-PD-L1 antibody.
OS served as the primary endpoint. Secondary endpoints included PFS, antitumor activity, safety, pharmacokinetics, immunogenicity, predictive biomarkers and patient-reported outcomes.
Results of an interim analysis showed a statistically significant improvement in OS with avelumab among all patients, as well as those with PD-L1-positive tumors. Avelumab exhibited a safety profile consistent with prior studies.
The FDA approved avelumab in 2017 for the treatment of patients with locally advanced or metastatic urothelial carcinoma whose disease progressed during or after platinum-containing chemotherapy, or who experienced disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.