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FDA Approves Takeda's Exkivity as First Oral Therapy for EGFR Exon20 NSCLC

  • Takeda's Exkivity (mobocertinib) receives FDA approval as the first oral therapy specifically designed for NSCLC patients with EGFR exon20 insertion mutations after chemotherapy.

  • Clinical trials demonstrated Exkivity's efficacy with 28% tumor shrinkage rate and median progression-free survival of seven months in treated patients.

  • The approval strengthens Takeda's Wave1 pipeline portfolio, with the drug projected to reach $600 million in peak sales pending potential first-line treatment approval.

Takeda Pharmaceutical has secured FDA approval for Exkivity (mobocertinib), marking a significant advancement in the treatment of non-small-cell lung cancer (NSCLC) with EGFR exon20 insertion mutations. The novel tyrosine kinase inhibitor (TKI) is approved for patients with locally advanced or metastatic disease who have progressed on platinum-based chemotherapy.

Breakthrough in Targeted Therapy

The approval represents a milestone in precision medicine, as Exkivity becomes the first oral therapy specifically designed for this distinct genetic subset of lung cancer patients. While Johnson & Johnson's Rybrevant (amivantamab) received FDA approval in May for the same indication, it requires intravenous administration, making Exkivity's oral formulation a potentially more convenient option for patients.

Clinical Efficacy and Patient Impact

The accelerated approval is supported by compelling phase 1/2 clinical data. Treatment with Exkivity demonstrated significant clinical benefit, achieving tumor shrinkage in 28% of patients and maintaining disease control for a median of seven months.
"EGFR Exon 20 insertion+ NSCLC is an underserved cancer that we have been unable to target effectively with traditional EGFR TKIs," noted Dr. Pasi Jänne from Dana Farber Cancer Institute, a principal investigator in the clinical trials.

Market Position and Treatment Landscape

While several EGFR-targeting TKIs are available for NSCLC, including AstraZeneca's Tagrisso (osimertinib) and Roche's Tarceva (erlotinib), none specifically address exon 20 insertions. This mutation affects approximately 10% of patients with EGFR mutations and 2% of all NSCLC cases. To facilitate patient identification, the FDA has simultaneously approved a companion diagnostic developed by Thermo Fisher Scientific.

Strategic Importance for Takeda

The approval strengthens Takeda's Wave1 pipeline, a strategic portfolio of therapies targeted for approval by fiscal year 2024 with projected combined peak sales of $10 billion. Exkivity alone is estimated to generate up to $600 million in peak sales, contingent upon potential approval for first-line treatment.
This success comes at a crucial time for Takeda, following recent setbacks including pevonedistat's disappointing phase 3 results in myelodysplastic syndromes and acute myeloid leukemia, and regulatory delays for other pipeline candidates. The company now turns its attention to maribavir, its candidate for cytomegalovirus infections in transplant patients, scheduled for FDA advisory committee review.
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Reference News

[1]
Takeda gets a win for its Wave1 pipeline, as Exkivity nabs FDA approval
pharmaphorum.com · May 13, 2025
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