Takeda Voluntarily Withdraws Mobocertinib from U.S. Market Due to Failed Confirmatory Trial

　　【Pharmaceutical Network Product InformationNon-small cell lung cancer (NSCLC) is the most common histological subtype of lung cancer. Among NSCLC cases, mutations involving EGFR exon 20 insertions (EGFR exon20ins) are associated with poor prognosis, and there is a lack of effective targeted treatment options in clinical practice. Currently, only three drugs targeting EGFR exon20ins mutations in NSCLC have been approved globally: Mobocertinib from Takeda, Amivantamab from Johnson & Johnson, and Dizgel's Sunvozertinib.
 

However, recently, Takeda Pharmaceutical Company Limited announced that, after consultation with the U.S. Food and Drug Administration (FDA), it will initiate the voluntary withdrawal of mobocertinib succinate (mobocertinib, trade name: Exkivity, hereinafter referred to as "mobocertinib") from the U.S. market.
 

Takeda stated that the reason for the withdrawal is that the Phase III EXCLAIM-2 confirmatory study of mobocertinib did not meet its primary endpoint. This is a Phase III, multicenter, open-label trial designed to evaluate the safety and efficacy of mobocertinib monotherapy compared to platinum-based chemotherapy as a first-line treatment for patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring epidermal growth factor receptor (EGFR) exon 20 insertion mutations. However, due to the failure to meet the confirmatory study data requirements set by the FDA and other regulatory agencies for the accelerated/conditional approval of mobocertinib, Takeda, after consultation with the FDA, decided to initiate the voluntary withdrawal of mobocertinib in the United States.
 

Data shows that Mobocertinib received FDA accelerated approval on September 15, 2021, for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) who have EGFR exon 20 insertion mutations (EGFR exon20ins) and whose disease has progressed during or after platinum-based chemotherapy. In China, it was approved for marketing by the National Medical Products Administration in January 2023, with the same indications, providing a treatment option for relevant patients in China.
 

In addition to this indication, Takeda also attempted to focus its research on first-line treatment, but the results were not as expected. In the Q1 2023 earnings report disclosed by Takeda Pharmaceutical Company Limited, the relevant R&D progress of Mobocertinib was revealed, showing that Mobocertinib was terminated in the EXCLAIM-2 study due to ineffectiveness.
 

Moreover, the sales of Mobocertinib have not met expectations. Takeda had projected in its earnings report that Mobocertinib sales could reach between $300 million and $600 million. However, as of March this year, the drug's sales within a year amounted to only $26 million. In response, Takeda Pharmaceutical Company Limited stated that the reason for the above situation was that Mobocertinib and Johnson & Johnson's Amivantamab (Rybrevant) split the second-line market.
 

Nowadays, Takeda Pharmaceutical Company Limited has decided to voluntarily withdraw the approved indication of Mobocertinib in the United States, which means it will no longer be marketed in the U.S. However, Takeda Pharmaceutical Company Limited also stated that the company is actively communicating with regulatory agencies in other countries and regions where Mobocertinib has been approved, including China, and will follow the requirements of local laws and regulations to formulate subsequent plans.
 

As Takeda's drug voluntarily withdraws from the U.S. market, it means that there are only two drugs globally targeting EGFR exon20ins non-small cell lung cancer: Dizhe Medicine's Sunvozertinib and Johnson & Johnson's Amivantamab sharing the market. As of now, Amivantamab has not been approved in China, while Dizhe Medicine’s Sunvozertinib was approved in August this year. In addition, several pharmaceutical companies in China, such as Alis and Luye Pharma, are also actively laying out plans for drugs targeting EGFR exon20ins NSCLC treatment, attempting to gain a share of the market. The future landscape of this drug market in China is worth keeping an eye on.
 

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