- Japanese drugmaker Takeda said Wednesday it's received Food and Drug Administration approval for the first oral therapy targeting a specific gene mutation in non-small cell lung cancer patients.
- The drug, known as mobocertinib or Exkivity, is designed to treat patients who have a mutated protein that can cause rapid cell growth, spreading cancer. Patients with exon 20 insertion mutations in a gene called EGFR —Exkivity's target — represent about 2% of all people diagnosed with non-small cell lung cancer.
- The clearance comes four months after U.S. regulators gave the green light to Johnson & Johnson's Rybrevant, an infusion designed to treat the same subset of lung cancer patients. In both cases, the FDA also approved a companion diagnostic to identify the mutation; for Exkivity, it's Thermo Fisher Scientific's Oncomine DX Target Test.
In the midst of a years-long makeover, Takeda is looking to new products such as Exkivity to shore up future growth and cement its place as a major player in the pharma industry. The $62 billion purchase of Shire in 2019 vaulted the Japanese company into the top 10 drugmakers by revenue.
Takeda has been expanding in oncology as part of that revamp. Earlier this year, for example, the company announced plans to buy cancer antibody drug specialist Maverick Therapeutics for $525 million.
The potential patient population for Exkivity is small, at about 2,000 patients a year in the U.S. But it's a group in need of new options for a difficult-to-treat cancer.
To win regulatory approval, researchers tested Exkivity in patients whose cancer had progressed despite treatment with chemotherapy. The Phase 1/2 trial of 114 patients measured the response rate of tumors and how long the effect lasted.
The results were strong enough to secure an accelerated approval from the FDA. Generally, companies then have to prove that the drug also has a clinical benefit, such as prolonging survival, to maintain marketing clearance.
Like other cancer drugs such as AstraZeneca's Iressa, Exkivity is targeting mutations in a gene known as epidermal growth factor receptor, or EGFR. But only Exkivity and J&J's Rybrevant are so far approved to treat the exon 20 insertion mutation specifically.