EMD Serono targeted therapy gets speedy FDA approval for lung cancer

An EMD Serono drug designed to treat lung cancer driven by a rare genetic alteration is now approved by the FDA, joining a growing list of targeted cancer therapies.

The FDA announced the decision for the drug, tepotinib, on Wednesday. EMD Serono, a division of Germany-based Merck KGaA, will market the pill under the name Tepmetko.

The regulatory approval covers adults whose non-small cell lung (NSCLC) cancer has spread and is characterized by an abnormal mesenchymal epithelial transition (MET) gene. These abnormalities include exon 14-skipping variants, which means that the cell’s protein-making machinery skips certain pieces of genetic code. MET exon 14 skipping is rare; the FDA has said that mutations leading to this genetic alteration are found in an estimated 3% to 4% of patients with lung cancer.

The EMD Serono drug is a kinase inhibitor, a type of drug that block enzymes key to the cellular processes driving cancer’s spread. The FDA’s decision is based on data from an ongoing open-label Phase 2 study enrolling 152 patients with advanced or metastatic NSCLC. That cancer must be characterized by MET exon 14 skipping alterations.

Patients in the study received the EMD Serono drug once daily at a dose of 450 mg until the disease progressed or the unacceptable toxicity was observed. The clinical trial’s main goal was to assess the overall response rate to treatment.

Of the 69 patients who had not previously received treatment for their cancer, the overall response rate was 43%. The median duration of that response was 10.8 months. In the 83 patients who previously received treatment for their cancer, the overall response rate was also 43% and the median duration of that response was 11.1 months. Data from the primary analysis of the study were published last September in the New England Journal of Medicine.

According to the FDA, the most common side effects observed in the study included swelling, fatigue, nausea, diarrhea, muscle pain, and breathing difficulty. The regulator also cautions that the drug can cause interstitial lung disease and liver toxicity, as well as harm to a fetus. Those risks are flagged on the drug’s label.

The accelerated FDA approval is based on the clinical data so far. In order to keep that approval, EMD Serono must conduct additional testing to confirm that the drug helps patients.

While immunotherapies have taken much of the spotlight in cancer, such treatments aren’t much help for patients whose cancers are driven by rare genetic alterations. The past year has been a watershed for new drugs that can address these signatures. Last May, Novartis’s capmatinib, which is marketed at Tabrecta, won the first FDA approval for a targeted cancer drug treating NSCLC characterized by mutations leading to MET exon 14 skipping. That drug, a pill, must be taken twice daily.

Approval of the Novartis drug was soon followed by a regulatory nod for Eli Lilly’s selpercatinib, a twice-a-day pill marketed as Retevmo. The decision includes NSCLC characterized by genetic alterations to the RET gene. Pralsetinib, a RET inhibitor developed by Blueprint Medicines and Roche as a treatment for NSCLC, was approved last September. The once-daily pill is marketed under the name Gavreto.

Tepmetko was first approved in Japan — a decision last March that came ahead of the FDA nod for Novartis’s Tabrecta. The agency’s latest decision sets up the two MET inhibitors for head-to-head competition in the U.S. The Novartis drug carries a wholesale price of $17,950 for a 28-day supply.

EMD Serono spokeswoman Julissa Viana said in an email that patients may be evaluated for treatment with Tepmetko using any available test that is approved to detect the presence of the MET exon 14 alteration. She added that the company has a partnership with ArcherDx, now a division of Invitae, to co-develop a companion diagnostic for Tepmetko in the U.S. That diagnostic is currently under review by the FDA. Until that test is approved, Viana said that patients will need to speak to a doctor about testing options.

EMD Serono may try to market its drug as giving patients a dosing advantage. Unlike the twice a day dosing of Tabrecta, the EMD Serono drug is taken once a day. That dose consists of two 225 mg tablets selling at the wholesale price of $20,898.60 for a 30-day supply. Following the Wednesday approval, Viana said that the drug is immediately available to U.S. patients.

Image by Flickr user Ed Uthman

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