Regeneron and Sanofi bust into new I-O market with FDA nod for Libtayo in basal cell carcinoma

Libtayo bottle and box
In a phase 2 trial, the response rate to Libtayo was 21% in metastatic basal cell carcinoma and 29% in locally advanced disease.(Sanofi Genzyme/Regeneron)

Regeneron and Sanofi’s anti-PD-1 cancer drug Libtayo has been pegged as an immuno-oncology laggard in diseases such as lung cancer, where well-established players like Merck’s Keytruda rule the market. But there’s one area where Regeneron and Libtayo can claim a lead: skin cancer.

In that department, the FDA has added another jewel to Regeneron and Sanofi’s crown, approving Libtayo to treat patients with advanced basal cell carcinoma (BCC). It’s the first immunotherapy drug cleared by the FDA to treat that disease, and it comes two years after Libtayo’s first approval in metastatic cutaneous squamous cell carcinoma.

The new approval, for patients whose BCC advanced after treatment with a hedgehog pathway inhibitor or who are not eligible for that class of drug, was based on results from a phase 2 trial. In that study, the drug spurred a response in 21% of patients with metastatic BCC and 29% of those with locally advanced disease, and most patients benefited for more than six months.

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When Sanofi and Regeneron reported data from the BCC trial at the virtual meeting of the European Society of Clinical Oncology last September, they estimated the probability of survival would be 92% in patients taking Libtayo.

“This is a horribly disfiguring disease in the advanced stage. We’ve seen some amazing improvements in patients,” Israel Lowy, M.D., Ph.D., Regeneron's senior vice president of translational and clinical science in oncology, said in an interview at the time.

The new approval will no doubt nudge Libtayo closer to the blockbuster status analysts have predicted the drug can achieve. The product brought in $251 million in sales in the first nine months of 2020, doubling its haul from the same period a year before. Analysts expect Libtayo’s dominance in skin cancer will turn it into a $1.4 billion-a-year product.

But Sanofi and Regeneron have ambitions beyond skin cancer. The companies are anticipating a verdict from the FDA by the end of this month on their bid for approval of Libtayo in advanced non-small cell lung cancer (NSCLC).

The FDA granted priority review to Libtayo in NSCLC in October after Sanofi and Regeneron released trial data showing the drug cut the risk of death by 43% in patients with PD-L1 levels of at least 50%, as compared to platinum chemotherapy.

RELATED: Watch out, Merck: FDA grants Regeneron, Sanofi's Libtayo priority review for coveted first-line use in lung cancer

Sanofi and Regeneron won’t have the benefit of being the sole PD-1 players in the lung cancer market, however. Merck’s Keytruda has approvals in NSCLC as a solo therapy and as part of a market-leading chemo combination. And Bristol Myers Squibb’s Opdivo-Yervoy combination was approved in first-line NSCLC last May.

Still, Libtayo’s developers see opportunities well beyond skin and lung cancers. They have a massive clinical trial program underway examining the drug’s potential in cervical cancer, solid tumors and blood cancers.