Merck KGaA and Pfizer have done it.
The biopharma partners came away with their first FDA approval of the checkpoint inhibitor avelumab, making it the fourth drug in this category to make it to the market. They beat AstraZeneca, which will now angle for its first approval of durvalumab as the 5th checkpoint.
The FDA gave out its approval for Merkel cell carcinoma under an accelerated approval process. The therapy, the first for a rare form of skin cancer, has also been given breakthrough drug status. The drug is currently in 30 clinical programs as the two players look to wedge their way into a multibillion-dollar market for drugs that are gaining wide use in treating cancer.
The approval marks another big advance for Pfizer, which paid $850 million upfront to partner with Germany’s Merck — a record sum. The approval of this anti-PD-L1 IgG1 monoclonal antibody also marks a major win for Merck KGaA, which has suffered through more than a decade without a blockbuster OK. Merck KGaA also came away with a package of regulatory and commercial milestones on avelumab worth up to $2 billion when it tied up with Pfizer in 2014.
The therapy will be sold as Bavencio. A spokesperson for Pfizer said the drug will cost $13,000 a month wholesale — ahead of payer discounts.
The new approval marks just how quickly the FDA can move when it wants to. The OK is based on a single arm study including 88 patients. Of the 88 patients who received Bavencio in the trial, says the FDA, 33% experienced complete or partial shrinkage of their tumors. The response lasted for more than six months in 86% of responding patients and more than 12 months in 45% of responding patients.
Not only was avelumab a big success as a checkpoint, the development program from start to first approval was executed swiftly.
“Three-and-a-half years after the first-in-human dose escalation studies, we are getting our first approval,” says Luciano Rossetti, the head of R&D at Merck Serono. And don’t expect anyone to start sitting on laurels. Checkpoint inhibition is a big field, and the Pfizer/Merck KGaA team plan to make their mark as the pioneers divvy up leadership roles in a wide array of cancers.
AstraZeneca rejigged its development campaign for the next checkpoint in the pipeline, relying heavily on its work in combining it with the CTLA-4 checkpoint tremelimumab to try and leapfrog the fast moving trio in the lead: Merck, Bristol-Myers Squibb and Roche/Genentech. They’ll be staring down some of the biggest rivals in the industry in a make-or-beak effort to distinguish its R&D operations, which has had several successes in cancer but continues to be plagued by setbacks in the clinic and at the FDA.
You can add Merck KGaA to the list now.
“You need to have a strong PD1/ PD-L1 to anchor combinations for other assets,” Rossetti tells me. And now they have one approved and entering the market.
The FDA accepted their application for urothelial carcinoma just a few weeks ago, and Rossetti says they’re pushing hard on ovarian, gastric and lung cancer work that could pave the way for near-term approvals.
“To be practical,” he adds, “the biggest opportunity is in non-small cell lung cancer.” One of those big opportunities is demonstrating an effect in low PD-L1 expressers with researchers also exploring high intensity therapy for high expressers.
Rossetti, though, is careful to offer no big promises on what the data will show. He’s acutely aware that when they modified the lung cancer study, the company was gambling on a disease that proved a disaster for Bristol-Myers Squibb and a chance for Merck to leap ahead of the pack.
The first thing that Rossetti did when he was named R&D chief in 2014 was to focus the pipeline, getting rid of 13 programs so they could concentrate on a few select efforts like avelumab. Now he says the company is interested in doing a few deals to help beef up its focus on combinations and more. But they’ll be very strategic about launching new trials.
“While skin cancer is one of the most common cancers, patients with a rare form called Merkel cell cancer have not had an approved treatment option until now,” said Richard Pazdur, MD, acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and director of the FDA’s Oncology Center of Excellence. “The scientific community continues to make advances targeting the body’s immune system mechanisms for the treatment of various types of cancer. These advancements are leading to new therapies—even in rare forms of cancer where treatment options are limited or non-existent.”
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