Pfizer and EMD Serono score first the approval for mMCC.

The U.S. Food & Drug Administration (FDA) has granted approval to Bavencio (avelumab), in the form of a 20mg/mL injection, to Pfizer and EMD Serono to treat adults and children aged 12 and over with metastatic Merkel cell carcinoma (mMCC). This follows the European Medicines Agency accepting the Marketing Authorization Application for avelumab to treat mMCC in October 2016.

The announcement marks the FDA’s first approval of any drug for mMCC. The approval covers patients who have not previously undergone chemotherapy, which was hitherto the only treatment option available. mMCC is a rare, very aggressive form of skin cancer that affects about 1,600 patients/year in the U.S. Fewer than 20% survive for more than five years.

“At the heart of this FDA approval is our drive to make a meaningful difference for patients with hard-to-treat cancers like mMCC,” said Belén Garijo, CEO Healthcare at Germany’s Merck KGaA, which owns EMD Serono. “Bavencio’s journey has included years of hard work – from the scientists who discovered this molecule in our labs to our alliance with Pfizer and to the study participants and investigators worldwide.”

Pfizer and EMD Serono formed their alliance in immuno-oncology in November 2014, when Pfizer first bought into avelumab, a monoclonal antibody, for $850 million up-front and up to $2 billion in potential milestone payments. Since then, all development has been funded jointly and the costs of commercialization will be shared between them on a 50-50 basis. The alliance includes co-promotion of Pfizer’s targeted lung cancer drug Xalkori in the U.S. and other markets.

Bavencio is designed to engage both the adaptive and the innate immune systems. The drug binds to the PD-L1 pathway, preventing tumor cells from using it for protection against white blood cells, such as T-cells, and exposing them to anti-tumor responses. It has also been shown to induce antibody-dependent cell-mediated cytotoxicity in vitro.

Bavencio was reviewed and approved through the FDA’s Breakthrough Therapy Designation and Priority Review programs and also received Orphan Drug designation. It received Accelerated Approval because it addresses an unmet medical need.

Data from the Javelin Merkel 200 trial, a single-arm, multi-study center trial of 88 patients with metastatic MCC who had been previously treated with at least one prior chemotherapy regimen, predicted a clinical benefit to patients, as 33% of participants experienced complete or partial shrinkage of their tumors. Further clinical trials are required to confirm this and are ongoing.

Avelumab is also being tested for other types of cancer types, including non-small cell lung, ovarian, gastric, bladder and renal cancers as part of the wider Javelin clinical trial program, which brings together multiple trials of PD-L1 inhibition with avelumab in oncology treatments. Pfizer and Merck KGaA are expected to file for it in the treatment of non-small cell lung cancer later this year.

Bavencio now joins several other potential blockbusters that target the PD-1/L1 pathway in the treatment of cancers. Already on the market for the treatment of melanoma, but also pushing for other approvals, are Opdivo (nivolumab) by Bristol-Myers Squibb and Keytruda (pembrolizumab) from Merck & Co.