Adverum said it would cease developing ADVM-022 for DME and focus on wet AMD, but the adverse events that prompted the shift could invite further scrutiny in an already competitive market.

The company plans an intensive review to determine the path forward for SER-287. A similar deep dive led to the successful revival of its C. difficile drug, SER-109, which it plans to submit for approval.

The company anticipates an addressable global market for progressive familial intrahepatic cholestasis of 2,500 patients, with initial plans to focus on around 100 physicians at 60 centers.

While not an outright rejection, the Deficiencies Preclude Discussion letter came as a surprise given the expectation of the drug’s approval for hyperphosphatemia in adults with chronic kidney disease on dialysis.

Merck unveiled event-free survival results for Keytruda in triple-negative breast cancer following failure earlier this year to gain approval on a surrogate endpoint.

The company has already expanded into Asia and shown early clinical data from its own cell therapy programs as it acquires Kite Pharma’s neoantigen TCR R&D program and manufacturing plant.