Janssen Submits sBLA for Darzalex Faspro to Treat Light Chain Amyloidosis

On September 10, Janssen announced the submission of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for the subcutaneous injection of Darzalex Faspro (daratumumab/hyaluronidase-fihj) for the treatment of patients with light-chain amyloidosis.

Light-Chain Amyloidosis is a rare, life-threatening disease in which plasma cells in the bone marrow produce abnormal light chains that form amyloid deposits. These deposits accumulate in vital organs and ultimately lead to organ deterioration. Light-chain amyloidosis can affect multiple organs, with the heart, kidneys, liver, spleen, gastrointestinal tract, and nervous system being the most severely impacted. Because the clinical symptoms of light-chain amyloidosis can mimic those of other diseases, clinical diagnosis is often delayed, resulting in a poor prognosis. Currently, the FDA has not approved any drugs for the treatment of light-chain amyloidosis.

Although light-chain amyloidosis is the most common type of amyloidosis, it remains a rare disease. There are currently approximately 30,000 to 45,000 patients in the United States and Europe, with about 4,500 new cases diagnosed annually in the United States.

Janssen’s current application is primarily based on the positive data from the Phase III ANDROMEDA study. This study enrolled 388 patients with newly diagnosed light-chain amyloidosis, who were randomized to receive either subcutaneous daratumumab in combination with bortezomib, cyclophosphamide, and dexamethasone (D-VCd) or VCd alone. The results demonstrated that the D-VCd regimen significantly improved the overall hematologic response rate compared with the VCd regimen, thereby meeting the primary endpoint.

Daratumumab is a monoclonal antibody targeting CD38, a membrane surface protein that is highly expressed on multiple myeloma cells at all stages of growth. It is the first globally marketed anti-CD38 monoclonal antibody developed by Janssen Pharmaceuticals. Daratumumab binds to CD38 and inhibits tumor cell growth, leading to the death of myeloma cells.