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FDA Approval: Treatment Advance for Patients with Rare Blood Cell Disorder

Rye Brook, N.Y., January 19, 2021 – On January 15, the U.S. Food and Drug Administration (FDA) announced approval of daratumumab plus hyaluronidase (Darzalex Faspro®) for adults with newly diagnosed light chain (AL) amyloidosis, a rare and serious blood cell disorder that may occur by itself or with myeloma. Daratumumab is used in combination with chemotherapy agents bortezomib (Velcade®) and cyclophosphamide (Cytoxan® or Neosar®), plus the corticosteroid dexamethasone in these patients.

Approximately 4,500 people in the U.S. develop this rare disease each year. AL amyloidosis is a life-threatening disorder that occurs when blood plasma cells in the bone marrow produce amyloid deposits, which build up in vital organs and eventually cause organ deterioration.

Daratumumab is the only CD38-directed antibody approved to be given subcutaneously to treat AL amyloidosis and has been approved for multiple myeloma since 2015. LLS supported early research into CD38 as a target in myeloma treatment over 15 years ago. LLS also supported research investments focused on improving outcomes for patients with AL amyloidosis both directly and through its collaboration with the International Waldenstrom’s Macroglobulinemia Foundation (IWMF). Dr. Morie Gertz (Mayo Clinic/Rochester MN) has recently completed studies examining clinical characteristics of AL in WM patients and the molecular basis of the disease. Dr. Bibiana Rius in the laboratory of Dr. Wiseman (Scripps Research Institute, CA) is exploring ways to block secretion of light chain proteins. These proteins ultimately lead to the formation of amyloid fibrils that damage organs such as the heart.

The recent FDA approval was based on results from the phase 3 ANDROMEDA study, which was presented at the American Society of Hematology (ASH) 2020 Annual Meeting. Patients who received daratumumab plus hyaluronidase along with the standard treatment combination of bortezomib, cyclophosphamide, and dexamethasone had a three times higher hematologic complete response rate than patients who received the standard three-drug regimen alone (42% versus 13.5%). The daratumumab-treated patients also had longer progression-free survival times.

LLS is committed to supporting leading-edge research for every type of blood cancer. Our robust academic grant and venture philanthropy programs support both foundational research, such as the early work on the CD38 gene that underpins the success of daratumumab, and clinical studies that are the final bridge that move effective treatments from the laboratory into clinical practice where they can improve the lives of people with all forms of blood cancer.