FDA Approves Daratumumab, Hyaluronidase Combination for AL Amyloidosis


The indication is the first of its kind for the rare, sometimes quickly fatal disease.


The US Food and Drug Administration (FDA) has approved daratumumab and hyaluronidase-fihj (DARZALEX FASPRO) in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd) for the treatment of adult patients newly diagnosed with light chain (AL) amyloidosis.

The decision makes the combination therapy the first indicated for patients with the rare, possibly fatal blood disorder.

AL amyloidosis is associated with abnormal protein production which leads to vital organ deterioration. Patients afflicted with the rare disease may suffer severe damage to their heart, kidneys, and/or liver. Up to 30% of the 4500 patients diagnosed in the US annually can die within the first year.

The FDA approval was based on the phase 3 findings from the ANDROMEDA study, in which the combination was assessed with VCd, versus lone VCd therapy, in patients with AL amyloidosis. Investigators reported at the American Society of Hematology (ASH) 2020 Annual Meeting that patients treated with the daratumumab and hyaluronidase combination experienced a three-fold improved hematologic complete response rate versus patients given lone VCd (42% vs 13%; P <.0001).

"We are delighted that DARZALEX FASPRO® utilizing our ENHANZE® technology has become the first and only FDA-approved treatment for newly diagnosed patients with AL amyloidosis," said Helen Torley, MD, president and chief executive officer of Halozyme Therapeutics, in a statement. "AL amyloidosis represents an area of high unmet medical need."

ANDROMEDA investigator Ray Comenzo, MD, spoke with Rare Disease Report® on the ongoing phase 3 trial back in 2018, alluding to the hope that DARZALEX FASRPO could eventually top VCd as the standard of therapy.

“It’s a very exciting prospect for the newly diagnosed amyloid patients of the future to potentially have a combination of 4 drugs that are safe and that are almost 100% effective,” Comenzo said at the time. “That’s what we expect; we expect many, many patients to be effectively treated and to recover.”

In May 2020, Janssen Pharmaceutical was granted FDA approval for daratumumab and hyaluronidase as a multiple-regimen subcutaneous formulation for the treatment of patients with multiple myeloma—4 regimens approved for 5 multiple myeloma indications.

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