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FDA approves first treatment for newly diagnosed light chain (AL) amyloidosis

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The U.S. Food and Drug Administration has approved daratumumab and hyaluronidase-fihj (Darzalex Faspro) in combination with bortezomib, cyclophosphamide, and dexamethasone (D-VCd) for the treatment of adult patients with newly diagnosed light chain (AL) amyloidosis. Daratumumab and hyaluronidase-fihj is the only FDA-approved treatment for this disorder and is subcutaneous formulation of daratumumab.

It is not indicated for the treatment of patients with light chain (AL) amyloidosis who have NYHA Class IIIB or Class IV cardiac disease or Mayo Stage IIIB outside of controlled clinical trials.

Approval is based on results from the Phase 3 ANDROMEDA study, which evaluated daratumumab and hyaluronidase-fihj in combination with VCd in patients with newly diagnosed AL amyloidosis compared with VCd alone. Patients receiving the combination therapy had a hematologic complete response rate (hemCR)

of 42% compared to 13% in patients receiving VCd (P < 0.0001).

“Today’s milestone is an important step for patients diagnosed with this rare disease,” said Isabelle Lousada, Founder and CEO, Amyloidosis Research Consortium, in a press release. “Sadly, most patients with AL amyloidosis are diagnosed more than one year after their initial symptoms present, at a time when they may already be experiencing organ deterioration or failure.4 I believe this approval will increase awareness of and education around this life-threatening disease and offer new hope for people with AL amyloidosis and their caregivers.”

Read the full press release here.