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1st FDA-Approved Drug for AL Amyloidosis

After decades of relying on treatments approved for other diseases, on January 15, 2021 the FDA approved the first drug for AL Amyloidosis. Truly a game-changing, monumental advancement in the treatment of this disease.


On January 15, 2021, the Food and Drug Administration granted accelerated approval to daratumumab plus hyaluronidase (Darzalex Faspro, Janssen Biotech Inc.) in combination with bortezomib, cyclophosphamide and dexamethasone (CyBorD) for newly diagnosed light chain (AL) amyloidosis. “AL amyloidosis is a devastating and potentially fatal blood disorder that, until now, did not have any U.S. FDA-approved therapies. This makes today’s approval of DARZALEX FASPRO a critical step forward for patients in the U.S. in dire need of treatment options,” said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.


Amyloidosis is a disease that occurs when amyloid proteins, which are abnormal proteins, accumulate in tissues and organs. When the amyloid proteins cluster together, they form deposits that damage the tissues and organs. AL amyloidosis most frequently affects the heart, kidneys, liver, nervous system and digestive tract. Until now there were no approved therapies for AL amyloidosis in the U.S., though it is currently being treated with chemotherapy, dexamethasone, stem cell transplants and supportive therapies. It is estimated that there are approximately 3,000 to 4,000 new cases of AL amyloidosis diagnosed annually in the U.S.


Efficacy was evaluated in ANDROMEDA (NCT03201965), an open-label, randomized, active-controlled trial in 388 patients with newly diagnosed AL amyloidosis with measurable disease and at least one affected organ according to consensus criteria. Patients were randomized to receive bortezomib, cyclophosphamide, and dexamethasone (VCd arm) or with Darzalex Faspro (D-VCd arm).

The hematologic complete response (HemCR) rate based on established consensus response criteria as evaluated by an independent review committee was 42.1% for the D-VCd arm and 13.5% for the VCd arm (odds ratio=4.8; 95% CI: 2.9, 8.1; p<0.0001).

The prescribing information includes a Warnings and Precautions that serious or fatal cardiac adverse reactions occurred in patients with light chain (AL) amyloidosis who received Darzalex Faspro in combination with bortezomib, cyclophosphamide and dexamethasone. Darzalex Faspro is not indicated and is not recommended 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.

The most common adverse reactions (≥20%) in patients with light chain (AL) amyloidosis who received the D-VCd regimen are upper respiratory tract infection, diarrhea, peripheral edema, constipation peripheral sensory neuropathy, fatigue, nausea, insomnia, dyspnea and cough.


Darzalex Faspro is an immunotherapy that works with your body to fight disease, preventing the abnormal plasma cells from creating excess light chains.

Darzalex Faspro is a subcutaneous injection, typically administered with several additional drugs intended to minimize reactions. Treatment usually begins with weekly injections for eight weeks, followed by bi-weekly injections for another eight weeks, and then monthly injections thereafter.


The FDA approved this application 7 weeks ahead of the FDA goal date. This application was granted accelerated approval based on response rate.

With FDA approval, this gives healthcare professionals even more ammunition in the treatment of amyloidosis, and should positively impact (i.e., reduce) complexities that patients often experienced in order to receive insurance company approval. Darzalex Faspro (daratumumab and hyaluronidase-fihj), approved for AL amyloidosis, now joins three other FDA-approved drugs for TTR amyloidosis  (Onpattro (patisiran), Tegsedi (inotersen) and Vyndamax (tafamidis)).



  1. Genmab announcement
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