FDA Approves First Therapy for Certain Patients With Light Chain Amyloidosis


Daratumumab and hyaluronidase-fihj, a subcutaneous formulation of daratumumab, is the first FDA-approved treatment for patients with AL amyloidosis.

Officials with the FDA have approved daratumumab and hyaluronidase-fihj (Darzalex Faspro; Janssen) in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd) for the treatment of adult patients with newly diagnosed light chain (AL) amyloidosis. According to Janssen, daratumumab and hyaluronidase-fihj, a subcutaneous formulation of daratumumab, is the first FDA-approved treatment for patients with AL amyloidosis.

AL amyloidosis is a rare blood cell disorder associated with the production of an abnormal protein, which leads to the deterioration of vital organs, most notably the heart, kidneys and liver. Approximately 4500 people in the United States develop AL amyloidosis each year. According to Janssen, individuals with AL amyloidosis often have a poor prognosis due to the delay in diagnosis, which frequently presents with nonspecific symptoms that can mimic other, more common conditions. As many as 30% of patients with AL amyloidosis die within the first year after diagnosis.

According to Isabelle Lousada, Founder and CEO, Amyloidosis Research Consortium, the accelerated approval of the daratumumab and hyaluronidase-fihj combination therapy is an important step for patients with AL amyloidosis.

"Sadly, most patients with AL amyloidosis are diagnosed more than 1 year after their initial symptoms present, at a time when they may already be experiencing organ deterioration or failure. 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," said Lousada, in a prepared statement.

According to Janssen, approval of the daratumumab and hyaluronidase-fihj combination was based on positive results from the ongoing phase 3 ANDROMEDA (NCT03201965) study, including the hematologic complete response rate (hemCR) measure. Study results were recently presented at the American Society of Hematology (ASH) 2020 Annual Meeting.

The ANDROMEDA study includes 388 patients with newly diagnosed AL amyloidosis with measurable hematologic disease and 1 or more organs affected. The primary endpoint is overall complete hematologic response rate by intent-to-treat (ITT).

In this study, patients received daratumumab and hyaluronidase-fihj 1,800 mg/ 30,000 units administered subcutaneously once weekly from weeks 1 to 8, once every 2 weeks from weeks 9 to 24, and once every 4 weeks, starting with week 25 until disease progression or unacceptable toxicity or a maximum of 2 years. Among patients who received D-VCd, 74% were exposed for 6 months or longer and 32% were exposed for greater than 1 year.

ANDROMEDA study results showed that patients with AL amyloidosis receiving treatment with daratumumab and hyaluronidase-fihj experienced a hemCR more than triple that of patients receiving VCd alone (42% for D-VCd and 13% for VCd; P<0.0001).

The most common adverse reactions (≥20%) were upper respiratory tract infection, diarrhea, peripheral edema, constipation, fatigue, peripheral sensory neuropathy, nausea, insomnia, dyspnea and cough. Serious adverse reactions occurred in 43% of patients with AL amyloidosis who received daratumumab and hyaluronidase-fihj in combination with VCd. Serious adverse reactions that occurred in at least 5% of patients in the D—VCd arm were pneumonia (9%), cardiac failure (8%) and sepsis (5%). Fatal adverse reactions occurred in 11% of patients. Fatal adverse reactions that occurred in more than 1 patient included cardiac arrest (4%), sudden death (3%), cardiac failure (3%) and sepsis (1%).

Continued approval of daratumumab and hyaluronidase-fihj for adult patients with AL may be contingent upon verification and description of clinical benefit in a confirmatory trial.


Darzalex Faspro (daratumumab and hyaluronidase-fihj) Becomes the First FDA-Approved Treatment for Patients with Newly Diagnosed Light Chain (AL) Amyloidosis [news release]. Horsham, PA; January 15, 2021: Janssen Pharmaceuticals. Accessed January 18, 2021. https://prnmedia.prnewswire.com/news-releases/darzalex-faspro-daratumumab-and-hyaluronidase-fihj-becomes-the-first-fda-approved-treatment-for-patients-with-newly-diagnosed-light-chain-al-amyloidosis-301209575.html

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