Janssen Pharmaceutical’s DARZALEX FASPRO gains FDA approval for AL amyloidosis treatment

Janssen Pharmaceutical, a subsidiary of Johnson & Johnson, has secured FDA approval for DARZALEX FASPRO (daratumumab and hyaluronidase-fihj), marking a major milestone in the treatment of light chain (AL) amyloidosis. This approval, granted under the accelerated approval pathway, positions DARZALEX FASPRO as the first and only FDA-approved treatment for AL amyloidosis.

AL amyloidosis is a rare and life-threatening blood cell disorder characterized by the abnormal production of amyloid proteins, which accumulate and damage vital organs, especially the heart, kidneys, and liver. With this new approval, DARZALEX FASPRO, a subcutaneous formulation of daratumumab, offers a critical treatment option for patients with newly diagnosed AL amyloidosis.

FDA Approval Based on Promising Clinical Trial Results

The approval follows encouraging data from the Phase 3 ANDROMEDA clinical trial, which assessed the combination of DARZALEX FASPRO with standard chemotherapy (bortezomib, cyclophosphamide, and dexamethasone, or VCd) compared to VCd alone. The trial demonstrated a significant improvement in treatment outcomes for those receiving the DARZALEX FASPRO combination, with patients showing a hematologic complete response (hemCR) rate more than triple that of those on VCd alone.

These positive results from ANDROMEDA underscore the potential of DARZALEX FASPRO to significantly impact patient outcomes in this underserved population. However, continued approval will depend on further verification and confirmation of the clinical benefits in a subsequent trial, as is customary with accelerated approvals.

Expert Insights on the Approval and Its Impact

Experts in the field are already heralding this approval as a turning point for AL amyloidosis patients. Isabelle Lousada, Founder and CEO of the Amyloidosis Research Consortium, emphasized the importance of this approval, noting the challenge of diagnosing AL amyloidosis in its early stages. “Sadly, most patients are diagnosed after significant organ damage has already occurred,” she explained. “This approval offers new hope for patients and their caregivers, while also increasing awareness of this rare and often overlooked disease.”

Dr. Raymond L. Comenzo, Director of the John C. Davis Myeloma and Amyloid Program at Tufts Medical Center and an investigator in the ANDROMEDA trial, also highlighted the urgency of addressing this condition. “Achieving a hematologic complete response is a critical treatment goal, and this approval provides the medical community with an important new option for treating newly diagnosed AL amyloidosis patients,” he said.

A Step Forward for AL Amyloidosis Treatment

While DARZALEX FASPRO is now approved for use in newly diagnosed AL amyloidosis patients, it is important to note that it is not recommended for patients with advanced heart disease (NYHA Class IIIB or IV cardiac disease or Mayo Stage IIIB) outside of controlled clinical trials. This limitation underscores the need for careful patient selection and continued research to fully understand the treatment’s benefits across different patient populations.

With the FDA’s approval of DARZALEX FASPRO, the treatment landscape for AL amyloidosis has taken a significant step forward, providing patients with a promising new option in their fight against this rare and challenging disease.