Darzalex FASPRO Receives FDA Approval for AL Amyloidosis
Darzalex FASPRO® (the subcuteneous injectable version of daratumumab) with bortezomib, cyclophosphamide and dexamethasone is now the first FDA-approved combination treatment for patients with newly diagnosed Light Chain (AL) Amyloidosis. The approval comes based on the Phase 3 AMDROMEDA study for this serious condition and came as an accelerated approval.
In AL Amyloidosis, patients produce an abnormal protein that can damage vital organs, most frequently the heart, kidneys and liver. Darzalex FASPRO is not recommended for 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.
Early detection of AL amyloidosis is critical because immediate treatment can spare organ deterioration or failure.
“Today’s milestone is an important step for patients diagnosed with this rare disease,” said Isabelle Lousada, Founder and CEO, Amyloidosis Research Consortium. “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. 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.”
In the study, the Velcade/Cytoxan/Dex triple combination was compared to the same combination with Darzalex FASPRO. Patients with the Darzalex FASPRO (42%) experienced a complete response more than triple that of patients receiving VCd alone (13%).
“There is an urgent need for awareness and treatment options to help in the fight against this serious blood cell disorder,” said Raymond L. Comenzo, M.D., Director, John C. Davis Myeloma and Amyloid Program, Tufts Medical Center, and ANDROMEDA study investigator. “Achieving hematologic complete response is an important treatment goal, and today’s approval based on this clinical endpoint will provide doctors and the larger medical community with a new option to treat newly diagnosed patients.”
Approximately 4,500 people in the U.S. develop this rare disease each year and the disease is hard to diagnosis, copying common diseases. Over 1480 of those patients visit 5 or more doctors before receiving a diagnosis and 72% are diagnosed more than a year after their first symptoms. As many as 30 patients with AL amyloidosis die within the first year after diagnosis.
Common adverse reactions (side effects) were found in over 20% of patients that included upper respiratory tract infection, diarrhea, peripheral edema, constipation, fatigue, peripheral sensory neuropathy, nausea, insomnia, dyspnea and cough. Serious adverse reactions occurred in 43 percent of patients who received DARZALEX FASPRO® in combination with VCd. Serious adverse reactions that occurred in at least 5 percent of patients in the D‑VCd arm were pneumonia (9 percent), cardiac failure (8 percent) and sepsis (5 percent). Fatal adverse reactions occurred in 11 percent of patients. Fatal adverse reactions that occurred in more than one patient included cardiac arrest (4 percent), sudden death (3 percent), cardiac failure (3 percent) and sepsis (1 percent).
Importantly, 72% of patients had baseline cardiac involvement.