With Sanofi entering the multiple myeloma market, J&J flashes data on new rare disease
Since its 2015 approval, Darzalex has earned J&J billions as one of the most potent drugs available for multiple myeloma, a common cancer that for years had few good therapies. Now, as Sanofi enters with a similar drug, the company is edging closer to expanding its uses.
Most notably, they have been testing the drug on AL amyloidosis, a rare and potentially fatal disease in which B cells produce misshapen antibodies. These abnormal antibodies scrunch together to form clumps called amyloid (similar to the plaques implicated in Alzheimer’s but with different proteins), which can build up in a number of organs and prevent them from functioning properly. Patients live a median of 6 months to 3 years after diagnosis. There are no specifically approved therapies, but some patients also have multiple myeloma — also a B cell malignancy — and doctors have tried using drugs developed for the cancer as a treatment.
In a pivotal Phase III trial presented this weekend at the European Hematology Association virtual meeting, J&J tested three medications often used to treat AL amyloidosis against those three medications plus Darzalex. Although there are no data yet on overall survival, the J&J combo cleared tumors at a far higher clip than the control, which included the multiple myeloma drug Velcade and two chemotherapies. The complete response rate was 53% compared with 18%. The drug also showed 42% higher progression free survival and proved better at preventing heart or kidney deterioration.
The company plans to file approval in the US and the EU later this year, according to a J&J presentation from April. The trial used the subcutaneous form of the drug. Approved earlier this year for multiple myeloma, it reduced the time it takes to administer the drug from 7 hours to 5 minutes.
In a statement, lead investigator Raymond Comenzo pointed to the long-running challenges in treating AL amyloidosis. The disease can fester for years before major symptoms appeared, and then they are often confused with more common ailments.
“Patients with AL amyloidosis often experience poor outcomes because their symptoms are confused with more common conditions, causing delays in diagnosis,” Comenzo said. “The newly diagnosed patient population in AL amyloidosis is the most challenging to treat, and many do not reach second-line therapy.
“The higher rate of hematologic complete response,” he added, before using the chemical name for the drug, “as well as the notable sustainment of major organ function in those receiving subcutaneous daratumumab in the ANDROMEDA study, suggest that subcutaneous daratumumab may be a promising treatment for newly diagnosed patients with AL amyloidosis who have been, for some time, in urgent need of new therapeutic options.”
Darzalex earned J&J a hair under $3 billion last year, but they will face competition going forward. Sanofi landed their first in-house cancer approval in a decade in March for Sarclisa, a multiple myeloma drug that works by the same mechanism as Darzalex, blocking a receptor that’s overexpressed on multiple myeloma cells, called CD38.
Other trials testing multiple myeloma drugs in amyloidosis are underway, including one — run by Southwest Oncology and the National Cancer Institute — on Sarclisa.
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