According to a story in the WSJ, the FDA has approved a new therapy for multiple myeloma patients.
The therapy is called Carvykti and it is now available as a treatment for adult multiple myeloma patients who have faced disease progression with prior therapies. It is a customized treatment first developed by Johnson & Johnson (J&J) in China.
The company also conducted separate studies in the United States due to concerns about quality and applicability. For instance, could this therapy have different effects in the diverse population within the U.S? J&J was able to replicate their Chinese studies in the U.S. and provide even stronger data for this therapy. Their new FDA approval demonstrates this could be a way to mitigate concern around other therapies developed outside of our country, bringing novel treatments to rare disease patients faster.
Multiple Myeloma
Multiple myeloma is a blood cancer which affects the plasma cells. There are some treatments for the cancer, but although they extend survival, they aren’t a cure.
Carvykti could significantly improve patient outcomes. One study in the U.S. demonstrated that 98% of patients given the therapy experienced a significant decrease in the cancer cells. 83% of all patients experienced a complete remission. These results came just 22 months after treatment.
These kind of results are unprecedented.
Carvykti
Carvykti is a chimeric antigen receptor T cell therapy (CAR-T). This type of therapy is one of the most novel ideas in cancer research right now. It works by taking the T-cells from a patient and then modifying them. T-cells are the white blood cells which fight infections for the bodies immune system. Scientists are able to modify these cells so that they target the cancer cells and kill them. These cancer-fighting cells are then administered back to the patient.
Although this approach is extremely promising, there are also some concerns. For instance, some studies have demonstrated very severe side effects as the immune system becomes overstimulated. Close monitoring of participants is essential.
Another concern is how complex the manufacturing process is and how complex the process of actually administering the treatment to patients is. This makes them incredibly expensive.
Expense
Sales of this class of therapies went from 340 million in 2018 to 1.7 billion last year. Carvykti on its own is expected to eventually reach 5 billion in sales annually.
These are one of the most expensive drugs ever created. Carvykti is $465,000 per patient.
That said, it is a one-time therapy which does help to minimize long term costs. Additionally, J&J anticipates that most insurance companies will cover the therapy.
A drug plant has been developed in Philadelphia to manufacture this treatment and J&J plans to initiate more U.S. based studies soon.
