- The Food and Drug Administration on Tuesday approved a first-of-its-kind treatment for multiple myeloma from Johnson & Johnson, but put restrictions on its use due to the drug’s potentially dangerous side effects.
- Healthcare providers offering the drug, which will be sold as Tecvayli, will need to follow guidelines set up in a Risk Evaluation and Mitigation Strategy, or REMS. Prescribers and pharmacies must be certified in the Tecvayli REMS program, which will focus on monitoring and counseling for patients.
- The FDA has required REMS for dozens of medicines since the program was authorized by Congress in 2007. The list includes Bristol Myers Squibb’s cell therapy Abecma, which won approval for multiple myeloma last year.
Tecvayli is part of a growing group of drugs that target a protein called BCMA found on malignant cells in multiple myeloma. GlaxoSmithKline was the first to market with a treatment aimed at BCMA, after winning FDA clearance for the antibody-drug conjugate Blenrep in 2020. Abecma also targets BCMA, but it’s a CAR-T therapy that’s produced using a patient’s own T cells.
J&J sells a CAR-T treatment of its own for multiple myeloma, called Carvykti. However, its newest medicine can be used “off the shelf,” rather than the personalized manufacturing process needed in cell therapy. It’s known as a bispecific therapy and is the first to target BCMA while also binding to the CD3 receptor on T cells. Bringing the cancerous and protective cells together helps spur the immune system into action to curb cancer growth.
While the REMS requirement was unexpected, the compelling data behind Tecvayli should help drive its sales to more than $2 billion a year, David Risinger, an analyst at SVB Securities, wrote in a note to clients.
Tecvayli represents J&J’s fourth approved treatment in multiple myeloma. While there are many other medicines available, there’s still a great need for new options because patients with the blood cancer often relapse after cycling through therapies or combinations of drugs. In the pivotal clinical trial for Tecvayli, patients had received a median of five prior treatments.
The study, dubbed MajesTEC-1, found that 63% of participants had a reduction or disappearance of cancerous cells following treatment with Tecvayli. About two-thirds of those patients experienced a response lasting for at least one year.
Still, 72% of patients who received Tecvayli in the trial suffered from cytokine release syndrome, an overreaction of the immune system that can be fatal. And 57% of participants who received the recommended dose had neurological side effects. That safety profile led the FDA to require the REMS program for the drug.
The decision suggests the FDA is “treading cautiously” with these new types of treatments, and bispecific therapies in the future may well have similar requirements, SVB’s Risinger wrote in his note to investors.
The FDA gave Tecvayli an accelerated approval based on the drug’s ability to fight malignant cells. The therapy may require additional data showing a clinical benefit to stay on the market.