A Johnson & Johnson drug for multiple myeloma has won FDA approval, adding a new therapy to the growing list of products that go after a hot target for this type of blood cancer. But this one comes in a formulation that sets it apart from the rest of that field.
New multiple myeloma treatments that have reached the market in recent years are given as intravenous infusions that can take up to an hour—not including the complex manufacturing process and patient preconditioning regimens required for the treatments that are cell therapies. J&J’s teclistamab is a subcutaneous injection.
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The approval announced Tuesday covers the treatment of adults whose multiple myeloma has relapsed or has not responded to four or more earlier lines of treatment. This drug developed by J&J subsidiary Janssen Biotech will be marketed under the name “Tecvayli,” the same brand name for the medicine in Europe, where it was first approved in August.
Tecvayli is a type of antibody drug called a bispecific T-cell engager. One part of the antibody binds to B-cell maturation antigen (BCMA), a protein that’s highly expressed on multiple myeloma cells. The other part binds to CD3, a receptor on the surface of T cells. The binding to T cells sparks the release of proinflammatory proteins that ruptures cancer cells, killing them.
The abundance of BCMA on multiple myeloma cells has made it a popular target for companies developing new therapies for this type of cancer, which affects plasma cells in bone marrow. In 2019, GSK’s Blenrep won the first FDA approval for a BCMA-targeting drug. That therapy, an antibody drug conjugate, is dosed as an infusion. Cell therapies were the next to pass the regulatory bar. Abecma, a CAR T-therapy from partners Bristol Myers Squibb and 2SeventyBio, won FDA approval last year as a fifth line of treatment for multiple myeloma. J&J also has an FDA-approved, CAR T-drug for multiple myeloma. In March, the regulator greenlit Carvykti, developed by Janssen and partner Legend Biotech, as a fifth-line treatment. CAR T-therapies are produced in a lengthy process that involves harvesting a patient’s T cells, engineering them in a lab, then reinfusing them into the patient. By contrast, Tecvayli is an off-the-shelf option that can be used right away.
The FDA based its Tecvayli approval on the results of a single-arm, open-label Phase 2 study that evaluated the therapy in 110 multiple myeloma patients who had previously received at least three prior treatments—none of them a therapy that targets BCMA. The overall response rate for Janssen’s BCMA-targeting drug was 61.8%. In those responders, the duration of response rate was estimated to be 90.6% at six months and 66.5% at nine months. The study results were presented at the annual meeting of the American Society of Clinical Oncology in June and also published in the New England Journal of Medicine.
Tecvayli’s easier dosing doesn’t mean it has a safety edge. The drug’s label carries a black box warning highlighting the potential risk of an excessive immune response called cytokine release syndrome (CRS). In addition, the warning flags the risk of neurologic toxicity. These potentially fatal adverse effects are also a risk of CAR T-therapies. The FDA noted that CRS occurred in 72% of Tecvayli study participants; neurologic toxicity was observed in in 57% of patients. CRS classified as Grade 3 in severity (Grade 5 is toxicity resulting in death) occurred in 0.6% of patients; Grade 3 or 4 neurologic toxicity was observed in 2.4% of patients.
In the first week of treatment, the drug is given in gradually increasing doses that enable clinicians to monitor for CRS or other problems. After the first week, the drug is injected once weekly. Tecvayli is available only through a program that informs prescribers and patients about the drug’s risks.
The regulatory nod for Tecvayli is an accelerated approval, which requires companies to provide additional clinical data to confirm a drug’s safety and efficacy. Janssen expects that Tecvayli will become available in the U.S. on or around Nov. 4. The company has set a $39,500 per month price for the drug. In the clinical trial, the average patient was treated for nine to 10 months. That means the total cost could reach up to $395,000, though Janssen expects the drug will be covered by Medicare and most commercial insurance plans. Janssen’s Carvykti, a one-time treatment, has a $465,000 list price.
Photo: Niels Wenstedt/BSR Agency, Getty Images
