A Johnson & Johnson drug addressing a novel target is now FDA approved, introducing a new treatment option for patients with advanced cases of multiple myeloma and further broadening the drugmaker’s product portfolio in this type of blood cancer.

The Thursday approval of the drug, talquetamab, covers the treatment of adults whose multiple myeloma has relapsed or has not responded to at least four prior lines of therapy. J&J subsidiary Janssen developed the drug as a subcutaneous injection given weekly or every two weeks after step-up dosing. The product will be marketed under the name Talvey.

Talvey is a bispecific T cell engager designed to bind to two targets, one on a T cell and the other on a cancer cell. On the T cell, the target is CD3, a protein that is also the target of other bispecific antibody drugs. On cancer cells, Talvey hits a novel target called GPRC5D. This protein is highly expressed on multiple myeloma cells, making it a good target for new drugs treating this cancer of plasma cells found in bone marrow.

The FDA decision for Talvey was based on the results of a single-arm, open-label Phase 2 study that included 187 patients who had received at least four earlier lines of therapy and were not exposed to a T cell redirection therapy. The main goal was to measure the overall response rate. At the biweekly dose of 0.8 mg, results showed 73.6% of patients achieved an overall response. Of those who showed a response, 58% of achieved a very good partial response or better at a median follow-up period six months; 33% achieved a complete response or better.

The results were comparable at the weekly dose of 0.4 mg, with 73% of patients achieving an overall response to the therapy. Among those responders, 57% achieved a very good partial response or better at a median follow-up of nearly 14 months; 35% achieved a complete response or better. An interim look at the clinical trial data were presented last December during the annual meeting of the American Society of Hematology.

The most common adverse reactions reported in the study include fever, cytokine release syndrome, a foul taste in the mouth, muscle pain, rash, and weight loss. Talvey’s label includes a black box warning regarding cytokine release syndrome, which is an excessive immune response. The warning also includes an alert for neurologic toxicity, including immune effector cell-associated neurotoxicity syndrome. These adverse effects are known complications of immunotherapies and can be managed by clinicians. But Talvey is available only under a Risk Evaluation and Mitigation Strategy, a program that mitigates the safety risks of a drug.

Mark Wildgust, vice president of oncology global medical affairs at Janssen, said in a June interview during the annual meeting of the American Society of Clinical Oncology that multiple myeloma remains an incurable disease. Despite the availability of various multiple myeloma drugs, many patients still relapse. He said Janssen’s strategy is to bring new treatment options for patients who have exhausted earlier lines of therapy.

“We think harnessing the immune system is the way to go,” he said.

Talvey is now J&J’s fifth multiple myeloma therapy. Its approval comes less than a year after the FDA nod for Tecvayli, a bispecific antibody whose cancer target is B-cell maturation agent, or BCMA. While other BCMA-targeting drugs are available, Talvey is the first drug approved (so far) that goes after GPRC5D. Wildgust noted that Talvey is dosed according to a patient’s weight, which enables a clinician to tailor the dose to optimize for both safety and efficacy.

The Talvey regulatory decision is an accelerated approval based on the response rate and the duration of response in the Phase 2 trial. J&J will need to verify the drug’s benefit with post-marketing confirmatory clinical testing. That Phase 3 study is ongoing. J&J has also been conducting clinical trials to evaluate Talvey in earlier lines of therapy and in combination with the company’s other multiple myeloma drugs.

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