FDA Grants Breakthrough Therapy Designation to Janssen Off-the-Shelf Cell Therapy for Multiple Myeloma

The U.S. Food and Drug Administration granted Janssen Pharmaceutical Breakthrough Therapy designation for its off-the-shelf cell therapy teclistamab for the treatment of relapsed or refractory multiple myeloma.

This designation for teclistamab follows a PRIME (PRIority MEdicines) designation from the European Medicines Agency earlier this year.

Multiple myeloma is an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow. When damaged, these plasma cells rapidly spread and replace normal cells with tumors in the bone marrow. In 2021, it is estimated that nearly 35,000 people will be diagnosed and more than 12,000 will die from the disease in the United States. While some patients with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems, or infections.

Teclistamab is an off-the-shelf, T-cell redirecting, bispecific antibody targeting both BCMA and CD3 receptors. BCMA is expressed at high levels on multiple myeloma cells. Teclistamab redirects CD3-positive T-cells to BCMA-expressing myeloma cells to induce killing of tumor cells. Results from preclinical studies demonstrate that teclistamab kills myeloma cell lines and bone marrow-derived myeloma cells from heavily pretreated patients.

The FDA grants Breakthrough Therapy designation to expedite the development and regulatory review of an investigational medicine that is intended to treat a serious or life-threatening condition and is based on preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.

The designation is supported by data from the phase 1 MajesTEC-1 study, an open-label, multicenter clinical trial evaluating the safety and efficacy of teclistamab in adults with measurable multiple myeloma that is relapsed or refractory to established therapies or be intolerant of those established multiple myeloma therapies.

Teclistamab is currently being evaluated in a phase 2 clinical study for the treatment of relapsed or refractory multiple myeloma and is also being explored in combination studies. In 2020, the European Commission and the FDA each granted teclistamab orphan drug designation for the treatment of multiple myeloma.