Janssen’s Teclistamab Gets FDA Breakthrough Therapy Designation

June 02, 2021 - Janssen Pharmaceuticals of Johnson & Johnson recently announced that FDA has granted Breakthrough Therapy Designation (BTD) for its refractory T-cell therapy for multiple myeloma, teclistamab. 

The bispecific antibody targets both B-cell maturation antigen (BCMA) andCD3 receptors. The therapy  also follows a PRIME (PRIority Medicines) designation from the European Medicines Agency (EMA). The designation was granted in January 2021.  

PRIME designation offers enhanced interaction to improve development plans and advance the evaluation of scientific advances that target a high unmet medical need, a Johnson & Johnson spokesperson said.

The BTD is the eleventh that Janssen’s Oncology Therapeutic Area has received. 

“We are pleased to have received Breakthrough Therapy and PRIME Designations for our novel bispecific antibody, teclistamab,” Peter Lebowitz, MD, PhD, global therapeutic area head of oncology, Janssen Research & Development. 

“This program exemplifies our commitment to advancing science for patients living with multiple myeloma, and it builds upon our robust portfolio in this disease,” Lebowitz continued. 

FDA grants BTD to speed up the development and regulatory review of an investigational medicine that is intended to treat a serious or life-threatening condition.

The agency grants a BTD based on preliminary clinical evidence that shows the drug may have improvement on at least one clinically significant endpoint over available therapy. 

Last year, the European Commission and FDA each granted teclistamab orphan drug designation for the treatment of multiple myeloma.

Orphan drug designation is a special status for a drug or biological product that treats a rare disease or condition. For a drug to quality for orphan drug designation, both the drug and the disease must meet certain criteria specified by FDA.

The BTD and PRIME designations are supported by data from the Phase 1 MajesTEC-1 study. The open-label, multicenter clinical trial evaluated the safety and efficacy of teclistamab in adults with relapsed or refractory multiple myeloma. 

The study enrolled 40 patients with multiple myeloma whose cancer had returned after a median of five other treatments or had not responded to treatment in the first place. Results showed that 58 percent of patients who received teclistamab achieved a very good partial response or better, while 40 percent achieved a complete response or better.

And after more than six months, researchers observed an overall response rate of 65 percent.  

Teclistamab is currently being evaluated in a Phase 2 clinical trial for the treatment of relapsed or refractory multiple myeloma and is also being explored in combination studies.

According to Johnson & Johnson, nearly 35,000 people will be diagnosed with multiple myeloma and over 12,000 people will die from the disease in the US in 2021. 

And while some patients with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms including bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems, or infections. 

At the beginning of April, FDA approved Abecma, the first cell-based gene therapy to treat adult patients with multiple myeloma who have not responded to or whose disease has returned after previous surgery.

Bristol Myers Squibb and bluebird bio’s Abecma is a BCMA-directed, genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy. Each dose of Abecma is a customized treatment created using a patient’s own T-cells. 

The patient’s T-cells are collected and genetically modified to include a new gene that targets and kills myeloma cells. Once the cells are modified, they are infused back into the patient, FDA explained. 

FDA based its approval on a multicenter study of 127 patients with relapsed myeloma. Of all patients, 28 percent showed a complete response or disappearance of all signs of multiple myeloma with Abecma, while 65 percent remained in complete response to the treatment for nearly a year after the study ended.