FDA Grants Breakthrough Therapy Designation to Toripalimab for Nasopharyngeal Carcinoma

The FDA has granted a Breakthrough Therapy designation to toripalimab for the treatment of patients with nasopharyngeal carcinoma, marking the first anti-PD-1 antibody from China to receive this designation, announced Junshi Biosciences, developer of this drug, in a press release.

The receipt of this designation is an important regulatory milestone for Toripalimab following the FDA’s prior Orphan Drug designation in May 2020 for this indication.

“The Breakthrough Therapy designation granted by FDA recognizes the significant clinical benefits toripalimab has shown for the treatment of nasopharyngeal carcinoma,” said Ning Li, MD, PhD, chief executive officer of Junshi Biosciences, in a statement. “Junshi prioritizes cancer types with high prevalence in China such as nasopharyngeal carcinoma, lung and liver cancer, but we found that great unmet medical needs also exist in other countries where patients are endangered by this deadly disease. The BTD will allow us to work with the FDA closely to bring the therapy to patients worldwide expeditiously.”

The supplemental New Drug Application (NDA) for toripalimab injection was accepted by the NMPA, and this application is the world’s first NDA of a monoclonal antibody for the treatment of recurrent or metastatic nasopharyngeal carcinoma.

The phase 3 JUPITER-02 clinical trial (NCT03581786) is evaluating toripalimab injection in combination with chemotherapy as frontline treatment of 280 patients with recurrent or metastatic nasopharyngeal carcinoma. The study has completed enrollment. The primary end point is progression-free survival, and secondary outcomes include overall survival, duration of response, disease control rate, patient-reported outcomes, and health-related quality of life.

The placebo-controlled, multicenter, double-blind study will randomize patients 1:1 to either toripalimab in combination with chemotherapy or placebo with chemotherapy. The chemotherapy regimen includes gemcitabine and cisplatin. Chemotherapy is given every 3 weeks in 3-week cycles.

To be included in this study, patients had to be at least 18 years of age and no older than 75. They had to have a histologically or cytologically confirmed diagnosis of nasopharyngeal carcinoma that is primarily metastatic with at least 1 measurable lesion and a life expectancy of at least 3 months. Patients were excluded from enrollment to the JUPITER-02 study if they had a history of severe hypersensitivity reactions, prior therapy targeting the PD-1 receptor, and a major surgical procedure other than for diagnosis of nasopharyngeal carcinoma within 28 days prior to randomization.

Toripalimab in combination with axitinib (Inlyta) has been granted an Orphan Drug designation by the FDA in March 2020, and the agent also received an Orphan Drug designation for the treatment of patients with nasopharyngeal carcinoma.

The agent previously received approval in China for the second-line treatment of patients with metastatic melanoma in December 2018, and more than 30 clinical trials are evaluating the use of this agent across over 10 different indications in China and the United States, among other countries.

_Reference

_{Junshi biosciences receives FDA breakthrough therapy designation for toripalimab for the treatment of nasopharyngeal carcinoma. News Release. Junshi Biosciences. September 10, 2020. Accessed September 11, 2020. https://bit.ly/3mdr6D0}