The U.S. Food and Drug Administration (FDA) has designated toripalimab (JS001) a breakthrough therapy as a potential treatment of nasopharyngeal carcinoma (NPC), one of the most common cancers in China and one lacking treatment options elsewhere.
This designation is given to medications for serious or life-threatening conditions that have the potential to provide significant advantages over current alternatives, and helps to speed their development, review, and possible approval.
Toripalimab, being developed by Junshi Biosciences, is the first anti-PD-1 antibody coming from China to be given this designation by the FDA; in May, it was granted orphan drug status by the FDA for the same indication.
“The breakthrough therapy designation granted by FDA recognizes the significant clinical benefits toripalimab has shown for the treatment of nasopharyngeal carcinoma,” Ning Li, CEO of Junshi Biosciences, said in a press release.
“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 [designation] will allow us to work with the FDA closely to bring the therapy to patients worldwide expeditiously,” Li said.
Toripalimab is a man-made monoclonal antibody designed to block the activity of PD-1, a protein receptor found on the surface of immune T-cells. By preventing PD-1 from interacting with PD-L1 and PD-L2, which are produced by cancer cells, toripalimab is designed to increase T-cells’ ability to recognize and eliminate cancer cells.
Originally approved in China as a second-line therapy for metastatic melanoma, toripalimab is currently being tested — both alone and in combination with other agents — as a potential treatment for more than 10 other types of cancers, including NPC, in some 30 clinical trials now underway in China, the U.S., and elsewhere.
The National Medical Products Administration (NMPA), the regulatory agency for China, recently agreed to review Junshi’s request for approval of toripalimab to treat advanced NPC in people who failed to respond to at least two prior lines of therapy.
That application was supported by data from the POLARIS-02 Phase 1/2 trial (NCT02915432), which is assessing the safety and preliminary efficacy of toripalimab in patients with advanced NPC and select other cancers who failed to respond to previous systemic treatment.
A Phase 3 trial called JUPITER-02 (NCT03581786) is also evaluating the safety and efficacy of toripalimab, when given alongside chemotherapy, as a first-line therapy to patients with recurrent or metastatic NPC. This trial is recruiting participants in China, Singapore, and Taiwan. For more information on contacts and locations, go here.
