Rare Daily Staff

The U.S. Food and Drug Administration granted Breakthrough Therapy designation to Junshi Biosciences’ and Coherus BioSciences’ toripalimab in combination with chemotherapy (gemcitabine and cisplatin) for the first line treatment of recurrent or metastatic nasopharyngeal carcinoma, a cancer that is rare in the United States but more common in certain parts of South Asia, the Middle East, and North Africa.

Breakthrough Therapy designation (BTD) is intended to expedite the development and regulatory review of drugs where preliminary clinical evidence demonstrates substantial improvement over existing therapies for a severe or life-threating disease. Drugs with BTD will be granted closer FDA guidance and various forms of support to avail patients with new therapy as soon as possible. The FDA had earlier granted BTD for toripalimab monotherapy for patients with recurrent or metastatic nasopharyngeal carcinoma (NPC) with disease progression on or after platinum-containing chemotherapy.

Junshi Biosciences expects to complete the biologics license application (BLA) submission for toripalimab plus chemotherapy for first line NPC and for toripalimab monotherapy for second- or third-line NPC later this quarter.

“We are pleased to have received Breakthrough Therapy designation for our novel PD-1 blocking antibody, toripalimab, for nasopharyngeal carcinoma, which is an aggressive cancer with no immuno-oncology treatment options approved in the United States,” said Patricia Keegan, chief medical officer of Junshi Biosciences.

The Breakthrough Therapy designation is supported by data from the phase 3 JUPITER-02 clinical trial evaluating toripalimab in combination with chemotherapy for the first-line treatment of NPC. In this study, toripalimab in combination with chemotherapy demonstrated a statistically significant and clinically meaningful improvement in progression free survival compared to chemotherapy alone assessed by a blinded independent review committee. The trial also met secondary endpoints of progression free survival assessed by the investigator and objective response rate assessed by the blinded independent review committee. There was also a longer duration of response, a higher disease control rate, and higher one- and two-year survival rates for the toripalimab arm. The safety profile of toripalimab is consistent with that observed in previously reported toripalimab clinical trials and the safety profile of this class of drugs.

Toripalimab is an anti-PD-1 monoclonal antibody developed for its ability to block PD-1 interactions with its ligands, PD-L1 and PD-L2, and for enhanced receptor internalization (endocytosis function). Blocking PD-1 interactions with PD-L1 and PD-L2 is thought to recharge the immune system’s ability to attack and kill tumor cells.

More than thirty company-sponsored toripalimab clinical studies covering more than fifteen indications have been conducted globally, including in China and the United States. Pivotal clinical trials are ongoing or completed evaluating the safety and efficacy of toripalimab for a broad range of tumor types including cancers of the lung, nasopharynx, esophagus, stomach, bladder, breast, liver, kidney and skin.

In China, toripalimab was the first domestic anti-PD-1 monoclonal antibody approved for marketing in 2018 for the second-line treatment of unresectable or metastatic melanoma. In December 2020, toripalimab was successfully included in the updated National Reimbursement Drug List.

In the United States, a rolling submission of the first toripalimab Biologics License Application (BLA) is underway for the treatment of recurrent or metastatic NPC. There are currently no PD-1 blocking antibodies indicated for use in NPC in the United States. Additionally, FDA has granted Fast Track status for the development of toripalimab for the treatment of mucosal melanoma and orphan drug designation for NPC, mucosal melanoma, and soft tissue sarcoma. Earlier in 2021 Coherus in-licensed rights to develop and commercialize toripalimab in the United States and Canada. Coherus and Junshi Biosciences plan to file additional toripalimab BLAs with the FDA over the next three years for multiple rare cancers and highly prevalent cancers.

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