FDA Grants Orphan Drug Designation for Toripalimab in ES-SCLC

The Phase 3 JUPITER-08 trial evaluated toripalimab plus chemotherapy, which received orphan drug designation from the FDA, in patients with extensive stage small cell lung cancer.

Toripalimab (TuoYi) has received orphan drug designation from the FDA based on results from the Phase 3 JUPITER-08 study (NCT04012606) in patients with extensive stage small cell lung cancer ( ES-SCLC), according to a press release from Coherus Biosciences.

Patients in the ongoing randomized double-blind trial are receiving toripalimab, a PD-1 inhibitor, plus a platinum-based chemotherapy drug such as cisplatin or carboplatin, and etoposide versus placebo plus the same backbone of chemotherapy as the first-line treatment of SE-SCCL.

“Toripalimab in combination with chemotherapy has demonstrated robust antitumor immunity and survival benefit in multiple tumor types, including low PD-L1 expression tumors. This differentiated clinical activity may result from the unique epitope binding and internalization properties of toripalimab,” said Theresa LaVallee, PhD, Chief Development Officer at Coherus, in the press release.

It is estimated that the trial will enroll 420 patients who will receive an injection of toripalimab, or placebo combined with chemotherapy at 240mg/6ml per vial every 3 weeks for up to 2 years of treatment.

Primary endpoints include progression-free survival (PFS) as assessed by the investigator and overall survival. Key secondary endpoints include PFS per RECIST 1.1 criteria assessed by a blinded independent review committee, objective response rate, duration of response, and disease control rate.

Eligibility criteria included being 18 years of age or older with histologically confirmed ES-SCLC and having received no prior therapy or immune checkpoint inhibitors for their disease. In addition, patients had to be without treatment for at least 6 months since their last chemotherapy or radiotherapy. Asymptomatic brain metastases that have already been treated were allowed.

Patients were excluded from the trial if they had previously received systemic treatment for ES-SCLC, prior treatment with a CD137 agonist or immune checkpoint inhibitor, or active central nervous system metastases or untreated.

Toripalimab is an anti-PD-1 monoclonal antibody that was engineered with the ability to block PD-1 interactions via PD-L1 and PD-L2 ligands. This enhances the immune system’s ability to attack and kill tumor cells.

The FDA also granted Priority Review for the Biologics License Application of toripalimab in recurrent or metastatic nasopharyngeal carcinoma. The law on prescription drug user fees has been set for April 2022.

Additionally, toripalimab was the first PD-L1 monoclonal antibody approved in China for 4 indications, including unresectable or metastatic melanoma after failure of standard systemic therapy; recurrent or metastatic nasopharyngeal carcinoma after failure of at least 2 lines of prior treatment; locally advanced urothelial carcinoma after failure of platinum-based chemotherapy or progression within 12 months of neoadjuvant platinum-based therapy; and in combination with cisplatin plus gemcitabine as first-line therapy for patients with locally recurrent or metastatic nasopharyngeal carcinoma.


Coherus and Junshi Biosciences announce that the PD-1 inhibitor toripalimab has been granted orphan drug designation for small cell lung cancer in the United States. Press release. Coherus and Junshi Biosciences. April 14, 2022. Accessed April 25, 2022. https://bit.ly/3vKDtMs

Back To Top