FDA Grants Orphan Drug Designation to Toripalimab in ES-SCLC


The phase 3 JUPITER-08 trial assessed toripalimab plus chemotherapy, which was granted an orphan drug designation by the FDA, in patients with extensive-stage small cell lung cancer.

Toripalimab (TuoYi) has been granted orphan drug designation by the FDA based on findings 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 included in the ongoing randomized double-blind trial are receiving toripalimab, a PD-1 inhibitor, plus a platinum-based chemotherapeutic such as cisplatin or carboplatin, and etoposide compared with a placebo plus the same chemotherapy backbone as first-line therapy for ES-SCLC.

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

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

The primary end points include progression-free survival (PFS) by investigator assessment and overall survival. The key secondary end points include PFS by RECIST 1.1 criteria assessed by a blinded independent review board, objective response rate, duration of response, and disease control rate.

Eligibility criteria included being 18 years or older with histologically confirmed ES-SCLC who received no prior treatment or immune checkpoint inhibitors for their disease. Additionally, patients needed to be treatment-free for at least 6 months since their last chemotherapy or radiotherapy. Asymptomatic brain metastases that have previously been treated were allowed.

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

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

The FDA has also granted priority review for toripalimab’s biologics license application in recurrent or metastatic nasopharyngeal carcinoma. The prescription drug user fee act has been set for April 2022.

Additionally, toripalimab was the first PD-L1 monoclonal antibody approved in China in 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 therapy; locally advanced urothelial carcinoma after failure of platinum-containing chemotherapy or progression within 12 months of neoadjuvant platinum-containing therapy; and in combination with cisplatin plus gemcitabine as a first-line treatment for patients with locally recurrent or metastatic nasopharyngeal carcinoma.


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

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