FDA Grants Orphan Drug Designation to Toripalimab for Small Cell Lung Cancer


The FDA has granted an orphan drug designation to the PD-1 inhibitor toripalimab as a potential therapeutic option for patients with small cell lung cancer.

The FDA has granted an orphan drug designation to the PD-1 inhibitor toripalimab as a potential therapeutic option for patients with small cell lung cancer (SCLC), according to an announcement from Coherus BioSciences, Inc. and Shanghai Junshi Biosciences Co., Ltd.1

The phase 3 JUPITER-08 trial (NCT04012606) is currently evaluating the PD-1 inhibitor in combination with chemotherapy compared with chemotherapy alone as a first-line treatment in patients with extensive-stage SCLC (ES-SCLC).2

“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, stated in a press release. “We are pleased to be working closely with our partner, Junshi Biosciences, to evaluate toripalimab in this underserved patient population and look forward to topline data from the pivotal first-line SCLC clinical trial expected later this year.”

No PD-1 inhibitors are currently FDA approved for the treatment of patients with SCLC. Toripalimab is an anti–PD-1 monoclonal antibody that was designed to block PD-1 interactions with its ligands, PD-L1 and PD-L2, and to enhance receptor internalization. Blocking interactions of PD-1 with PD-L1 and PD-L2 increase the ability of the immune system to attack and eliminate tumor cells.

The ongoing, randomized, double-blind, placebo-controlled, multicenter JUPITER-08 trial is evaluating toripalimab or placebo in combination with cisplatin or carboplatin plus etoposide in patients with histologically or cytologically confirmed ES-SCLC who are at least 18 years of age and who have an ECOG performance status of 0 or 1.

Patients were required to be treatment free for at least 6 months from their last course of chemotherapy or radiotherapy. They also needed to have a life expectancy of at least 8 weeks, at least 1 measurable lesion per RECIST v1.1 criteria, and acceptable hematologic and end-organ function. Notably, those with asymptomatic brain metastases who received previous treatment were permitted.

If patients received prior systemic treatment or immune checkpoint inhibitors, they were excluded. Patients also could not have active or untreated central nervous system metastases, spinal cord compression that was not definitively treated with surgery and/or radiation, cancerous meningitis, uncontrolled or symptomatic hypercalcemia, or other malignant tumors within 5 years prior to the first does of study treatment, among other criteria.

Patients in the investigative arm received a toripalimab injection plus chemotherapy every 3 weeks for up to 2 years. Those in the control arm received matching placebo plus chemotherapy.

The primary end points of the study were investigator-assessed progression-free survival (PFS) and overall survival (OS). Secondary end points were PFS per RECIST v1.1 criteria, objective response rate, duration of response, disease control rate, time to response, OS rate, PFS rate, and safety.

“For non–small cell lung cancer without oncogenic mutations, multiple immuno-oncology drugs, including toripalimab, have been shown to improve survival when added to chemotherapy as compared to chemotherapy alone, whereas treatment options for SCLC patients are limited to chemotherapy with one of two PD-L1 inhibitors,” Patricia Keegan, MD, chief medical officer of Junshi Biosciences, added in the press release “We appreciate the FDA’s recognition of our endeavors to develop new therapies for SCLC patients and, based on experience in other cancers, are hopeful that toripalimab may provide a significant advance over chemotherapy in the JUPITER-08 study.”

In November 2021, the FDA granted priority review to a biologics license application for toripalimab in combination with gemcitabine and cisplatin in the frontline treatment of patients with advanced recurrent or metastatic nasopharyngeal carcinoma, and for use as a single agent in the second- or later-line treatment of patients with recurrent or metastatic nasopharyngeal carcinoma following platinum-containing chemotherapy.3

Additionally, in January 2021, the FDA granted fast track designation for toripalimab for the treatment of mucosal melanoma.4 Orphan drug designations have also been granted to toripalimab in esophageal cancer, nasopharyngeal carcinoma, and soft tissue sarcoma.


  1. 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 Biosciences, Inc. April 14, 2022. Accessed April 21, 2022. https://bit.ly/3v3XlLv
  2. Toripalimab in combination with platinum plus etoposide in patients with extensive-stage small cell lung cancer. ClinicalTrials.gov. Updated September 30, 2020. Accessed April 21, 2022. https://clinicaltrials.gov/ct2/show/NCT04012606
  3. Coherus and Junshi Biosciences announce FDA acceptance of BLA filing for toripalimab for treatment of nasopharyngeal carcinoma. News release. Coherus BioSciences, Inc. and Shanghai Junshi Biosciences Co., Ltd. November 1, 2021. Accessed April 21, 2022. https://bit.ly/3CwoLeh
  4. FDA grants toripalimab fast track designation for mucosal melanoma. News release. Junshi Biosciences. January 24, 2021. Accessed April 21, 2022. http://bit.ly/369BENm
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