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


The FDA has granted orphan drug designation to toripalimab for the treatment of patients with small cell lung cancer.

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

The designation was based on the phase 3 JUPITER-08 trial (NCT04012606) which is currently examining the PD-1 inhibitor in combination with chemotherapy compared to chemotherapy alone in the first-line for 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,” said Theresa LaVallee, PhD, chief development officer at Coherus, in the press release.

Toripalimab, an anti–PD-1 monoclonal antibody, was designed with the ability to block PD-1 interactions with its ligands, PD-L1 and PD-L2, as well as with the ability 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.

There are currently no PD-1 inhibitors approved by the FDA for the treatment of patients with SCLC.

The ongoing, randomized, multicenter JUPITER-08 trial looks to evaluate toripalimab or placebo in combination with cisplatin or carboplatin plus etoposide in patients with ES-SCLC.

To be eligible for the double-blind, placebo-controlled trial, patients are required to be at least 18 years of age with histologically or cytologically confirmed ES-SCLC. Other enrollment criteria includes an ECOG performance status of 0 or 1, a life expectancy of at least 8 months, at least 1 measurable lesion per RECIST v1.1 criteria, and acceptable hematologic and end-organ function. Additionally, patients must be treatment free for at least 6 months from their last course of chemotherapy or radiotherapy, and those with asymptomatic brain metastases who received previous treatment were eligible.

With an estimated enrollment of 420 participants, those enrolled within the investigative arm received an injection of toripalimab plus chemotherapy every 3 weeks for up to 2 years and those in the control arm received matching placebo plus chemotherapy.

Primary end points of the JUPITER-08 study are investigator-assessed progression-free survival (PFS) and overall survival (OS). Secondary end points include PFS per RECIST v1.1 criteria, objective response rate, duration of response, OS rate, PFS rate, safety, disease control rate, incidence of adverse events, and time to response.

Back in November 2021, the FDA accepted a biologics license application for toripalimab in combination with gemcitabine and cisplatin for first-line treatment of patients with advanced recurrent or metastatic nasopharyngeal carcinoma (NPC) as well as a monotherapy option for patients with recurrent or metastatic NPC after platinum-containing chemotherapy as treatment in the second-line or above.

The FDA also granted fast track designation for toripalimab in January 2021, for the first-line treatment of patients with mucosal melanoma, and orphan drug designations have also been granted to toripalimab for treatment use in esophageal cancer, nasopharyngeal carcinoma, and soft tissue sarcoma.

“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,” added Patricia Keegan, MD, chief medical officer of Junshi Biosciences, 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.”

  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 22, 2022. https://bit.ly/3K0mqLy
  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 22, 2022. https://clinicaltrials.gov/ct2/show/NCT04012606
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