Toripalimab, sold under the brand name Loqtorzi, is a

monoclonal antibody A monoclonal antibody (mAb, more rarely called moAb) is an antibody produced from a cell lineage made by cloning a unique white blood cell. All subsequent antibodies derived this way trace back to a unique parent cell. Monoclonal antibodie ...

used for the treatment of melanoma and nasopharyngeal carcinoma. Toripalimab is a recombinant humanized

programmed cell death protein 1 Programmed cell death protein 1 (PD-1), (CD279 cluster of differentiation 279). PD-1 is a protein encoded in humans by the ''PDCD1'' gene. PD-1 is a cell surface receptor on T cells and B cells that has a role in regulating the immune system's re ...

(PD-1)

that acts as a

checkpoint inhibitor Checkpoint inhibitor therapy is a form of Treatment of cancer, cancer immunotherapy. The therapy targets immune checkpoints, key regulators of the immune system that when stimulated can dampen the immune response to an immunologic stimulus. Some ca ...

. In 2018, toripalimab was approved in China for the treatment of unresectable or metastatic

melanoma Melanoma is the most dangerous type of skin cancer; it develops from the melanin-producing cells known as melanocytes. It typically occurs in the skin, but may rarely occur in the mouth, intestines, or eye (uveal melanoma). In very rare case ...

that has failed previous systemic therapy. In October 2023, the US

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respo ...

(FDA) approved toripalimab for the first-line treatment of adults with metastatic or recurrent, locally advanced

nasopharyngeal carcinoma Nasopharyngeal carcinoma (NPC), or nasopharynx cancer, is the most common cancer originating in the nasopharynx, most commonly in the postero-lateral nasopharynx or pharyngeal recess (fossa of Rosenmüller), accounting for 50% of cases. NPC occurs ...

when used with

cisplatin Cisplatin is a chemical compound with chemical formula, formula ''cis''-. It is a coordination complex of platinum that is used as a chemotherapy medication used to treat a number of cancers. These include testicular cancer, ovarian cancer, c ...

and

gemcitabine Gemcitabine, sold under the brand name Gemzar, among others, is a chemotherapy medication used to treat cancers. It is used to treat testicular cancer, breast cancer, ovarian cancer, non-small cell lung cancer, pancreatic cancer, and bladder ca ...

Medical uses

Toripalimab is

indicated In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis ...

in combination with cisplatin and gemcitabine for the first-line treatment of adults with metastatic or recurrent locally advanced

. It is also indicated as a single agent for adults with recurrent unresectable or metastatic nasopharyngeal carcinoma with disease progression on or after a platinum-containing chemotherapy.

History

Efficacy of toripalimab with cisplatin and gemcitabine was evaluated in JUPITER-02 (NCT03581786), a randomized, multicenter, single region, double-blind, placebo-controlled trial in 289 participants with metastatic or recurrent, locally advanced nasopharyngeal carcinoma who had not received previous systemic chemotherapy for recurrent or metastatic disease. Participants were randomized (1:1) to either toripalimab with cisplatin and gemcitabine, followed by toripalimab, or placebo with cisplatin and gemcitabine, followed by placebo. Efficacy of toripalimab as a single agent was evaluated in POLARIS-02 (NCT02915432), an open-label, multicenter, single country, multicohort trial in 172 participants with unresectable or metastatic nasopharyngeal carcinoma who had received prior platinum-based chemotherapy or had disease progression within six months of completion of platinum-based chemotherapy administered as neoadjuvant, adjuvant, or definitive chemoradiation treatment for locally advanced disease. Participants received toripalimab until disease progression per RECIST v1.1 or unacceptable toxicity. The FDA granted the application for toripalimab

priority review Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease. The priority review voucher program ...

breakthrough therapy Breakthrough therapy is a United States Food and Drug Administration designation that expedites drug development that was created by Congress under Section 902 of the 9 July 2012 Food and Drug Administration Safety and Innovation Act. The FDA's "b ...

, and

orphan drug An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ...

designations.

Society and culture

Legal status

In July 2024, the

Committee for Medicinal Products for Human Use The Committee for Medicinal Products for Human Use (CHMP), formerly known as the Committee for Proprietary Medicinal Products (CPMP), is the European Medicines Agency's committee responsible for elaborating the agency's opinions on all issues regar ...

(CHMP) of the

European Medicines Agency The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of pharmaceutical products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products ...

adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Loqtorzi, intended for the treatment of nasopharyngeal carcinoma and esophageal squamous cell carcinoma. The applicant for this medicinal product is TMC Pharma (EU) Limited. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. Loqtorzi was authorized for medical use in the European Union in September 2024.

References

External links

* * {{Authority control Antineoplastic drugs Monoclonal antibodies Orphan drugs