What types of cancer can toripalimab treat?

Last updated: 19 March 2024

In 2023, Loqtorzi (toripalimab) got its first FDA approval. This came years after it had already been approved in China as a treatment for nasopharyngeal carcinoma (NPC). Toripalimab may also get its EMA approval soon. So far, only good news for NPC patients.

What about patients with other types of cancer?

Loqtorzi (toripalimab) has been approved in China for the treatment of various types of cancer, with ongoing research to investigate its potential for broader cancer therapy.

Luckily, you don't have to go digging for all the research results. Here's a simple overview of the types of cancer toripalimab may be able to treat. Including the latest trial data, and your options for accessing the medicine quickly.

Toripalimab: nasopharyngeal carcinoma is just a start

Loqtorzi(toripalimab) is a type of immunotherapy known as an immune checkpoint inhibitor. It targets the PD-1 protein on specific immune cells, helping them become more effective at killing cancer cells.

Over the past decade, various immune checkpoint inhibitors have been approved by the FDA in the US. Examples are pembrolizumab, nivolumab, and cemiplimab. All of them have shown effectiveness against multiple types of cancer.

Given this trend, it's not surprising that toripalimab is also being studied for its potential in treating cancers beyond NPC. Supporting this, toripalimab has been approved by China's NMPA for years to treat conditions such as melanoma, non-small cell lung cancer (NSCLC), and esophageal cancer ³.

Currently, Loqtorzi (toripalimab) is FDA-approved specifically for adults with NPC. It's indicated for use either alone as a second-line treatment or in combination with chemotherapy as a first-line treatment ¹.

As more clinical trials unfold, the scope of toripalimab's application may expand in the future. Let's take a look at what we know now.

Can toripalimab be used for NSCLC?

Yes, and it is approved for this indication by the Chinese regulator NMPA ³. More specifically, toripalimab is indicated for use with chemotherapy as a first-line treatment of metastatic non-squamous non-small cell lung cancer (NSCLC) without EGFR or ALK mutations.

Although toripalimab is not yet approved to treat NSCLC in any other country, it does have an FDA orphan drug designation for this indication ⁸. Even before the medicine gets further approvals, there are some China-based clinical trials to give us some insight into its efficacy. Below are some highlights.

CHOICE-01 Phase 3 trial

This trial focused on patients with advanced NSCLC without EGFR/ALK mutations and who had not received previous treatment. In the trial, they received either toripalimab with chemotherapy, or placebo.

The main reported results were:

Median progression-free survival was 8.4 months for the treatment group, compared to 5.6 for the placebo group;
At the time of interim analysis, a median overall survival (OS) had not yet been reached for the toripalimab plus chemo group. Median OS is reached when half of the patients in the group have passed away. At the same moment in time, the median OS for the placebo group was 17.1 months. In short, this means that OS was significantly higher for the patients treated with toripalimab plus chemo ⁴.

LungMate 002

In this phase 2 trial, the safety and efficacy of toripalimab were analyzed, when used as a first-line therapy along with chemo in II-III stage NSCLC patients.

The main reported results were:

76% of patients treated with toripalimab and chemo had their tumors shrink or disappear;
At 12 months after start of treatment, the progression-free survival (PFS) was 76%, and the overall survival (OS) was 90%;
Patients who had surgery had better PFS and OS compared to those who didn't. Specifically, at 12 months, PFS was 42.9% for non-surgery patients and 88.9% for surgery patients. OS was 64.3% for non-surgery patients and 100.0% for surgery patients ⁵.

The study results shared above show a positive perspective of toripalimab in NSCLC treatment. While the medicine is not approved outside of China for this indication, these results could serve to provide insight for medical professionals looking for additional NSCLC treatment options.

Can toripalimab be used for melanoma?

Yes, and this is an approved indication for the medicine in China ³. More specifically, toripalimab is indicated for treating inoperable or metastatic melanoma after failure of other systemic therapy ³. In the USA, the FDA has also granted toripalimab, in combination with axitinib, an orphan drug designation for mucosal melanoma ⁸.

Loqtorzi's approval for melanoma treatment in China was based on the phase 2 POLARIS-01 trial. The main reported outcomes of the trial were:

17.3% of patients treated with toripalimab had their tumors shrink or disappear;
57.5% of patients either responded to the treatment or had a stable disease;
The median progression-free survival was 3.6 months;
The median overall survival was 22.2 months ⁶.

Toripalimab is also being studied for the treatment of melanoma in other settings - e.g. as a first-line treatment in combination with chemotherapy. Multiple studies are ongoing and will hopefully deliver insights into Loqtorzi's full treatment potential in melanoma.

Can toripalimab be used for esophageal cancer?

Yes. In China, toripalimab, in combination with chemotherapy, is approved as a first-line treatment of advanced/recurrent or distant metastatic esophageal squamous cell carcinoma (ESCC) ³. In the USA, toripalimab is designated an orphan drug for this indication ⁸.

In the phase 3 JUPITER-06 study, these survival rate results were reported:

Median overall survival (OS) was 17 months for the toripalimab plus chemo group, as compared to 11 months for the placebo group;
12 months after the start of the trial, the OS rate was 66% for the toripalimab plus chemo group, and 43.7% for the placebo group ⁷.

An additional, phase 2 trial studied toripalimab in combination with chemoradiotherapy as a treatment of advanced ESCC. Some of the reported results were:

62% of the patients who completed the chemoradiotherapy had a complete response;
The median duration of response was 12.1 months;
12 months after the start of treatment, the overall survival rate was 78.4% and the 1-year progression-free survival was 54.5% ⁹.

What other cancers can toripalimab treat?

We've shared a lot of trial results on toripalimab. But there's a lot more.

Additional research is looking at toripalimab in the context of soft tissue sarcoma, lymphoma, renal cell carcinoma, small cell lung cancer, and more ⁶.

Most of the study results for these indications are preliminary. However, the one conclusion we can already safely draw is that toripalimab has a larger role to play in cancer treatment. Time and data will clarify what that role will be. And, hopefully, the orphan drug status of the treatment will help it get approval and reach patients quicker.

Is Loqtorzi(toripalimab) not approved or available in your country for your condition? If you and your doctor are of the opinion that the treatment might benefit you, get in touch with our team of medicine access experts. We can give you personalized support in accessing Loqtorzi before it's locally available.