What is it prescribed for?
PENPULIMAB-KCQX is prescribed for adults with a type of head and neck cancer called non-keratinizing nasopharyngeal carcinoma (NPC). This cancer starts in the upper part of the throat, behind the nose.
It can be used:
- With chemotherapy (cisplatin or carboplatin plus gemcitabine) as the first treatment when the cancer has come back (recurrent) or spread to other parts of the body (metastatic).
- Alone (single drug) for patients whose cancer has spread and did not improve after platinum-based chemotherapy and at least one other type of treatment.
This medication was approved by the FDA on April 23, 2025.
What is the name of the drug and what does it do?
The medicine is called PENPULIMAB-KCQX. It belongs to a group of drugs known as immune checkpoint inhibitors.
It helps the body’s own immune system fight cancer cells. By removing the “brakes” that cancer puts on the immune system, this medicine allows immune cells to attack the tumor.
How does it work?
PENPULIMAB-KCQX blocks a pathway in the body called PD-1/PD-L1. Normally, this pathway helps keep the immune system from attacking healthy cells. But many cancers use this same pathway to hide from immune attack.
By blocking PD-1, PENPULIMAB-KCQX:
- “Releases the brakes” on immune T-cells
- Helps these immune cells recognize and destroy cancer cells
- Can slow tumor growth
What did the research discover?
Two main research studies were done:
- First-line treatment study (AK105-304)
- Compared PENPULIMAB-KCQX + chemotherapy to placebo + chemotherapy.
- Patients taking PENPULIMAB-KCQX had a longer time before their cancer worsened (median 9.6 months vs. 7 months).
- The risk of the cancer progressing was reduced by about 55% (see Kaplan-Meier graph on page 20).
- Later-line treatment study (AK105-202)
- Tested PENPULIMAB-KCQX alone in patients whose cancer kept growing after multiple treatments.
- About 28% of patients saw their tumors shrink.
- Nearly half of those responses lasted more than 1 year .
What are some of the side effects?
Because PENPULIMAB-KCQX activates the immune system, it can sometimes cause it to attack normal organs. These are called immune-related side effects.
Common side effects (when used with chemotherapy) include:
- Nausea and vomiting
- Low thyroid hormone (hypothyroidism)
- Constipation
- Tiredness
- Rash
- Fever
- Decreased appetite and weight loss
When used alone, the most common side effects are:
- Anemia (low red blood cells)
- Hypothyroidism
Serious but less common risks:
- Lung problems (pneumonitis)
- Liver inflammation (hepatitis)
- Severe diarrhea (colitis)
- Diabetes or other hormone gland problems
- Severe skin reactions
- Kidney problems
Pregnant women must not use this drug because it can cause harm to unborn babies. Breastfeeding is also not recommended.
What are the dosage recommendations and how is it prescribed?
- With chemotherapy (first-line treatment):
- 200 mg IV (through the vein) every 3 weeks, over 60 minutes.
- Given together with gemcitabine + cisplatin or carboplatin.
- Treatment can continue up to 24 months, unless the cancer worsens or side effects are too severe.
- Alone (after other treatments):
- 200 mg IV every 2 weeks, over 60 minutes.
- Also continued up to 24 months if tolerated.
No dose reduction is recommended, but doctors may pause or permanently stop the drug if serious side effects occur.
Source:
PENPULIMAB-KCQX injection, for intravenous use [prescribing information]. Zhongshan (China): Akeso Biopharma Co., Ltd.; 2025 Apr. Accessed by: https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/761258Orig1s000correctedlbl.pdf
