© Merck KGaA, Darmstadt Germany
The Food and Drug Administration approved pimicotinib, sold as Pimjetmi by EMD Serono, on Tuesday for adults with symptomatic tenosynovial giant cell tumor when removing the tumor through surgery could worsen their ability to function or cause serious harm.
The November 10 approval gives this group of patients another medicine option. It is not an approval for every person with the disease, and the findings do not show that the drug is a cure or that it extends survival.
Tenosynovial giant cell tumor, often shortened to TGCT, is a rare tumor that grows in or around joints. It can cause pain, swelling and trouble moving the affected joint. The company’s approval announcement describes it as locally aggressive: the tumor can damage nearby bone, joints and other tissue.
What the trial found
The FDA based its decision on MANEUVER, an international trial that randomly assigned patients to pimicotinib or a placebo, a treatment without the active medicine. Neither the patients nor the trial investigators knew which treatment patients were receiving during the blinded comparison.
At week 25, the overall response rate was 56% with pimicotinib and 3.2% with placebo, according to an independent review of tumor measurements cited by the FDA. A response means the tumors met the trial’s standards for shrinking or disappearing on scans. It does not mean that 56% of patients were cured.
The trial also found statistically significant improvements in movement of the affected joint, worst pain and physical function compared with placebo. Patients had symptomatic disease that could be measured, including at least one tumor measuring 2 centimeters or more, and surgery posed a risk of dysfunction or serious complications.
Among patients whose tumors responded to pimicotinib, 32, or 91%, had responses lasting at least six months, and 27, or 77%, had responses lasting at least 12 months. The median response duration had not yet been reached in the analysis the FDA cited.
Warnings and daily dose
The recommended dose is 50 milligrams by mouth once a day, with or without food. The FDA said the prescribing information includes warnings for liver toxicity, harm to an embryo or fetus, and a rise in serum creatinine without an effect on kidney function. Creatinine is a substance measured in blood tests used to assess the kidneys.
Common side effects included itching, rash, swelling of the face or around the eyes, fatigue, nausea, headache and swelling in the hands or feet. Changes in several blood-test results were also common. Treatment decisions and monitoring should be discussed with a patient’s healthcare team.
U.S. approval, not an international clearance
The review took place under Project Orbis, an FDA program for working with other drug regulators. Israel’s Ministry of Health was among the participating agencies, alongside regulators in Australia, Canada, Switzerland and the United Kingdom. The European Medicines Agency was an observer.
That collaboration does not establish an Israeli approval. The FDA said applications at other agencies could still be in the submission process or under review. Its announcement did not give a U.S. price or launch date.
JBizNews Desk | Health
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