Nirogacestat
Desmoid Tumors
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Drug Status
Government Approvals
US(FDA)
WHO Essential Medicine
NO
Known Teratogen
No
Pharmaceutical Class
NA
Controlled Drug Substance
No
Nirogacestat
Summary
Nirogacestat is used to treat adults with desmoid tumors. These are noncancerous tumors that often occur in the stomach, arms, and legs and cannot be removed by surgery.
Nirogacestat works by inhibiting a protein called gamma secretase. This protein normally signals tumors to grow. By blocking its action, Nirogacestat slows or stops the growth of desmoid tumors.
The usual daily dose for adults is 150 mg taken orally twice a day. This can be either three 50 mg tablets or one 150 mg tablet. The medication should be swallowed whole, without crushing or chewing.
Common side effects of Nirogacestat include diarrhea, nausea, fatigue, rash, and headache. Serious adverse effects can include severe diarrhea, ovarian toxicity, liver toxicity, and nonmelanoma skin cancers.
Nirogacestat can cause severe diarrhea, ovarian toxicity, liver toxicity, nonmelanoma skin cancers, and electrolyte abnormalities. It can also cause fetal harm, so women of reproductive potential should use effective contraception during treatment. The safety and effectiveness of Nirogacestat in children have not been established.
Indications and Purpose
How does Nirogacestat work?
Nirogacestat is a gamma secretase inhibitor that blocks the proteolytic activation of the Notch receptor. This action prevents the activation of pathways that contribute to tumor growth, thereby slowing or stopping the growth of desmoid tumors.
Is Nirogacestat effective?
The efficacy of Nirogacestat was evaluated in a clinical trial (DeFi) involving 142 adult patients with progressing desmoid tumors. The study showed that Nirogacestat significantly improved progression-free survival and objective response rate compared to placebo, indicating its effectiveness in treating desmoid tumors.
What is Nirogacestat?
Nirogacestat is used to treat adults with desmoid tumors, which are noncancerous tumors that often occur in the stomach, arms, and legs. It works by inhibiting gamma secretase, blocking the action of an abnormal protein that signals tumors to grow, thereby slowing or stopping tumor growth.
Directions for Use
For how long do I take Nirogacestat?
Nirogacestat is typically used until disease progression or unacceptable toxicity occurs. The exact duration of use will depend on the individual's response to treatment and the doctor's recommendation.
How do I take Nirogacestat?
Take Nirogacestat orally twice a day, with or without food, at the same times each day. Swallow the tablets whole without crushing or chewing. Avoid consuming grapefruit, starfruit, Seville oranges, and their juices while taking this medication.
How should I store Nirogacestat?
Store Nirogacestat in its original container, tightly closed, at room temperature between 68°F to 77°F (20°C to 25°C). Keep it away from light, excess heat, and moisture, and out of reach of children. Do not store it in the bathroom.
What is the usual dose of Nirogacestat?
The usual daily dose for adults is 150 mg taken orally twice a day. Each dose can be either three 50 mg tablets or one 150 mg tablet. The safety and effectiveness of Nirogacestat in children have not been established, so there is no recommended dose for children.
Warnings and Precautions
Can I take Nirogacestat with other prescription drugs?
Nirogacestat interacts with CYP3A inhibitors and inducers, which can affect its effectiveness and safety. Avoid using it with strong or moderate CYP3A inhibitors like grapefruit products, and inducers like rifampin. It also interacts with gastric acid-reducing agents, which can decrease its effectiveness.
Can Nirogacestat be taken safely while breastfeeding?
Women are advised not to breastfeed during treatment with Nirogacestat and for 1 week after the last dose due to the potential for serious adverse reactions in breastfed children.
Can Nirogacestat be taken safely while pregnant?
Nirogacestat can cause fetal harm based on animal studies. Women of reproductive potential should use effective contraception during treatment and for 1 week after the last dose. A pregnancy test is required before starting treatment. If pregnancy occurs, the medication should be stopped immediately.
Is Nirogacestat safe for the elderly?
There is limited information on the use of Nirogacestat in elderly patients, as clinical studies did not include sufficient numbers of patients aged 65 and older to determine if they respond differently than younger adults. Elderly patients should use this medication under close supervision of their healthcare provider.
Who should avoid taking Nirogacestat?
Important warnings for Nirogacestat include the risk of severe diarrhea, ovarian toxicity, liver toxicity, non-melanoma skin cancers, and electrolyte abnormalities. It can also cause fetal harm, so effective contraception is advised during treatment. There are no specific contraindications, but patients should inform their doctor of any allergies or medical conditions.
