Leniolisib
Primary Immunodeficiency Diseases
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Drug Status
Government Approvals
US(FDA), UK(BNF)
WHO Essential Medicine
NO
Known Teratogen
No
Pharmaceutical Class
Kinase Inhibitor
Controlled Drug Substance
No
Leniolisib
Summary
Leniolisib is used to treat a genetic condition called activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and children aged 12 years and older. This condition affects the immune system, leading to recurrent infections, swollen lymph nodes, spleen enlargement, and an increased risk of certain types of lymphoma.
Leniolisib works by selectively inhibiting an enzyme called PI3Kdelta that is involved in immune system signaling. By blocking this enzyme, Leniolisib reduces the hyperactivity of the mTOR/Akt pathway, leading to improved regulation of B and T cells and ultimately enhancing immune function in patients with APDS.
The usual daily dose of Leniolisib for both adults and children aged 12 years and older weighing 45 kg or more is 70 mg. It is taken orally, twice daily, approximately 12 hours apart. There is no recommended dosage for patients weighing less than 45 kg.
The most commonly reported side effects of Leniolisib include headache, sinusitis, and atopic dermatitis. Other common side effects are diarrhea, fatigue, pyrexia (fever), back pain, neck pain, and alopecia (hair loss).
Leniolisib may cause fetal harm, so women of reproductive potential should verify their pregnancy status before starting treatment and use effective contraception. Live vaccinations may be less effective during treatment. Leniolisib should not be used in patients with hypersensitivity to the drug or its ingredients. Patients with liver impairment should use it cautiously.
Indications and Purpose
How does Leniolisib work?
Leniolisib works by inhibiting the PI3K-delta enzyme, which is involved in the regulation of the immune system. By blocking this enzyme, Leniolisib reduces the production of PIP3 and the hyperactivity of the mTOR/Akt pathway, leading to improved regulation of B and T cells and better immune function.
Is Leniolisib effective?
The efficacy of Leniolisib was evaluated in a 12-week, placebo-controlled study involving adult and pediatric patients aged 12 years and older with activated phosphoinositide 3-kinase delta syndrome (APDS). The study showed statistically significant improvements in lymphoproliferation and normalization of immunophenotype, demonstrating the effectiveness of Leniolisib in treating APDS.
What is Leniolisib?
Leniolisib is used to treat activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and children aged 12 years and older. It works by inhibiting the PI3K-delta enzyme, which helps regulate the immune system. By blocking abnormal signals, Leniolisib improves immune function and reduces symptoms like recurrent infections and lymph node swelling.
Directions for Use
For how long do I take Leniolisib?
The typical duration of use for Leniolisib is not specified in the provided content. Treatment duration may vary based on individual patient needs and response to the medication. Always follow your doctor's advice regarding the length of treatment.
How do I take Leniolisib?
Leniolisib should be taken orally, twice a day, approximately 12 hours apart, with or without food. There are no specific food restrictions mentioned, but it is important to take the medication at the same times each day for consistency. Always follow your doctor's instructions regarding dosage and administration.
How long does it take for Leniolisib to start working?
Leniolisib reaches steady-state concentrations in the body after approximately 2 to 3 days of treatment. However, the time it takes to notice improvements in symptoms may vary depending on the individual and the condition being treated. Consult your doctor for more personalized information.
How should I store Leniolisib?
Store Leniolisib in its original container, tightly closed, and out of reach of children. Keep it at room temperature, away from excess heat and moisture, and do not store it in the bathroom. Ensure that the container is child-resistant to prevent accidental ingestion by children.
What is the usual dose of Leniolisib?
The usual daily dose of Leniolisib for both adults and children aged 12 years and older, weighing 45 kg or more, is 70 mg taken orally twice a day, approximately 12 hours apart. There is no recommended dosage for patients weighing less than 45 kg.
Warnings and Precautions
Can I take Leniolisib with other prescription drugs?
Leniolisib interacts with strong and moderate CYP3A4 inhibitors, which can increase its exposure, and with strong and moderate CYP3A4 inducers, which can reduce its efficacy. Avoid using Leniolisib with drugs metabolized by CYP1A2 with a narrow therapeutic index, as it inhibits CYP1A2. Consult your doctor for a complete list of potential drug interactions.
Can Leniolisib be taken safely while breastfeeding?
There is no data on the presence of Leniolisib in human milk or its effects on the breastfed infant. Due to the potential for serious adverse reactions in the breastfed child, women are advised not to breastfeed during treatment with Leniolisib and for one week after the last dose.
Can Leniolisib be taken safely while pregnant?
Leniolisib can cause fetal harm based on animal studies, and there is no available data from human studies. Women of reproductive potential should verify pregnancy status before starting treatment and use effective contraception during treatment and for one week after the last dose. If you become pregnant while taking Leniolisib, contact your doctor immediately.
Is Leniolisib safe for the elderly?
There is no specific information on the use of Leniolisib in patients aged 65 and older, as clinical studies did not include this age group. Therefore, it cannot be determined if they respond differently from younger patients. Elderly patients should consult their doctor for personalized advice.
Who should avoid taking Leniolisib?
Leniolisib may cause fetal harm, so it is important to verify pregnancy status before starting treatment. Women of reproductive potential should use effective contraception during treatment and for one week after the last dose. Live vaccinations may be less effective during treatment. Patients with liver problems should consult their doctor, as the use of Leniolisib in moderate to severe hepatic impairment is not recommended.
