Palovarotene
Heterotopic Ossification
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Drug Status
Government Approvals
US(FDA)
WHO Essential Medicine
NO
Known Teratogen
No
Pharmaceutical Class
Retinoid
Controlled Drug Substance
No
Palovarotene
Summary
Palovarotene is used to treat fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder causing abnormal bone formation in muscles and soft tissues.
Palovarotene is a retinoid that acts as a retinoic acid receptor agonist. It inhibits a signaling pathway responsible for abnormal bone growth, helping manage FOP symptoms and slow disease progression.
The usual daily dose for adults and children aged 14 years and older is 5 mg. For younger children, the dose is weight-based, ranging from 2.5 mg to 5 mg daily. It's taken orally, preferably with food.
Common side effects include dry skin, dry lips, joint pain, itching, and rash. Serious side effects can include mood changes, night blindness, and premature closure of growth plates in bones.
Palovarotene is contraindicated during pregnancy due to risk of fetal harm. It can affect bone growth in children. It should not be used with strong or moderate CYP3A4 inhibitors or inducers, or with vitamin A supplements. It can cause mood changes and night blindness.
Indications and Purpose
How does Palovarotene work?
Palovarotene is a retinoid that acts as a retinoic acid receptor agonist, specifically targeting the gamma subtype. It reduces abnormal bone formation by inhibiting the BMP/ALK2 signaling pathway, which is responsible for heterotopic ossification in fibrodysplasia ossificans progressiva (FOP). This action helps manage symptoms and slow the progression of the disease.
Is Palovarotene effective?
Palovarotene has been shown to reduce the volume of new heterotopic ossification in patients with fibrodysplasia ossificans progressiva (FOP). Clinical studies have demonstrated its effectiveness in managing symptoms and reducing the formation of new bone in soft tissues. However, it is important to note that Palovarotene does not cure FOP but helps control its progression.
What is Palovarotene?
Palovarotene is used to reduce new bone formation in patients with fibrodysplasia ossificans progressiva (FOP). It belongs to a class of medications called retinoids and works by inhibiting the signaling pathway responsible for abnormal bone growth. While it helps manage symptoms, it does not cure FOP. Always follow your doctor's instructions when using this medication.
Directions for Use
For how long do I take Palovarotene?
Palovarotene is used as a long-term treatment to manage symptoms of fibrodysplasia ossificans progressiva (FOP). The duration of use depends on the individual's condition and response to treatment. It is important to follow your doctor's guidance and not stop taking the medication without consulting them.
How do I take Palovarotene?
Take Palovarotene with food, preferably at the same time each day. Swallow the capsules whole, or if unable, open them and sprinkle the contents on a teaspoon of soft food like applesauce or yogurt, and consume within one hour. Avoid grapefruit, pomelo, or juices containing these fruits, as they may interact with the medication.
How should I store Palovarotene?
Store Palovarotene in its original container, tightly closed, at room temperature away from excess heat and moisture. Keep it out of reach of children. Do not store it in the bathroom. If the capsules are opened and sprinkled on food, consume them within one hour, ensuring they are not exposed to direct sunlight.
What is the usual dose of Palovarotene?
For adults and children aged 14 years and older, the usual daily dose of Palovarotene is 5 mg. For children aged 8 to 13 years for females and 10 to 13 years for males, the dose is weight-based, ranging from 2.5 mg to 5 mg daily. Always follow your doctor's specific dosage instructions.
Warnings and Precautions
Can I take Palovarotene with other prescription drugs?
Palovarotene should not be used with strong or moderate CYP3A4 inhibitors, as they can increase the risk of side effects. Avoid using it with strong CYP3A4 inducers, which can reduce its effectiveness. Additionally, avoid taking vitamin A supplements or other retinoids, as they can lead to hypervitaminosis A. Always inform your doctor about all medications and supplements you are taking.
Can Palovarotene be taken safely while breastfeeding?
There is no data on the presence of Palovarotene in human milk or its effects on the breastfed infant. Due to the potential for serious adverse reactions in breastfed infants, it is recommended that females do not breastfeed during treatment with Palovarotene and for at least one month after the final dose. Consult your healthcare provider for personalized advice.
Can Palovarotene be taken safely while pregnant?
Palovarotene is contraindicated during pregnancy due to the risk of fetal harm, including birth defects. It is a retinoid, a class of drugs known to cause teratogenic effects. Females of reproductive potential should use effective contraception starting at least one month before treatment, during treatment, and for one month after the last dose. If pregnancy occurs, discontinue Palovarotene immediately and consult a healthcare provider.
Is it safe to exercise while taking Palovarotene?
Palovarotene does not specifically limit the ability to exercise. However, it can cause side effects like joint pain, muscle pain, and fatigue, which might affect physical activity. If you experience these symptoms, consult your doctor for advice on managing them and safely continuing your exercise routine.
Is Palovarotene safe for the elderly?
There is no specific information on the use of Palovarotene in the elderly. However, as with any medication, elderly patients should use Palovarotene with caution, starting at the lower end of the dosing range. This is due to the potential for decreased liver, kidney, or heart function and the presence of other medical conditions or medications. Always consult a healthcare provider for personalized advice.
Who should avoid taking Palovarotene?
Palovarotene is contraindicated in pregnancy due to the risk of fetal harm. It can cause premature epiphyseal closure in growing children, affecting bone growth. Patients should avoid donating blood during treatment and for one week after. Palovarotene may cause mood changes, night blindness, and skin sensitivity to sunlight. Always consult your doctor for personalized advice and follow their instructions carefully.
