Ganaxolone
Generic Name: ganaxolone
Brand Names:
Ztalmy
11 DESCRIPTION ZTALMY (ganaxolone) oral suspension contains ganaxolone, a neuroactive steroid gamma-aminobutyric acid A (GABA A ) receptor positive modulator. Ganaxolone (1-[(3R, 5S, 8R, 9S, 10S, 13S, 14S, 17S)-3-hydroxy-3, 10, 13-trimethyl-1, 2, 4, 5, 6, 7, 8, 9, 11, 12, 14, 15, 16, 17-tetradecahydrocyclopenta[a]phenanthren-17-yl]ethanone) is a methyl-substituted (at the 3β position) analog of the endogenous neurosteroid allopregnanolone, a derivative of progesterone.
Overview
11 DESCRIPTION ZTALMY (ganaxolone) oral suspension contains ganaxolone, a neuroactive steroid gamma-aminobutyric acid A (GABA A ) receptor positive modulator. Ganaxolone (1-[(3R, 5S, 8R, 9S, 10S, 13S, 14S, 17S)-3-hydroxy-3, 10, 13-trimethyl-1, 2, 4, 5, 6, 7, 8, 9, 11, 12, 14, 15, 16, 17-tetradecahydrocyclopenta[a]phenanthren-17-yl]ethanone) is a methyl-substituted (at the 3β position) analog of the endogenous neurosteroid allopregnanolone, a derivative of progesterone.
Uses
1 INDICATIONS AND USAGE ZTALMY is indicated for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older. ZTALMY is a neuroactive steroid gamma-aminobutyric acid (GABA) A receptor positive modulator indicated for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older. ( 1 )
Dosage
2 DOSAGE AND ADMINISTRATION Administer ZTALMY orally three times daily with food. ( 2.1 ) Titrate ZTALMY gradually according to the recommended schedules. See full prescribing information. ( 2.1 ) Dosage for patients weighing 28 kg or less ( 2.1 ): the starting dosage is 2 mg/kg three times daily (6 mg/kg/day) the maximum dosage is 21 mg/kg three times daily (63 mg/kg/day). Dosage for patients weighing over 28 kg ( 2.1 ): the starting dosage is 50 mg three times daily (150 mg daily) the maximum dosage is 600 mg three times daily (1800 mg daily). Patients with severe hepatic impairment: see full prescribing information for dosage recommendation. ( 2.3 ) 2.1 Dosage Information ZTALMY is administered by mouth three times daily and must be taken with food [see Clinical Pharmacology (12.3) ] .
Side Effects
6 ADVERSE REACTIONS The following important adverse reactions are described elsewhere in the labeling: Somnolence and Sedation [see Warnings and Precautions (5.1) ] Suicidal Behavior and Ideation [see Warnings and Precautions (5.2) ] Withdrawal of Antiepileptic Drugs [see Warnings and Precautions (5.3) ] Most common adverse reactions (incidence of at least 5% for ZTALMY and at least twice the rate of placebo) are somnolence, pyrexia, salivary hypersecretion, and seasonal allergy. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Immedica at 1-844-627-4687 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Interactions
7 DRUG INTERACTIONS UGT inhibitors may increase ganaxolone exposures; consider reduction of a stable ZTALMY dosage when initiating a UGT inhibitor. ( 7.1 ) Cytochrome P450 inducers will decrease ganaxolone exposure. It is recommended to avoid concomitant use with strong or moderate CYP3A4 inducers; if unavoidable, consider a dosage increase of ZTALMY, but do not exceed the maximum recommended dosage. ( 7.2 ) 7.1 Effect of UGT Inhibitors on ZTALMY Concomitant use of ZTALMY and UGT inhibitors (e.g., valproic acid) may increase the exposure of ganaxolone, which may increase the risk of ZTALMY associated adverse reactions in patients who have titrated to a stable ZTALMY dosage.
Warnings
5 WARNINGS AND PRECAUTIONS Somnolence and Sedation: Monitor for somnolence and sedation and advise patients not to drive or operate machinery until they have gained sufficient experience with ZTALMY. Concomitant use with other CNS depressants or alcohol could potentiate adverse effects. ( 5.1 ) Suicidal Behavior and Ideation: Monitor patients for suicidal behavior and thoughts. ( 5.2 ) Withdrawal of Antiepileptic Drugs: ZTALMY should be withdrawn gradually to minimize the risk of increased seizure frequency and status epilepticus. ( 5.3 ) 5.1 Somnolence and Sedation ZTALMY can cause somnolence and sedation. In Study 1 [see Clinical Studies (14) ] , the incidence of somnolence and sedation was 44% in patients treated with ZTALMY, compared with 24% in patients receiving placebo. 4 CONTRAINDICATIONS None. None. ( 4 )
Pregnancy
8.1 Pregnancy Pregnancy Exposure Registry There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to antiepileptic drugs (AEDs), such as ZTALMY, during pregnancy. Encourage women who are taking ZTALMY during pregnancy to enroll in the North American Antiepileptic Drug (NAAED) Pregnancy Registry by calling the toll-free number 1-888-233-2334 or visiting http://www.aedpregnancyregistry.org/. Risk Summary There are no available data on ZTALMY use in pregnant women to inform a drug-associated risk of adverse developmental outcomes.
Storage
16.2 Storage and Handling Store ZTALMY in its original bottle in an upright position at 20°C to 25°C (68°F to 77°F); excursions permitted from 15°C to 30°C (59°F to 86°F) [see USP Controlled Room Temperature]. Keep the cap tightly closed. Use within 30 days of first opening the bottle, then discard any remainder.
Frequently Asked Questions
What is Ganaxolone used for?▼
1 INDICATIONS AND USAGE ZTALMY is indicated for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older. ZTALMY is a neuroactive steroid gamma-aminobutyric acid (GABA) A receptor positive modulator indicated for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older. ( 1 )
What are the side effects of Ganaxolone?▼
6 ADVERSE REACTIONS The following important adverse reactions are described elsewhere in the labeling: Somnolence and Sedation [see Warnings and Precautions (5.1) ] Suicidal Behavior and Ideation [see Warnings and Precautions (5.2) ] Withdrawal of Antiepileptic Drugs [see Warnings and Precautions (5.3) ] Most common adverse reactions (incidence of at least 5% for ZTALMY and at least twice the rate of placebo) are somnolence, pyrexia, salivary hypersecretion, and seasonal allergy. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Immedica at 1-844-627-4687 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Can I take Ganaxolone during pregnancy?▼
8.1 Pregnancy Pregnancy Exposure Registry There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to antiepileptic drugs (AEDs), such as ZTALMY, during pregnancy. Encourage women who are taking ZTALMY during pregnancy to enroll in the North American Antiepileptic Drug (NAAED) Pregnancy Registry by calling the toll-free number 1-888-233-2334 or visiting http://www.aedpregnancyregistry.org/. Risk Summary There are no available data on ZTALMY use in pregnant women to inform a drug-associated risk of adverse developmental outcomes.
What are the important warnings for Ganaxolone?▼
5 WARNINGS AND PRECAUTIONS Somnolence and Sedation: Monitor for somnolence and sedation and advise patients not to drive or operate machinery until they have gained sufficient experience with ZTALMY. Concomitant use with other CNS depressants or alcohol could potentiate adverse effects. ( 5.1 ) Suicidal Behavior and Ideation: Monitor patients for suicidal behavior and thoughts. ( 5.2 ) Withdrawal of Antiepileptic Drugs: ZTALMY should be withdrawn gradually to minimize the risk of increased seizure frequency and status epilepticus. ( 5.3 ) 5.1 Somnolence and Sedation ZTALMY can cause somnolence and sedation. In Study 1 [see Clinical Studies (14) ] , the incidence of somnolence and sedation was 44% in patients treated with ZTALMY, compared with 24% in patients receiving placebo. 4 CONTRAINDICATIONS None. None. ( 4 )
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Medical Disclaimer
This drug information is for educational purposes only and should not replace professional medical advice. Drug information is sourced from the FDA National Drug Code Directory and Structured Product Labeling. Always consult with a healthcare provider before starting, stopping, or changing any medication.