Helpful Resources About ZTALMY and Living With CDKL5 Deficiency Disorder

ZTALMY is a prescription medicine used to treat seizures associated with CDKL5 deficiency disorder (CDD) in people 2 years of age and older. It's the first and only treatment indicated specifically for this use.

ZTALMY is a federally controlled substance (CV) because it contains ganaxolone that can be abused and lead to dependence.

ZTALMY can cause serious side effects, including sleepiness. Taking ZTALMY with central nervous system (CNS) depressants, such as sleep and anxiety medicines, may increase sleepiness.

Like other antiepileptic drugs, ZTALMY may cause suicidal thoughts or actions in a very small number of people, about 1 in 500. Pay attention to changes, especially sudden changes in mood, behaviors, thoughts, or feelings. Keep all follow-up visits with your healthcare provider as scheduled.

Do not stop taking ZTALMY without first talking to your healthcare provider. Stopping ZTALMY suddenly can cause you or your child to have seizures more often or seizures that do not stop (status epilepticus).

See the Medication Guide for more information about serious side effects. Call your healthcare provider between visits as needed, especially if you are worried about symptoms.

The most common side effects of ZTALMY include sleepiness, fever, excessive saliva or drooling, and seasonal allergy.

These are not all the possible side effects of ZTALMY. To learn about other possible side effects, ask your healthcare provider or pharmacist. Tell your healthcare provider about any side effect that bothers your child or that does not go away. If you need medical advice about side effects, talk to your healthcare provider.

ZTALMY was proven effective in a clinical study of 101 people living with CDD, ages 2 to 19. About half of participants (49) took ZTALMY and about half of participants (51) received placebo. ZTALMY reduced the monthly frequency of seizures associated with CDD by a median of 31% vs 7% for placebo. Participants in the clinical study were taking on average 2 to 3 other seizure medicines and were still having frequent seizures.

Individual results may vary.

ZTALMY is the first seizure medicine that is a neuroactive steroid. Exactly how ZTALMY works to treat seizures in CDD is not fully understood, but it is thought to reduce seizures by enhancing the activity of GABA (an inhibitory neurotransmitter) in the brain.

Your healthcare provider will tell you how to give ZTALMY to your child, including how much and when. It’s important to follow their exact instructions. Read the How to Give ZTALMY Guide for information on how to prepare and administer ZTALMY.

In case of an overdose, get medical help or contact a certified Poison Control Center expert right away.

Yes. The ZTALMY cherry-flavored formula is sugar-free, suitable for ketogenic diets.

ZTALMY is approved to be used adjunctively (taken with other antiseizure medications or ASMs) or as monotherapy (taken as single medication). In the clinical study, 98% of participants taking ZTALMY were also taking other seizure medicines. The most common ones were valproate, levetiracetam, clobazam, and vigabatrin. The study did not include every ASM approved for patients with epilepsy.

Tell your healthcare provider about all the medicines your child takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Be sure to tell your healthcare provider if your child takes alcohol, opioids, or antidepressants.

The Medication Guide and Instructions for Use and How to Give ZTALMY Guide can help you understand how to prepare doses and administer ZTALMY. You can also speak to your healthcare provider or the specialty pharmacy for more information on how to give your child ZTALMY.

Speak to your healthcare provider or the specialty pharmacy if your child misses a dose.

Store ZTALMY in its original bottle, in an upright position, and at 68°F to 77°F (20°C to 25°C). Use ZTALMY within 30 days of first opening the bottle, then properly dispose of any unused medicine after 30 days. Always keep ZTALMY and all medicines out of reach of children. Keep the child-resistant caps tightly closed.

Keep ZTALMY in a safe place to prevent misuse and abuse. ZTALMY is a federally controlled substance (CV) because it contains ganaxolone, which can be abused and can lead to dependence.

The active ingredient in ZTALMY is ganaxolone.

Inactive ingredients in ZTALMY are artificial cherry flavor, citric acid, hypromellose, methylparaben, polyvinyl alcohol, propylparaben, purified water, simethicone emulsion, sodium benzoate, sodium citrate, sodium lauryl sulfate, and sucralose.

Talk to your healthcare provider to see if ZTALMY is right for your child. The Doctor Discussion Guide can help you prepare questions and notes about your child’s seizures and ZTALMY.

ZTALMY prescriptions are supplied by ZTALMY One™, a comprehensive support program for you and your child through Orsini Specialty Pharmacy. After your doctor prescribes ZTALMY, a ZTALMY One Patient Care Coordinator will contact you to let you know your prescription is in process.

Once your insurance coverage is confirmed, you will receive a call from your Patient Care Coordinator who will schedule your prescription delivery and discuss any out-of-pocket costs. It’s important to answer calls from ZTALMY One (1-844-982-5691) to avoid delays with your delivery.

To learn more about getting your child's prescription, watch this video.

If you have a question when you receive your child's prescription, you can call ZTALMY One at 1-844-ZTALMY-1 (1-844-982-5691). Our Patient Care Coordinators are available Monday through Friday, 8 am to 8 pm ET, and our pharmacists are available 24/7.

Check out the Getting Started Brochure to learn more about ZTALMY One services, dosing information, and how to give ZTALMY.