Helpful Resources About ZTALMY and Living With CDKL5 Deficiency Disorder

• ZTALMY is a prescription medicine used to treat seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in people 2 years of age and older.
• ZTALMY is a federally controlled substance (CV) because it contains ganaxolone that can be abused and lead to dependence. Keep ZTALMY in a safe place to prevent misuse and abuse. Selling or giving away ZTALMY may harm others and is against the law. Tell your healthcare provider if you or your child have ever abused or been dependent on alcohol, prescription medicines or street drugs.
• It is not known if ZTALMY is safe and effective in children under 2 years of age.

ZTALMY can cause serious side effects, including:

ZTALMY may cause sleepiness. Taking ZTALMY with central nervous system (CNS) depressants including alcohol may increase sleepiness. Do not drive, operate heavy machinery, or do other dangerous activities until you know how ZTALMY affects you or your child.

Like other antiepileptic drugs, ZTALMY may cause suicidal thoughts or actions in a very small number of people, about 1 in 500. Call your healthcare provider right away if you or your child have any of these symptoms, especially if they are new, worse, or worry you:

• thoughts about suicide or dying
• attempt to commit suicide
• new or worse depression
• new or worse anxiety
• feeling agitated or restless
• panic attacks
• trouble sleeping (insomnia)
• new or worse irritability
• acting aggressive, being angry, or violent
• acting on dangerous impulses
• an extreme increase in activity and talking (mania)
• other unusual changes in behavior or mood

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 "What is the most important information I should know about ZTALMY?"

The most common side effects of ZTALMY include:

• sleepiness
• fever
• excessive saliva or drooling
• seasonal allergy

These are not all of the possible side effects of ZTALMY. For more information, ask your healthcare provider or pharmacist. Tell your healthcare provider about any side effect that bothers you or your child or that does not go away. Call your healthcare provider for medical advice about side effects.

In the clinical study, ZTALMY was proven effective in people with CDD ages 2 to 19. ZTALMY reduced the median monthly frequency of seizures associated with CDD by 31% vs 7% for placebo. Participants in the clinical study were taking on average 2 to 3 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.

• Read How to Give ZTALMY for information on how to prepare and administer ZTALMY.
• Take or give ZTALMY exactly as your healthcare provider tells you.
• Your healthcare provider will tell you how much ZTALMY to take or give and when to take or give it.
• Measure each dose of ZTALMY using the bottle adapter and oral dosing syringes provided by your pharmacist. Do not use a household teaspoon or tablespoon. If your dose of ZTALMY is less than 1 mL, your pharmacist will provide you with an appropriately sized syringe to take or give ZTALMY.
• Take or give ZTALMY with food.
• In case of overdose, get medical help or contact a live Poison Center expert right away.

ZTALMY is a sugar-free, cherry-flavored liquid, suitable for ketogenic diets.

In the clinical study, 98% of participants taking ZTALMY were also taking other seizure medicines. The most common seizure medicines that were taken were valproate, levetiracetam, clobazam, and vigabatrin.

Your healthcare provider may adjust your child's dose based on the other medicines your child is currently taking.

Tell your healthcare provider about all the medicines you or your child take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Especially tell your healthcare provider if you or your child take:

• alcohol
• opioids
• antidepressants

Know the medicines your child takes. Keep a list of them to show your healthcare provider or pharmacist when your child gets a new medicine.

Refer to the Instructions for Use and Medication Guide. You can also download How to Give ZTALMY to help you prepare and administer ZTALMY. You can also speak to your healthcare provider or pharmacist for help on preparing and giving the prescribed dose.

Speak to your healthcare provider if your child misses a dose.

• Store ZTALMY at 68°F to 77°F (20°C to 25°C)
• Always store ZTALMY in its original bottle in an upright position
• Keep the child-resistant cap tightly closed.
• Use ZTALMY within 30 days of first opening the bottle. Throw away (dispose of) any unused medicine after 30 days.

Keep ZTALMY and all medicines out of reach of children.

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

Active ingredient: ganaxolone.

Inactive ingredients: 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 about ZTALMY. You may use the Doctor Discussion Guide to prepare for a conversation with your healthcare provider.

Once your insurance coverage is confirmed, you will receive a call from a 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.

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.