ZTALMY and seizure reduction

31 percent seizure reduction with ZTALMY 31 percent seizure reduction with ZTALMY VS 7 percent seizure reduction with placebo 7 percent seizure reduction with placebo

Proven effective in people with CDKL5 deficiency disorder (CDD)

In the clinical study, ZTALMY significantly reduced the monthly seizure frequency over the 17-week treatment period. About half of participants (49) took ZTALMY and about half of participants (51) received placebo. Those taking ZTALMY experienced a 4X reduction in median seizure frequency (31%) compared with placebo (7%).

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1 in 4 experienced at least a 50 percent seizure reduction 1 in 4 experienced at least a 50 percent seizure reduction

Reduced the frequency of seizures by at least half for some study participants

One in 4 participants taking ZTALMY had at least a 50% reduction in frequency of seizures vs 1 in 10 participants taking placebo.

Individual results may vary.

Studied in people with treatment-resistant seizures associated with CDD

  • 101 children and young adults with CDD aged 2 to 19 years participated in the clinical study
  • Participants had previously tried and discontinued between 1 and 16 seizure medicines
  • Participants were taking on average 2 to 3 seizure medicines and were still having frequent seizures
  • Major motor seizure types assessed in the clinical study included atonic or drop, generalized tonic-clonic or focal to bilateral tonic-clonic, bilateral clonic and bilateral tonic

Side effects of ZTALMY

  • In the clinical study, the most common side effects for ZTALMY (those that occurred in ≥5% of participants and twice the rate of placebo) included*:
    • - Sleepiness
    • - Fever
    • - Excessive salivation or drooling
    • - Seasonal allergy
  • 4% of participants taking ZTALMY discontinued treatment due to side effects vs 8% of participants taking placebo
    • - Reasons for discontinuing ZTALMY were sleepiness and seizure (for 1 participant) and seizure (for 1 participant)

*These are not all of the possible side effects of ZTALMY. Call your healthcare provider for medical advice about side effects.

In the clinical study, ZTALMY was used in combination with other seizure medicines

98 percent

of participants taking ZTALMY were also taking other seizure medicines

Most common seizure medicines taken with ZTALMY:

-Valproate -Levetiracetam

-Clobazam -Vigabatrin

Tell your healthcare provider about all the medicines you or your child take, including prescription and over-the counter medicines, vitamins, and herbal supplements. ZTALMY may affect the way other medicines work, and other medicines may affect how ZTALMY works. Do not stop or start taking other medicines without talking to your healthcare provider.

Longer-term safety and seizure data for ZTALMY

Following the 17-week clinical trial, 88 of the 101 participants (87%) chose to enter an open-label extension study which continued to evaluate the safety of ZTALMY for up to 2 years. An open-label study means participants and researchers knew that all participants were being treated with ZTALMY. There was no placebo group. While the main purpose was to continue to study the safety of ZTALMY, seizure frequency was also measured.

Of the 88 participants who initially entered the open-label study, 50 continued in the study through 2 years.

Reasons for discontinuation from the open-label study included lack of efficacy (13 participants), caregiver choice (12 participants), adverse event (10 participants), healthcare provider decision (1 participant), death (1 participant). The death occurred due to sepsis, and investigators assessed that it was unrelated to study drug.

Safety findings were similar to the clinical trial

Data from all 88 participants in the open‑label study was analyzed.

The safety findings were similar to the clinical trial and no new safety concerns were identified.

 

 

The most common side effects reported were:

  • Sleepiness 17% (15/88 participants)
  • Seizure 11% (10/88 participants)
  • Decreased appetite 6% (5/88 participants)
 

Seizure frequency findings were similar to the clinical trial

Data from the 50 participants who continued to take ZTALMY at the 2-year mark were analyzed.

2 years

2 years sustained seizure frequency reduction.

 

Seizure frequency results at 2 years

  • At 2 years (months 22-24), the median reduction in monthly† seizure frequency was 48%
†Monthly = 28 days.
quote

“Once we added ZTALMY to her treatment
plan, we did notice fewer seizures over time. And I’m so glad we made the choice for her.”

— Kelly, mom to Evie, who lives with CDD

Your child may respond differently to ZTALMY.

How ZTALMY works

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.

IMPORTANT SAFETY INFORMATION

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

Sleepiness: 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.

What is ZTALMY?

IMPORTANT SAFETY INFORMATION

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

ZTALMY can cause serious side effects, including:

Sleepiness: 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.

Suicidal Thoughts or Actions: Like all 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:

Pay attention to changes, especially sudden changes in mood, behaviors, thoughts, or feelings. Keep all follow-up visits with your healthcare provider as scheduled.

Stopping ZTALMY: 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).

What should I tell my healthcare provider?

Before taking ZTALMY, tell your healthcare provider about all of your or your child’s medical conditions, including if you or your child:

Tell your healthcare provider about all the medicines you or your child take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. ZTALMY may affect the way other medicines work, and other medicines may affect how ZTALMY works. Do not stop or start taking other medicines without talking to your healthcare provider.

Especially tell your healthcare provider if you or your child take: alcohol; opioids; antidepressants.

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

What should be avoided while taking ZTALMY?

Do not drive, operate heavy machinery, or do other dangerous activities until you know how ZTALMY affects you or your child. ZTALMY may cause you or your child to feel sleepy.

What are the side effects of ZTALMY?

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

The most common side effects of ZTALMY include:

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 doctor for medical advice about side effects. You may report side effects to the FDA at www.fda.gov/medwatch or 1-800-FDA-1088. You may also contact Marinus Pharmaceuticals at 844-627-4687.

For additional information, please click here for Medication Guide and Instructions for Use and discuss with your doctor.