The First Seizure Medicine Specifically for People With CDKL5 Deficiency Disorder Aged 2 Years and Older

Evie is living with CDD.

ZTALMY and seizure reduction

31 percent seizure reduction with ZTALMY

Proven effective in people 2 years and older 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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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 between 1 and 4 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

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 loved one 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 efficacy, safety, and tolerability 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.

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.

People treated with ZTALMY experienced a sustained reduction in seizure frequency.

Seizure frequency results at 2 years

At 2 years (months 22-24), the median reduction in monthly^† seizure frequency was 48%

^†Monthly = 28 days.

“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 loved one 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.

The body of the nerve cell, and the tentacle-like nerve cell endings connected to other cells

The role of GABA

The exact cause of seizures in CDD, like in other epilepsies, is not fully known. Seizures may result from an imbalance of excitatory (stimulating) and inhibitory (calming) signals in the brain.
GABA, the main inhibitory neurotransmitter, plays an important role in sending inhibitory signals to calm excited neurons (nerve cells). When these signals do not work as they should, neurons may become overexcited, which can lead to seizures.

GABA receptors on the neuron where ZTALMY binds

The ZTALMY difference

GABA sends inhibitory signals by attaching to certain GABA receptors found on neurons. Depending on their location, some of these receptors produce short bursts of inhibition, while others produce continuous inhibition.
ZTALMY is a different kind of seizure medicine that enhances both types of inhibition by working on certain GABA receptors in different locations on the neuron. This dual approach may help to calm overexcited neurons.

Program Eligibility and Terms and Conditions

The copay savings program requires a valid prescription for ZTALMY® (ganaxolone) oral suspension, CV consistent with the Prescribing Information.

Patient must have commercial insurance. Offer is not valid for cash-paying patients.

Patient must reside in the U.S. or U.S. Territory.

Patients are responsible for as low as a zero-dollar copayment for a ZTALMY prescription for a maximum 30-day supply per fill. The value of the copay savings program offer is limited to an annual maximum per calendar year. For maximum annual benefit limit information, please contact ZTALMY One™ (1‑844‑982‑5691).

Patients are not eligible to use this offer if they are eligible for or enrolled in a state or federally funded insurance program, including but not limited to Medicare, Medicaid, TRICARE, Veteran Affairs health care or a state prescription drug assistance program.

Patients are responsible for reporting use of the copay savings program to any commercial insurer, health plan, or other third party who pays for or reimburses any part of the prescription filled using the copay savings program, as may be required. Patients should not use the copay savings program if their insurer or health plan prohibits use of manufacturer copay programs.

Offer not valid where prohibited by law.

Copay Savings Program accepted only by the ZTALMY One designated specialty pharmacy.

If patient’s insurance changes, the pharmacy must be notified immediately. Based on patient’s insurance change, patient may no longer be able to participate in the copay savings program.

Offer is not health insurance.

Offer good only in the U.S.

Offer is limited to one per person during this offering period and is not transferable.

Offer is not conditioned on any past, present, or future purchase, including refills.

Immedica Pharma US Inc. reserves the right to rescind, revoke or amend this offer and program eligibility requirements at any time without notice.