Diacomit

Approved Indications

Diacomit is used to treat seizures associated with Dravet syndrome, a rare and severe form of epilepsy that usually starts in infancy. It is approved for use in adults and children who are at least 2 years old and weigh at least 7 kilograms (approximately 15 pounds).

Combination Therapy

This medication is not used as a standalone treatment. It must be taken in combination with clobazam. In some cases, a doctor may prescribe it alongside other anti-seizure medications as part of a comprehensive treatment plan.

Diacomit is typically added to a patient's existing treatment regimen when seizures are not adequately controlled by other medications alone. It is available as capsules and as a powder for oral suspension.

Mechanism of Action

The exact way Diacomit works to reduce seizures is not fully understood, but researchers believe it functions through two main processes in the body.

Enhancing Brain Chemicals

Diacomit is thought to increase the activity of gamma-aminobutyric acid (GABA). GABA is a neurotransmitter, a type of chemical messenger that helps calm the central nervous system. By increasing the inhibitory effects of GABA, the medication helps prevent the rapid and excessive firing of nerve cells that causes seizures.

Interaction with Other Medications

Diacomit is typically used in combination with other seizure medications, such as clobazam. It works as an enzyme inhibitor, meaning it slows down the liver's ability to break down certain other drugs. This results in higher levels of those medications remaining in the bloodstream for a longer period, which can enhance their effectiveness in controlling seizures.

Because Diacomit changes how the body handles other drugs, healthcare providers must monitor the levels of all seizure medications closely to ensure they remain within a safe and effective range.

Common Side Effects

The most frequently reported side effects of Diacomit include:

• Drowsiness or extreme sleepiness
• Decreased appetite and weight loss
• Nausea and vomiting
• Agitation or irritability
• Difficulty with coordination or balance (ataxia)
• Tremors
• Difficulty sleeping (insomnia)
• Upper respiratory infections, such as bronchitis

Serious Side Effects

While less common, some side effects can be severe. Contact a healthcare provider immediately if you experience:

• Changes in mood or behavior: New or worsening depression, anxiety, panic attacks, or thoughts of self-harm or suicide.
• Blood cell changes: Signs of low white blood cell counts (neutropenia) or low platelet counts (thrombocytopenia), such as frequent infections, fever, sore throat, or unusual bruising and bleeding.
• Liver problems: Symptoms like yellowing of the skin or eyes (jaundice), dark urine, or pain in the upper right stomach area.
• Severe allergic reactions: Rash, hives, or swelling of the face, lips, or tongue.

Sleepiness and Sedation

Diacomit can cause significant drowsiness or sleepiness, which may interfere with daily activities. This effect is more common when the medication is used in combination with clobazam. Healthcare providers may need to adjust the dosage of other medications if excessive sleepiness occurs.

Appetite and Weight Changes

A common concern with this medication is a decrease in appetite and subsequent weight loss. In children, healthcare providers usually monitor weight and growth closely during treatment.

Blood Cell Count Monitoring

This medication can lead to low levels of white blood cells (neutropenia) and platelets (thrombocytopenia). These conditions can increase the risk of infection or bleeding. Regular blood tests are typically performed before starting treatment and every six months during therapy.

Suicidal Thoughts and Behavior

Antiepileptic drugs, including Diacomit, may increase the risk of suicidal thoughts or actions in a small number of people. It is important to monitor for any new or worsening depression, anxiety, or unusual changes in mood or behavior.

Withdrawal and Seizure Risk

Stopping Diacomit abruptly can cause an increase in the frequency of seizures or lead to status epilepticus, a continuous state of seizure that is a medical emergency. The dose should only be changed or stopped under the direction of a healthcare professional, usually through a gradual reduction.

Phenylketonuria (PKU) Warning

The powder for oral suspension contains phenylalanine. This is an important consideration for individuals with phenylketonuria, a genetic disorder where the body cannot process this amino acid.

Drug Interactions

Diacomit can significantly affect how the body processes other medications. It may increase the levels of certain drugs in the blood, potentially leading to increased side effects. A thorough review of all current medications is necessary before starting treatment.

Pregnancy

Information regarding the use of Diacomit during pregnancy is limited. Animal studies have shown that the medication may cause developmental issues or harm to the fetus at doses similar to those used in humans. Because of these potential risks, Diacomit is generally used during pregnancy only when the benefit to the mother justifies the risk to the fetus.

It is important not to start or stop taking seizure medication without consulting a healthcare provider, as uncontrolled seizures can pose risks to both the mother and the fetus. Patients who become pregnant while taking Diacomit are often encouraged to enroll in the North American Antiepileptic Drug (NAAED) Pregnancy Registry. This registry monitors the health of babies born to women who take seizure medications to better understand the drug's effects.

Breastfeeding

It is currently unknown if Diacomit is present in human breast milk, or what effects it may have on a nursing infant or milk production. In animal studies, the drug was found to be excreted in milk. Due to the potential for adverse reactions in the infant, it is important to discuss the risks and benefits of breastfeeding with a healthcare provider while using this medication.

Oral Formulations

Diacomit is supplied in the following formats:

• Capsules: These are intended to be swallowed whole with food and water.
• Powder for oral suspension: This form is provided in individual packets. The powder must be mixed with water to create a liquid suspension and taken immediately after preparation.

FDA Approval and Indications

The U.S. Food and Drug Administration (FDA) approved Diacomit (stiripentol) in 2018. It is specifically indicated for the treatment of seizures associated with Dravet syndrome in patients 2 years of age and older who are taking clobazam. There is no clinical data to support the use of Diacomit as a standalone treatment for Dravet syndrome.

Regulatory Designations

Diacomit received Orphan Drug designation from the FDA. This status is granted to medications intended for the treatment of rare diseases or disorders that affect fewer than 200,000 people in the United States.

Safety Requirements and Monitoring

While Diacomit does not have a Boxed Warning, the FDA requires that the drug be dispensed with a Medication Guide to inform patients about potential serious risks. Like other antiepileptic drugs, Diacomit carries a regulatory warning regarding the risk of suicidal thoughts or behaviors. Additionally, the FDA-approved labeling recommends that healthcare providers monitor blood counts, including white blood cells and platelets, before starting treatment and every six months during therapy due to the risk of neutropenia and thrombocytopenia.

Insurance and Pharmacy Factors

The price of Diacomit can vary based on several factors related to how the medication is obtained:

• Insurance Plans: Coverage levels differ between private insurance, Medicare, and Medicaid. Some plans may require prior authorization from a healthcare provider before they will cover the cost.
• Specialty Pharmacies: Diacomit is typically distributed through specialty pharmacies rather than standard retail pharmacies. These pharmacies coordinate with insurance companies and may offer support services for managing the medication.
• Dosage: The total cost is affected by the prescribed strength and whether the medication is provided as capsules or as a powder for oral suspension.

Financial Support Options

There are often programs available to help manage the expense of this medication:

• Copay Assistance: Manufacturers may offer savings cards for patients with commercial insurance to reduce their out-of-pocket costs.
• Patient Assistance Programs: For those who are uninsured or underinsured, programs may provide the medication at a reduced cost or for free based on financial need and eligibility requirements.

As a brand-name drug without a generic equivalent, the cost remains higher than many older seizure medications. Patients should consult their pharmacist or insurance provider for specific pricing information based on their individual plan.