UCB presents late-breaking posters at Child Neurology Society Meeting

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Oct 04, 2023, 13:06 ET

ATLANTA, Oct. 4, 2023 /PRNewswire/ -- The Child Neurology Society Meeting, a prominent event in the field of pediatric neurology, is set to showcase cutting-edge research and developments in child neurology. As part of this conference, UCB will present two late-breaking posters that shed light on the care and management of Dravet syndrome.

Poster 1: Outcomes Among Individuals With Dravet Syndrome Using Fenfluramine: A Retrospective Analysis Using U.S. Claims Data

Fintepla® (fenfluramine) 2.2 mg/mL oral solution)

Dravet syndrome, a rare and severe developmental epileptic encephalopathy, has long presented challenges in terms of treatment and care. However, a new retrospective analysis utilizing U.S. claims data has unveiled promising outcomes for individuals with Dravet syndrome who were treated with fenfluramine, following its approval in June 2020 for the management of seizures associated with Dravet syndrome in the United States.¹

The objective of this analysis was to quantify the relationship between fenfluramine and seizure-related healthcare events through claims data. The study utilized the Komodo U.S. healthcare claims database to measure healthcare utilization, including rescue anti-seizure medication use and healthcare visits. Individuals with Dravet syndrome were identified by ICD-10 codes. Patients receiving prior/concomitant cannabidiol or stiripentol were excluded.¹

Among the 108 individuals with Dravet syndrome included in the analysis, 91 individuals (84%) had continuous fenfluramine use for a minimum of 6 months. This subset exhibited a 77% reduction in rescue antiseizure medication use (P<0.001). Moreover, the reductions extended to healthcare visits, with a 50% decrease in ER visits, a 27% reduction in outpatient visits, a 24% reduction in neurology visits, and a 24% reduction in inpatient hospitalizations.¹

The findings suggest that meaningful reductions in healthcare utilization were observed among individuals with Dravet syndrome treated with fenfluramine for at least 6 months. The 6-month persistency rate of fenfluramine usage indicates not only its efficacy but also its tolerability in this patient population.¹

Authors: Srihari Jaganathan, Derek Ems, Rob Sederman, Chen Chen, Shuang Wu

Poster 2: Diagnosis and Information Management of Dravet Syndrome in Recently Diagnosed Pediatric Patients: Results From a Dravet Syndrome Foundation Caregiver Insight Survey

The second late-breaking poster explored the challenges surrounding the early diagnosis of Dravet syndrome. Caregivers of pediatric patients aged ≤4 years, diagnosed with Dravet syndrome, were surveyed to gather insights into their experiences.²

The survey, distributed via the Dravet Syndrome Foundation Family Network, received responses from 73 caregivers. The results highlighted that the average age at symptom onset was 5 months, while the average age at Dravet syndrome diagnosis was 12 months. Changes in healthcare providers were common, with the most frequent reasons being clinical expertise and trust. The poster further highlights the vital role that patient advocacy groups and online resources play in providing caregivers with valuable information and support during the diagnosis and management process.²

Authors: Andrea Wilkinson, BA, Danya Kaye, BA, Veronica Hood, PhD, Mary Anne Meskis, Laurie Bailey, BCPA, Rebecca Burns, PharmD, PhD, Amélie Lothe, PhD

These posters underscore the advancements in understanding and managing Dravet syndrome, as well as the pivotal role of both medical interventions and patient advocacy resources in enhancing the lives of those affected by this condition.^1,2

About FINTEPLA^® (fenfluramine)¹

In the U.S., FINTEPLA is approved for the treatment of seizures associated with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) in patients 2 years of age and older.

FINTEPLA is available in the U.S. through the FINTEPLA Risk Evaluation and Mitigation Strategy (REMS). Further information is available at www.FinteplaREMS.com or by telephone at 1-877-964-3649.

Please see full Prescribing Information, including Boxed Warning, for additional important information on FINTEPLA.

INDICATION

FINTEPLA is a prescription medicine used to treat seizures associated with Dravet syndrome and Lennox-Gastaut syndrome in patients 2 years of age and older.
It is not known if FINTEPLA is safe and effective in children less than 2 years of age.

IMPORTANT SAFETY INFORMATION

FINTEPLA can cause serious side effects, including:

1. Problems with the valves in the heart (valvular heart disease) and high blood pressure in the arteries of the lungs (pulmonary arterial hypertension) have been associated with fenfluramine, the active ingredient in FINTEPLA. Your healthcare provider will do a test called an echocardiogram to check your heart and to evaluate for high blood pressure in the arteries of the lungs before you start taking FINTEPLA, again every 6 months during treatment, and one time 3 to 6 months after you take your last dose of FINTEPLA.

Call your healthcare provider right away if you develop any of these signs and symptoms of heart or lung problems during treatment with FINTEPLA:

shortness of breath
chest pain
tiredness or weakness, especially with increased activity
sensations of a rapid, fluttering heartbeat (palpitations)
lightheadedness or fainting
irregular pulse
swollen ankles or feet
bluish color of your lips and skin (cyanosis)

Because of the risk of heart valve problems (valvular heart disease) and high blood pressure in arteries of lungs (pulmonary arterial hypertension), FINTEPLA is only available through a restricted program called the FINTEPLA Risk Evaluation and Mitigation Strategy (REMS). Before you or your child receives FINTEPLA, your healthcare provider or pharmacist will make sure you understand how to take FINTEPLA safely. If you have any questions about FINTEPLA, ask your healthcare provider, visit www.FinteplaREMS.com, or call 1-877-964-3649.

2. Decreased appetite and decreased weight. Decreased appetite and decreased weight are both serious and common side effects of FINTEPLA in people with Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS).

Your weight should be checked regularly during your treatment with FINTEPLA.
Your healthcare provider may need to make changes to your FINTEPLA dose if your weight decreases. In some cases, FINTEPLA may need to be stopped.

3. Sleepiness, sedation, and lack of energy (lethargy). These are both serious and common side effects of FINTEPLA in people with Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS). Taking FINTEPLA 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 FINTEPLA affects you.

4. Like all other antiepileptic drugs, FINTEPLA 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 have any of these symptoms, especially if they are new, worse, or worry you:

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

How can I watch for early symptoms of suicidal thoughts and actions?

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

Suicidal thoughts or actions can be caused by things other than medicines. If you have suicidal thoughts or actions, your healthcare provider may check for other causes.

5. Do not stop taking FINTEPLA without first talking to your healthcare provider. Stopping a seizure medicine such as FINTEPLA can suddenly cause you to have seizures more often or seizures that do not stop (status epilepticus).

Call your healthcare provider between visits as needed, especially if you are worried about symptoms.

Do not take FINTEPLA if you:

are allergic to fenfluramine or any of the ingredients in FINTEPLA. See below for a complete list of ingredients in FINTEPLA.
are taking or have stopped taking medicines called monoamine oxidase inhibitors (MAOIs) in the last 14 days. This may cause a serious or life-threatening problem called serotonin syndrome. If you are not sure whether or not you are taking one of these medicines, contact your healthcare provider.

Before taking FINTEPLA, tell your healthcare provider about all of your medical conditions, including if you:

have heart problems
have or have had weight loss
have or have had depression, mood problems, or suicidal thoughts or behavior
have kidney problems
have liver problems
are pregnant or plan to become pregnant. Tell your healthcare provider right away if you become pregnant while taking FINTEPLA. You and your healthcare provider will decide if you should take FINTEPLA while you are pregnant.
- If you become pregnant while taking FINTEPLA, talk to your healthcare provider about registering with the North American Antiepileptic Drug Pregnancy Registry. You can enroll in this registry by calling 1-888-233-2334 or go to www.aedpregnancyregistry.org. The purpose of this registry is to collect information about the safety of antiepileptic drugs during pregnancy.
are breastfeeding or plan to breastfeed. It is not known if FINTEPLA passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby while taking FINTEPLA.

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

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

How should I take FINTEPLA?

Read the Instructions for Use for information on the right way to use FINTEPLA.
Take FINTEPLA exactly as your healthcare provider tells you to take it.
Your healthcare provider will tell you how much FINTEPLA to take and when to take it.
FINTEPLA may be taken with or without food.
Measure your dose of FINTEPLA using the dosing syringe that is provided by the pharmacy. Do not use a household teaspoon or tablespoon.
FINTEPLA can be given through gastric and nasogastric feeding tubes.

What should I avoid while taking FINTEPLA?

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

What are the possible side effects of FINTEPLA?

FINTEPLA may cause serious side effects, including:

See "FINTEPLA can cause serious side effects" above
- Serotonin syndrome. Serotonin syndrome is a life-threatening problem that can happen in people taking FINTEPLA, especially if FINTEPLA is taken with certain other medicines including: anti-depressant medicines called SSRIs, SNRIs, TCAs, and MAOIs; tryptophan; lithium; antipsychotics; St. John's Wort; dextromethorphan; tramadol.

Call your healthcare provider right away if you have any of the following symptoms of serotonin syndrome:

- - mental status changes such as seeing things that are not there (hallucinations), agitation, or coma
  - changes in blood pressure
  - tight muscles
  - fast heartbeat
  - nausea, vomiting, diarrhea
  - high body temperature
  - trouble walking
- High blood pressure (hypertension). Hypertension is both a serious and common side effect. FINTEPLA can cause your blood pressure to increase even if you have never had high blood pressure before. Your healthcare provider will check your blood pressure while you are taking FINTEPLA.
- Increased pressure in your eyes (glaucoma). Symptoms of glaucoma may include:
  - red eyes
  - seeing halos or bright colors around lights
  - nausea or vomiting
  - decreased vision
  - eye pain or discomfort
  - blurred vision

If you have any of these symptoms, call your healthcare provider right away.

- The most common side effects of FINTEPLA when used to treat Dravet syndrome (DS) include:
  - decreased appetite
  - diarrhea
  - low energy
  - respiratory infection
  - decreased weight
  - fever
  - constipation
  - abnormal echocardiogram
  - sleepiness
  - problems with movement, balance, and walking
  - increased drooling
  - increased blood pressure
  - vomiting
  - falls
  - seizures that do not stop
  - weakness

The most common side effects of FINTEPLA when used to treat Lennox-Gastaut syndrome (LGS) include:
- - diarrhea
  - tiredness
  - vomiting
  - sleepiness
  - decreased appetite

These are not all the possible side effects of FINTEPLA. For more information, ask your healthcare provider or pharmacist. Tell your healthcare provider if you have any side effect that bothers you or that does not go away.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1–800–FDA–1088.

Keep FINTEPLA and all medicines out of the reach of children.

General information about the safe and effective use of FINTEPLA.
Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide. Do not use FINTEPLA for a condition for which it was not prescribed. Do not give FINTEPLA to other people, even if they have the same symptoms that you have. It may harm them.

What are the ingredients in FINTEPLA?
Active ingredient: fenfluramine hydrochloride
Inactive ingredients: cherry flavor, citric acid, ethylparaben, hydroxyethylcellulose, methylparaben, potassium citrate, sucralose, and water.
FINTEPLA contains no ingredient made from gluten-containing grain (wheat, barley, or rye) and contains not more than 0.1% of carbohydrates, which is from the cherry flavoring.

Please see full Prescribing Information, including Medication Guide, for additional Important Safety Information on FINTEPLA.

For further information, contact UCB:

Corporate Communications
Becky Malone, U.S. Media Relations
T +919.605.9600
[email protected]

Investor Relations
Antje Witte
T +32.2.559.9414
[email protected]

About UCB

UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With approximately 8,600 people in approximately 40 countries, the company generated revenue of € 5.8 billion in 2021. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCBUSA.

References

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1. Srihari Jaganathan, Derek Ems, Rob Sederman, Chen Chen, Shuang Wu; Outcomes Among Individuals with Dravet Syndrome Using Fenfluramine: A Retrospective Analysis Using U.S. Claims Data
2. Andrea Wilkinson, BA, Danya Kaye, BA, Veronica Hood, Ph.D., Mary Anne Meskis, Laurie Bailey, BCPA, Rebecca Burns, PharmD, Ph.D., Amélie Lothe, Ph.D.; Diagnosis and Information Management of Dravet Syndrome in Recently Diagnosed Pediatric Patients: Results from a Dravet Syndrome Foundation Caregiver Insight Survey