UCB presents new data about the real-world experience of FINTEPLA® (fenfluramine) and rare epilepsy syndromes at 2023 American Epilepsy Society (AES) Annual Meeting

Rhea-AI Summary

Study on Zogenix Inc. (ZGNX) FINTEPLA Shows Positive Efficacy and Tolerability Over 12 Months

Positive

• Positive efficacy and tolerability of FINTEPLA over 12 months
• Real-world data highlights the need for caregiver support for people with developmental and epileptic encephalopathies
• Literature review emphasizes the importance of impactful conversations between patients, caregivers, and clinicians

Negative

• None.

• Study presents new persistence analysis of FINTEPLA, evaluating the efficacy and tolerability of FINTEPLA over a span of 12 months¹
• Real-world data looks into the need for resources to support caregivers – both parents and siblings – of people living with developmental and epileptic encephalopathies such as Dravet and Lennox-Gastaut syndromes in the transition from pediatric to long-term adult care²
• Systematic literature review underscores the need for more impactful conversations between patients, caregivers and clinicians about sudden unexpected death in epilepsy (SUDEP)³
• These research and findings demonstrate UCB's commitment to symptom management as well as holistic patient care

ATLANTA, Dec. 2, 2023 /PRNewswire/ -- UCB, a global biopharmaceutical company, today announced data being presented at the American Epilepsy Society (AES) Annual Meeting, taking place December 1–5 in Orlando, Florida, that focus on the real-world experiences of people living with rare epilepsy syndromes and their caregivers, in addition to several other presentations of clinical data evaluating the efficacy, safety and tolerability of FINTEPLA as a treatment for seizures associated with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS).

People living with a severe group of rare epilepsies called developmental and epileptic encephalopathies (DEEs), including DS and LGS, experience frequent and prolonged seizures, significant behavioral and developmental delays, movement and balance issues, and sleep difficulties.⁴ These ongoing challenges can create significant obstacles for both patients and their caregivers, as the conditions often require consistent and lifelong management to mitigate the dangerous side effects and detrimental impacts on quality of life.⁵

"It is critical to address not only seizures but also the non-seizure outcomes that can severely impact overall quality of life for people living with rare epilepsies and their caregivers," said Brad Chapman, Head of U.S. Epilepsy and Rare Syndromes at UCB. "The robust data being presented at AES reflect UCB's commitment to research and resources that support the rare epilepsy community. By continuing to deepen our understanding of the real-world experiences faced by people living with rare epilepsy syndromes (acute repetitive seizures and focal epilepsy), we will be able to better support their journey to finding appropriate treatment options faster and provide resources and education over the long term."

The real-world evidence being presented at AES supports the need to integrate the lived experiences of the rare epilepsy community into the development of treatment options and resources, to bring more holistic care to people living with rare epilepsies and their caregivers.

"Providing proactive education and counseling for people living with rare epilepsy syndromes and their caregivers is critical for improving seizure control, appropriate treatment adherence and management of risks like SUDEP," said Tom Stanton, President of the Danny Did Foundation and study investigator. "To achieve effective communication, neurologists need support to help them initiate difficult discussions about potentially negative impacts on quality of life. With the proper tools, providers can play a big role to empower and inform patients and caregivers."

The real-world data on fenfluramine, long-term care planning for rare epilepsy syndrome patients, and SUDEP counselling being presented at AES include:

• A 12-Month Persistence Analysis of Fenfluramine, Valproate, and Levetiracetam in Individuals with Dravet Syndrome: A Comparison Using U.S. Claims Data¹
  • A new analysis utilizing U.S. real-world data of fenfluramine in people living with DS.¹
  • Data from this late-breaking poster presentation featured U.S. healthcare claims data from Komodo Healthcare showing the persistency of fenfluramine – defined as the duration of continuous treatment without a gap of more than 90 days.¹
• A Survey of Rare Epilepsy Parents and Adult Siblings: To Assess Resources Needed to Prepare Families Living in the U.S. for Long-term Adult Care Planning for Their Loved One²
  • Survey assessed resources and needs required for caregivers' long-term care planning for a family member diagnosed with a developmental and epileptic encephalopathy (DEE). ²
  • Data from a survey of 135 caregivers of people with rare epilepsy syndromes, including parents and adult siblings, found that only 22% believed they had adequate access to long-term care planning information, while 53% did not have information available to them, and 24% were unsure. Those surveyed cited frustration in finding support and resources for patients with rare epilepsy syndromes and concern about how patients and their caregivers will be supported as they transition into adulthood.²
  • No family support programs or resources currently exist for long-term adult care planning or for the transfer of primary care for those living with rare epilepsy syndromes, uncovering a significant unmet need for additional support to facilitate future medical, legal and financial planning and ensure caregivers have access to resources and support at every step of their care journey.²
• Bridging The Gap Between Neurologists and People with Epilepsy/Caregivers: Systematic Literature Review about SUDEP Conversations³
  • Research looks to understand and learn more about caregivers' needs and experiences and neurologists' attitudes and behaviors toward SUDEP conversations. ³
  • A critical element of care for people living with epilepsy and rare epilepsy syndromes is the continuous management of risk related to sudden unexpected death in epilepsy (SUDEP), a leading disease-related cause of mortality among people living with epilepsy, affecting approximately one in 1,000 adults with epilepsy yearly⁶ and one in 150 for those with inadequate seizure control.⁷ A systematic literature review of studies found a gap between what people living with epilepsy and their caregivers want regarding information about SUDEP and the information shared with them by their neurologists.³
  • While many neurologists reported not actively discussing SUDEP to avoid causing fear or negative emotions, data show that patients and caregivers want to have more proactive discussions about SUDEP to provide them with the opportunity to take preventive measures. Increasing education for people with epilepsy and their caregivers about SUDEP, as well as providing neurologists with support and training to become adept at initiating these difficult discussions, are critical to help build a trusting partnership to enhance epilepsy management and, in turn, reduce the risks of SUDEP.³

In addition to the other accepted posters at AES, UCB will present additional clinical data on FINTEPLA. Click here to learn more.

About Epilepsy
Epilepsy is a common neurological condition worldwide and affects approximately 50 million people.⁸ Epilepsy and seizures can develop in any person at any age⁹ and is usually diagnosed after a person has had at least two seizures (or after one seizure with a high risk for more) that were not caused by some known medical condition.¹⁰

About UCB in Epilepsy
For three decades, we've been committed to people living with epilepsy and their families, surrounding the patient and caregiver through every step of their care journey. At our core, we have a responsibility to elevate the healthcare ecosystem. Because of this, we have established our legitimacy on the front lines of epilepsy research, development, and treatment innovation. But our past breakthroughs are only a prologue to our future. We will continue to reimagine how we care for patients, leveraging today's expertise for a better tomorrow. With so much experience behind us and so much potential ahead, we are more invested than ever in profoundly improving the lives of those living with or caring for those with epilepsy or a rare epilepsy syndrome – through our relentless pursuit of a seizure-free life.

About FINTEPLA^® (fenfluramine) Oral Solution ¹¹

FINTEPLA is indicated 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 only through a restricted distribution program called the FINTEPLA REMS. Further information is available at www.FINTEPLAREMS.com or by telephone at +1 877 964 3649.

IMPORTANT SAFETY INFORMATION

BOXED WARNING: VALVULAR HEART DISEASE and PULMONARY ARTERIAL HYPERTENSION

• There is an association between serotonergic drugs with 5-HT2B receptor agonist activity, including fenfluramine (the active ingredient in FINTEPLA), and valvular heart disease and pulmonary arterial hypertension.
• Echocardiogram assessments are required before, during, and after treatment with FINTEPLA.
• FINTEPLA is available only through a restricted program called the FINTEPLA REMS.

CONTRAINDICATIONS
FINTEPLA is contraindicated in patients with hypersensitivity to fenfluramine or any of the excipients in FINTEPLA and with concomitant use, or within 14 days of the administration, of monoamine oxidase inhibitors because of an increased risk of serotonin syndrome.

WARNINGS AND PRECAUTIONS

Valvular Heart Disease and Pulmonary Arterial Hypertension (see Boxed Warning): Because of the association between serotonergic drugs with 5-HT2B receptor agonist activity, including fenfluramine (the active ingredient in FINTEPLA), and valvular heart disease (VHD) and pulmonary arterial hypertension (PAH), cardiac monitoring via echocardiogram is required prior to starting treatment, during treatment, and after treatment with FINTEPLA concludes. Cardiac monitoring via echocardiogram can aid in early detection of these conditions. In clinical trials for DS and LGS of up to 3 years in duration, no patient receiving FINTEPLA developed VHD or PAH.

Monitoring: Prior to starting treatment, patients must undergo an echocardiogram to evaluate for VHD and PAH. Echocardiograms should be repeated every 6 months, and once at 3-6 months post treatment with FINTEPLA.

The prescriber must consider the benefits versus the risks of initiating or continuing treatment with FINTEPLA if any of the following signs are observed via echocardiogram: valvular abnormality or new abnormality; VHD indicated by mild or greater aortic regurgitation or moderate or greater mitral regurgitation, with additional characteristics of VHD (eg, valve thickening or restrictive valve motion); PAH indicated by elevated right heart/pulmonary artery pressure (PASP >35 mmHg).

FINTEPLA REMS Program (see Boxed Warning): FINTEPLA is available only through a restricted distribution program called the FINTEPLA Risk Evaluation and Mitigation Strategy (REMS) Program. Prescribers must be certified by enrolling in the FINTEPLA REMS. Prescribers must counsel patients receiving FINTEPLA about the risk of VHD and PAH, how to recognize signs and symptoms of VHD and PAH, the need for baseline (pretreatment) and periodic cardiac monitoring via echocardiogram during FINTEPLA treatment, and cardiac monitoring after FINTEPLA treatment. Patients must enroll in the FINTEPLA REMS and comply with ongoing monitoring requirements. The pharmacy must be certified by enrolling in the FINTEPLA REMS and must only dispense to patients who are authorized to receive FINTEPLA. Wholesalers and distributors must only distribute to certified pharmacies. Further information is available at www.FINTEPLAREMS.com or by telephone at 1-877-964-3649.

Decreased Appetite and Decreased Weight: FINTEPLA can cause decreases in appetite and weight. Decreases in weight appear to be dose related. Approximately half of the patients with LGS and most patients with DS resumed the expected measured increases in weight during the open-label extension studies. Weight should be monitored regularly during treatment with FINTEPLA, and dose modifications should be considered if a decrease in weight is observed.

Somnolence, Sedation, and Lethargy: FINTEPLA can cause somnolence, sedation, and lethargy. Other central nervous system (CNS) depressants, including alcohol, could potentiate these effects of FINTEPLA. Prescribers should monitor patients for somnolence and sedation and should advise patients not to drive or operate machinery until they have gained sufficient experience on FINTEPLA to gauge whether it adversely affects their ability to drive or operate machinery.

Suicidal Behavior and Ideation: Antiepileptic drugs (AEDs), including FINTEPLA, increase the risk of suicidal thoughts or behaviors in patients taking these drugs for any indication. Patients treated with an AED for any indication should be monitored for the emergence or worsening of depression, suicidal thoughts or behaviors, or any unusual changes in mood or behavior.

Anyone considering prescribing FINTEPLA or any other AED must balance the risk of suicidal thoughts or behaviors with the risks of untreated illness. Epilepsy and many other illnesses for which AEDs are prescribed are themselves associated with morbidity and mortality and an increased risk of suicidal thoughts and behaviors. Should suicidal thoughts and behaviors emerge during treatment, consider whether the emergence of these symptoms in any given patient may be related to the illness being treated.

Withdrawal of Antiepileptic Drugs: As with most AEDs, FINTEPLA should generally be withdrawn gradually because of the risk of increased seizure frequency and status epilepticus. If withdrawal is needed because of a serious adverse reaction, rapid discontinuation can be considered.

Serotonin Syndrome: Serotonin syndrome, a potentially life-threatening condition, may occur with FINTEPLA, particularly during concomitant administration of FINTEPLA with other serotonergic drugs, including, but not limited to, selective serotonin-norepinephrine reuptake inhibitors (SNRIs), selective serotonin reuptake inhibitors (SSRIs), tricyclic antidepressants (TCAs), bupropion, triptans, dietary supplements (eg, St. John's Wort, tryptophan), drugs that impair metabolism of serotonin (including monoamine oxidase inhibitors [MAOIs], which are contraindicated with FINTEPLA), dextromethorphan, lithium, tramadol, and antipsychotics with serotonergic agonist activity. Patients should be monitored for the emergence of signs and symptoms of serotonin syndrome, which include mental status changes (eg, agitation, hallucinations, coma), autonomic instability (eg, tachycardia, labile blood pressure, hyperthermia), neuromuscular signs (eg, hyperreflexia, incoordination), and/or gastrointestinal symptoms (eg, nausea, vomiting, diarrhea). If serotonin syndrome is suspected, treatment with FINTEPLA should be stopped immediately and symptomatic treatment should be started.

Increase in Blood Pressure: FINTEPLA can cause an increase in blood pressure. Rare cases of significant elevation in blood pressure, including hypertensive crisis, has been reported in adult patients treated with fenfluramine, including patients without a history of hypertension. In clinical trials for DS and LGS of up to 3 years in duration, no pediatric or adult patient receiving FINTEPLA developed hypertensive crisis. Monitor blood pressure in patients treated with FINTEPLA.

Glaucoma: Fenfluramine can cause mydriasis and can precipitate angle closure glaucoma. Consider discontinuing treatment with FINTEPLA in patients with acute decreases in visual acuity or ocular pain.

ADVERSE REACTIONS
The most common adverse reactions observed in DS studies (incidence at least 10% and greater than placebo) were decreased appetite; somnolence, sedation, lethargy; diarrhea; constipation; abnormal echocardiogram; fatigue, malaise, asthenia; ataxia, balance disorder, gait disturbance; blood pressure increased; drooling, salivary hypersecretion; pyrexia; upper respiratory tract infection; vomiting; decreased weight; fall; status epilepticus.

The most common adverse reactions observed in the LGS study (incidence at least 10% and greater than placebo) were diarrhea; decreased appetite; fatigue; somnolence; vomiting.

DRUG INTERACTIONS
Strong CYP1A2, CYP2B6, or CYP3A Inducers: Coadministration with strong CYP1A2, CYP2B6, or CYP3A inducers will decrease fenfluramine plasma concentrations. If coadministration of a strong CYP1A2, CYP2B6, or CYP3A inducer with FINTEPLA is necessary, monitor the patient for reduced efficacy and consider increasing the dosage of FINTEPLA as needed. If a strong CYP1A2, CYP2B6, or CYP3A inducer is discontinued during maintenance treatment with FINTEPLA, consider gradual reduction in the FINTEPLA dosage to the dose administered prior to initiating the inducer.

Strong CYP1A2 or CYP2D6 Inhibitors: Coadministration with strong CYP1A2 or CYP2D6 inhibitors will increase fenfluramine plasma concentrations. If FINTEPLA is coadministered with strong CYP1A2 or CYP2D6 inhibitors, the maximum daily dosage of FINTEPLA is 20 mg. If a strong CYP1A2 or CYP2D6 inhibitor is discontinued during maintenance treatment with FINTEPLA, consider gradual increase in the FINTEPLA dosage to the dose recommended without CYP1A2 or CYP2D6 inhibitors. If FINTEPLA is coadministered with stiripentol and a strong CYP1A2 or CYP2D6 inhibitor, the maximum daily dosage of FINTEPLA is 17 mg.

USE IN SPECIFIC POPULATIONS
In patients with severe impairment of kidney function (estimated glomerular filtration rate [eGFR]) 15 to 29 mL/min/1.73m², dosage adjustments are recommended. FINTEPLA has not been studied in patients with kidney failure (eGFR <15 mL/min/1.73m²).

Combined molar exposures of fenfluramine and norfenfluramine were increased in subjects with various degrees of hepatic impairment (Child-Pugh Class A, B, and C), necessitating a dosage adjustment in these patients.

To report SUSPECTED ADVERSE REACTIONS, contact UCB, Inc. at 1‑844-599-2273 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information, including Boxed Warning and 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
Becky.Malone@ucb.com

Investor Relations
Antje Witte
T +32.2.559.9414
antje.witte@ucb.com

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

1. Jaganathan S, et al. A 12-month persistence analysis of fenfluramine, valproate, and levetiracetam in individuals with Dravet Syndrome: a comparison using US claims data. Poster presented at American Epilepsy Society Annual Meeting; December 1-5, 2023; Orlando, Florida.
2. Laurie D Bailey, et al. A Survey of Rare Epilepsy Parents and Adult Siblings: To Assess Resources Needed to Prepare Families Living in the U.S. for Long-term Adult Care Planning for Their Loved. Abstract presented at American Epilepsy Society Annual Meeting; December 1-5, 2023; Orlando, Florida.
3. Luker J, et al. Bridging the gap between neurologists and people with epilepsy/caregivers: systematic literature review about SUDEP conversations. Poster presented at American Epilepsy Society Annual Meeting; December 1-5, 2023; Orlando, Florida.
4. Epilepsy Foundation. Developmental and Epileptic Encephalopathy (DEE). Available at: https://www.epilepsy.com/what-is-epilepsy/syndromes/developmental-and-epileptic-encephalopathy. Accessed November 2023.
5. Salom R, Aras LM, Piñero J, Duñabeitia JA. The psychosocial impact of caring for children with Dravet Syndrome. Epilepsy Behav Rep. 2023;24:100619.
6. Harden C, et al. Practice guideline summary: Sudden unexpected death in epilepsy incidence rates and risk factors: Report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology and the American Epilepsy Society. Neurology. 2017;88:1674-1680.
7. Child Neurology Foundation. SUDEP. Available at: https://www.childneurologyfoundation.org/sudep. Accessed November 2023.
8. Meyer AC, Dua T, Ma J, et al. Global disparities in the epilepsy treatment gap: a systemic review. Bull World Health Organ. 2010;88: 260-266.
9. Epilepsy Foundation. Who Can Get Epilepsy? Available at: https://www.epilepsy.com/learn/about-epilepsy-basics/who-gets-epilepsy. Accessed November 2023.
10. Epilepsy Foundation: What is Epilepsy? Available at: https://www.epilepsy.com/learn/about-epilepsy-basics. Accessed November 2023.
11. FINTEPLA (fenfluramine) oral solution: U.S. prescribing information. Smyrna, GA: UCB, Inc.

FINTEPLA^® is a registered trademark of the UCB Group of Companies.

©2023 UCB, Inc., Smyrna, GA 30080. All rights reserved. US-FA-2300166

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FAQ

What is the focus of the study on FINTEPLA by Zogenix Inc. (ZGNX)?

The study evaluates the efficacy and tolerability of FINTEPLA over a 12-month period.

What does the real-world data highlight about caregiver support for people with developmental and epileptic encephalopathies?

It emphasizes the need for resources to support caregivers, including parents and siblings, in the transition from pediatric to long-term adult care.

What is the significance of the systematic literature review related to FINTEPLA?

It underscores the need for more impactful conversations between patients, caregivers, and clinicians.

How long was the span of the study on FINTEPLA?

The study evaluated the efficacy and tolerability of FINTEPLA over a 12-month period.