Aquestive Therapeutics Gains FDA Orphan Drug Status for Libervant in Young Children with Seizure Clusters

WARREN, N.J., Dec. 19, 2024 - Aquestive Therapeutics, Inc., a company focused on developing innovative pharmaceutical solutions, has announced that the FDA has granted seven years of orphan drug exclusivity to Libervant® (diazepam) Buccal Film. This drug is designed for acute treatment of episodic, frequent seizure activity distinct from a patient's typical seizure pattern, specifically for children with epilepsy aged two to five years. The buccal film was approved in April 2024, with the FDA recognizing its administration method as a significant improvement over the rectal route, enhancing patient care.

Libervant stands out as the first oral rescue therapy for this young patient group, providing a vital, non-invasive treatment option, as highlighted by Aquestive's CEO, Daniel Barber. The company remains committed to raising awareness among healthcare providers about Libervant's benefits and ensuring access to the product for patients and caregivers.

The orphan drug designation by the FDA's Office of Orphan Products Development aims to support treatments for rare diseases affecting fewer than 200,000 people in the U.S. It offers benefits like financial incentives and market exclusivity upon approval. Libervant was initially granted orphan drug designation in November 2016, and its current exclusivity extends to April 2031 for the specified pediatric group.

Libervant® (diazepam) Buccal Film is notable for being the only FDA-approved oral medication for treating seizure clusters in young epilepsy patients. It is designed to treat sudden, frequent seizure episodes that differ from a patient's usual patterns. Aquestive developed Libervant as an alternative to existing device-based treatments like rectal gels and nasal sprays. The FDA had granted tentative approval for patients aged 12 and older in August 2022, with market access for this group pending until January 2027 due to existing drug exclusivity.

Important safety information for Libervant includes contraindications for children allergic to diazepam or with acute narrow-angle glaucoma. The drug, being a benzodiazepine, poses risks when combined with opioids, alcohol, or other central nervous system depressants, potentially causing severe drowsiness, breathing problems, coma, and even death. Emergency assistance is advised if symptoms like shallow breathing or excessive sleepiness occur.

Libervant carries a risk of abuse, misuse, and addiction, even when used as prescribed. Serious side effects, including coma and death, have been reported in cases of misuse. Withdrawal symptoms can occur if the medication is stopped suddenly, potentially leading to severe and life-threatening conditions. Patients may experience long-lasting withdrawal effects like anxiety, memory issues, and depression. It is crucial to follow the prescribed dosage and consult healthcare providers before making any changes to the treatment plan.

Libervant may cause sleepiness or dizziness, affecting motor skills and cognitive functions. Children should avoid activities like driving or operating machinery until the drug's effects are understood. The medication may also lead to suicidal thoughts or actions in a small number of patients, necessitating careful monitoring and communication with healthcare providers.

The most common side effects of Libervant include sleepiness and headaches. Patients are encouraged to discuss any side effects with their doctors and report them to the FDA for further review.

Aquestive Therapeutics is committed to advancing pharmaceutical solutions that improve patient care through novel delivery technologies. The company has multiple commercialized products and collaborates with other pharmaceutical firms to bring new treatments to market using proprietary technologies like PharmFilm®. Naast its focus on epilepsy treatment, Aquestive is developing products for severe allergic reactions and dermatological conditions.