FDA lifts clinical hold on Filana's simufilam, clearing path for TSC epilepsy study

Filana Therapeutics, Inc. announced on September 22, 2026 that the U.S. Food and Drug Administration lifted the clinical hold on the company's investigational new drug application, enabling Filana to initiate a planned Phase 2a proof-of-concept study in patients aged 12 to 55 with TSC-related epilepsy.¹ Patient screening is expected to begin by the first quarter of 2027.¹

Filana CEO Rick Barry said while on hold, the company completed key trial-readiness activities, including engaging a clinical research organization, identifying clinical trial sites, and recruiting potential investigators, and that with investigational drug supply in place, the Phase 2a study can now progress toward site initiation and patient enrollment.¹

The trial itself is described as a 16-week multicenter, randomized, double-blind clinical trial to evaluate two doses of simufilam in patients with refractory TSC-related seizures.¹ It is planned to be conducted at 13 sites in the U.S. with a target enrollment of 40 subjects aged 12 to 55.¹ All subjects who complete the double-blind treatment phase are eligible to participate in a 48-week extension study.¹ The study is designed to assess safety, tolerability, pharmacokinetics, and seizure-related measures, including seizure frequency, seizure intensity and duration, nighttime seizures, and sleep-related outcomes.¹

The company describes simufilam as an oral small molecule intended to modulate the filamin A protein.¹ Filana holds an exclusive worldwide license from Yale University to intellectual property supporting the use of simufilam in TSC-related epilepsy.¹

On the disease itself, the release notes epilepsy is the most common health issue affecting the TSC community, with 80% to 90% of TSC patients experiencing seizures, and TSC-related epilepsy affects approximately 45,000 people in the U.S.¹ Even with multiple approved treatments, more than 60% of TSC patients remain refractory to antiepileptic therapy.¹