Expanded Access Protocol of BHV-0223 for Patients With Amyotrophic Lateral Sclerosis (ALS)

BHV-0223 Expanded Access Protocol in Patients With Amyotrophic Lateral Sclerosis

This is an open label expanded access protocol for the treatment of up to approximately 250 adult patients with amyotrophic lateral sclerosis (ALS) who have difficulty swallowing oral riluzole tablets and may be able to derive benefit from treatment with an alternative oral formulation of riluzole.

Riluzole is indicated in the U.S. for the treatment of patients with amyotrophic lateral sclerosis (ALS). The commercially available dosage form is a 50mg oral tablet. This expanded access protocol (EAP) is designed to provide access to a dissolving tablet formulation of riluzole designed for sublingual (SL) administration, in patients with ALS who, in the opinion and clinical judgement of the treating physician, would benefit from treatment with BHV-0223.

Inclusion Criteria:

• Patients with diagnosed ALS of any type or duration
• Current or previous treatment with oral riluzole tablets, or patients who have never taken riluzole oral tablets, or patients who have successfully completed a clinical trial with BHV-0223 and were not withdrawn prematurely due to adverse events
• Swallowing difficulties, or patient or caregiver report choking one or more times per week, or investigator deems appropriate to treat with sublingual BHV-0223 because (s)he deems the patient cannot be satisfactorily treated with Rilutek®
• Adequate hepatic function

Exclusion Criteria:

• Patient with history of severe hypersensitivity reaction to riluzole oral tablets or BHV-0223
• Patient is known to have any other acute or chronic liver disease