CAMBRIDGE, Mass., United States – May 5, 2026
Amylyx Pharmaceuticals Inc. has announced the launch of a U.S. Expanded Access Program (EAP) for adults with post-bariatric hypoglycemia (PBH), enabling eligible patients to access avexitide, an investigational first-in-class GLP-1 receptor antagonist, ahead of potential regulatory approval. The program is designed for patients with serious unmet medical need who are unable to participate in clinical trials, providing early access to a therapy targeting the root cause of recurrent hypoglycemia, while the drug continues evaluation in the Phase 3 LUCIDITY trial. This positions avexitide as a potential first approved treatment for PBH, a condition currently lacking any FDA-approved therapies.
Expanded Access Program Targets Unmet Patient Need
The Expanded Access Program allows physicians across the United States to request avexitide for eligible patients who have exhausted existing treatment options and meet strict clinical criteria. PBH is a chronic metabolic disorder that develops after bariatric procedures such as Roux-en-Y gastric bypass, causing dangerous and unpredictable drops in blood glucose levels.
These episodes can lead to severe neurological complications including confusion, seizures, and loss of consciousness, significantly impacting patient safety and quality of life. By offering access outside clinical trials, Amylyx Pharmaceuticals Inc. is addressing a critical treatment gap, especially for patients who cannot enroll in ongoing studies but require immediate therapeutic intervention.
Avexitide Shows Strong Therapeutic Potential
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Avexitide is a targeted GLP-1 receptor antagonist designed to block excessive GLP-1 signaling, which is the primary driver of hyperinsulinemic hypoglycemia in PBH patients. By controlling this pathway, the therapy aims to reduce inappropriate insulin secretion and stabilize blood glucose levels.
Clinical evidence from Phase 1 and Phase 2 studies has already demonstrated significant reductions in hypoglycemic events, supporting its advancement into late-stage development. The ongoing LUCIDITY Phase 3 trial is evaluating efficacy and safety in a controlled setting, with topline data expected in Q3 2026 and a potential commercial launch targeted for 2027, making this one of the most closely watched programs in metabolic disease therapeutics.
Regulatory Momentum and Commercial Outlook
Amylyx Pharmaceuticals Inc. has secured multiple designations from the U.S. Food and Drug Administration, including Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations, highlighting both the innovation and urgency of this therapy. These designations are expected to accelerate regulatory review and approval timelines, improving the chances of faster market entry.
With PBH affecting an estimated 8 percent of bariatric surgery patients in the U.S., the condition represents a significant and underserved market opportunity. The Expanded Access Program not only supports early patient access but also strengthens the company’s commercial positioning, reflecting a broader trend where pharmaceutical companies are bridging development and access through patient-focused programs.
Source: Amylyx Pharmaceuticals press release
