Lexeo Therapeutics (NASDAQ:LXEO) shares skyrocketed 25% on Tuesday after the clinical-stage genetic medicine company announced significant progress in its discussions with the U.S. Food and Drug Administration (FDA) regarding an accelerated approval pathway for LX2006, its investigational treatment for Friedreich ataxia cardiomyopathy.
According to Lexeo, the FDA is open to allowing pooled data from the company’s ongoing Phase I/II studies to be combined with pivotal trial results in support of a Biologics License Application (BLA) under the Accelerated Approval pathway. This regulatory feedback could reduce the size and duration of the pivotal trial, potentially expediting Lexeo’s path to BLA submission.
Interim results from the clinical program have been encouraging. Participants with abnormally high left ventricular mass index (LVMI) at baseline achieved a mean reduction of 18% at six months and 23% at twelve months, exceeding the FDA-aligned efficacy target of a 10% reduction.
In addition, the company reported clinically meaningful improvement in the modified Friedreich Ataxia Rating Scale (mFARS), demonstrating slower disease progression and improved motor function. Among participants with abnormal baseline LVMI, all six achieved normal readings at their most recent evaluation.
Lexeo expects to launch its pivotal LX2006 study in the first half of 2026, pending final protocol approval. The FDA has already granted the therapy multiple designations, including Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Orphan Drug, and Fast Track status — all aimed at expediting development and review.
The treatment has been well tolerated, with no Grade 3 or higher serious adverse events reported among the 17 participants included in the most recent safety analysis.
The positive regulatory developments significantly strengthen Lexeo’s outlook, positioning LX2006 as one of the most advanced genetic therapy candidates for Friedreich ataxia–related heart disease.