Wave Life Sciences Announces FDA Feedback Supports Registrational Pathway for WVE-006 (RNA Editing) in AATD
Globe Newswire•01/10/2026•07:30 ET
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Key Highlights
- ➤FDA supports WVE-006 registrational pathway for AATD treatment (WVE)
- ➤Two-year registrational trial planned, with one-year interim analysis for potential accelerated approval
- ➤FDA supports Wave’s current LC-MS assay measuring M-AAT and Z-AAT proteins
- ➤Wave expects 600 mg monthly multidose cohort data in fourth quarter of 2026
Expert Statements
Christopher Wright, MD, PhD, Chief Medical Officer at Wave Life Sciences
“A registrational trial design that offers an efficient path to full approval, without the need for a separate confirmatory trial, is advantageous as we work to bring WVE-006 to the ~200,000 individuals living with Pi*ZZ AATD in the U.S. and Europe.”
Christopher Wright, MD, PhD, Chief Medical Officer at Wave Life Sciences
“Our discussions with key opinion leaders underscore the high unmet need and burden of the current standard of care, IV augmentation therapy, which may leave patients with depleted AAT levels during lung exacerbations, and does not address the risk of liver disease.”
Christopher Wright, MD, PhD, Chief Medical Officer at Wave Life Sciences
“WVE-006 avoids the irreversible bystander edits, indels and other off-target edits associated with DNA base editing.”
Christopher Wright, MD, PhD, Chief Medical Officer at Wave Life Sciences
“With the potential to address both lung and liver AATD with infrequent subcutaneous dosing and a favorable safety profile, we believe WVE-006 could meaningfully improve patients’ quality of life and offer a comprehensive treatment approach.”
Following meeting with FDA, Wave plans for a single, two-year registrational trial, with a one-year interim analysis for potential accelerated approval based on AATD biomarkers
WVE-006 (RNA editing) has potential to offer the AATD community a new standard-of-care treatment to protect the liver and lung with a safe and convenient therapy
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