Regeneron Gains EMA Accelerated Assessment Acceptance for Otarmeni in OTOF Hearing Loss

Regeneron Pharmaceuticals' EMA filing acceptance for Otarmeni (lunsotogene parvec) under Accelerated Assessment compresses the EU review clock for a dual-AAV1 vector gene therapy that already holds FDA accelerated approval, placing CMC teams and QA functions under simultaneous multi-jurisdictional scrutiny for an intracochlear biologic with no established EU precedent.

The Marketing Authorization Application (MAA) is supported by data from the pivotal CHORD trial, in which 24 participants aged 10 months to 16 years received a single intracochlear infusion of Otarmeni, either unilaterally (n=10) or bilaterally (n=14). An earlier 20-participant data cut from the same trial underpinned the FDA's April 2026 accelerated approval. Regulatory submissions are also planned for Japan, meaning manufacturing and release specifications will need to satisfy at least three major authority frameworks concurrently.

For QA directors and regulatory leads, the manufacturing profile warrants close attention. Otarmeni uses a dual-vector AAV1 construct, necessary because the full-length OTOF coding sequence exceeds the carrying capacity of a single AAV capsid, delivered via cochlear infusion under general anesthesia. The Myo15 promoter is designed to restrict transgene expression to inner hair cells, but cell-specificity claims of this kind typically attract detailed 21 CFR Part 211-equivalent scrutiny on potency assay design and lot-release criteria. EMA's Committee for Medicinal Products for Human Use (CHMP) will apply comparable expectations under its advanced therapy medicinal product (ATMP) framework.

The ultra-rare patient population, approximately 46 affected newborns per year in the EU, means commercial batch sizes will remain small, amplifying the process validation challenge. Sterility assurance for an intracochlear route of administration, combined with the cold-chain and fill-finish requirements typical of viral vector products, leaves limited margin for out-of-specification events at any stage of the supply chain. Accelerated Assessment compresses the standard 210-day CHMP review timeline, which tightens the window for responding to Day 120 and Day 180 questions without disrupting manufacturing continuity.

Otarmeni previously received EMA Orphan Designation, which carries its own post-authorisation obligations around pharmacovigilance and registry commitments, obligations that will layer onto any risk management plan negotiated during the accelerated review. If approved, it would be the first gene therapy authorised in the EU for OTOF-related hearing loss.

The outcome of CHMP's Accelerated Assessment will serve as a measurable indicator of how prepared Regeneron's CMC package is to satisfy dual FDA-EMA expectations for a novel intracochlear AAV gene therapy at commercial scale.

Source: Regeneron Pharmaceuticals via GlobeNewswire, 22 May 2026.