Competitive Approval RiskIf a competitor secures regulatory approval first, the investment case will depend more on demonstrating a clearly superior clinical outcome than on being first to market.
Direct Competitor ThreatRegeneron's advancing otoferlin program could capture significant U.S. and E.U. market share, constraining commercial prospects for competing therapies.
Market Size ConstraintsA relatively small addressable patient population raises questions about long-term revenue scale even if clinical success is achieved.