FDA Rejects Outlook Therapeutics Eye Drug for Wet AMD Due to Lack of Substantial Evidence

Outlook Therapeutics stock plunged after the FDA issued a complete response letter, citing a lack of substantial evidence of effectiveness for its eye drug ONS-5010. The FDA recommended confirmatory evidence of efficacy to support the application. The company plans to meet with the FDA to explore pathways for potential approval and continue efforts to expand into additional European markets.

Outlook Therapeutics, Inc. (OTLK) saw its stock price plummet following the U.S. Food and Drug Administration (FDA) issuing a complete response letter (CRL) for the company's resubmitted biologics license application (BLA) for ONS-5010. The FDA cited a lack of substantial evidence of effectiveness for the drug, which is intended to treat wet age-related macular degeneration (wet AMD). The FDA recommended that confirmatory evidence of efficacy be submitted to support the application.

The FDA's CRL indicated that ONS-5010 did not meet the primary efficacy endpoint in its NORSE EIGHT trial, although NORSE TWO did meet its endpoint. Despite the setback, Outlook Therapeutics intends to engage with the FDA to clarify the necessary steps for potential approval. The company also plans to seek broader market access in Europe, where its product LYTENAVA™ has already received marketing authorization.

LYTENAVA™ (bevacizumab gamma) is the first and only authorized ophthalmic formulation of bevacizumab for wet AMD treatment in the EU and UK. The drug became commercially available in Germany and the UK in June 2025, providing a treatment option for wet AMD in those regions. This market expansion could potentially position Outlook Therapeutics as a leader in the market segment.

Outlook Therapeutics will hold a conference call and webcast on August 28, 2025, to discuss this development further. The company's CEO, Bob Jahr, commented that while they are disappointed with the outcome, they remain committed to providing patients with a safe and effective alternative to compounded Avastin manufactured in the United States.

The FDA's CRL identified only one deficiency, for a lack of substantial evidence of effectiveness, suggesting a focused area for the company to address rather than multiple issues. Outlook Therapeutics plans to meet with the FDA to gain clarity on their requirements, indicating a proactive approach to resolving the concerns raised about their application.

Institutional investors have been actively tracking Outlook Therapeutics' stock. According to Quiver Quantitative, 34 institutional investors added shares of OTLK to their portfolio, while 40 decreased their positions in the most recent quarter. Recent analyst ratings have been mixed, with one firm issuing a "Buy" rating on the stock. Analysts have also issued price targets for OTLK, with a median target of $12.0.

References:
[1] https://www.quiverquant.com/news/Outlook+Therapeutics+Receives+Complete+Response+Letter+from+FDA+for+ONS-5010+BLA+Resubmission