FDA Delivers Crushing Blow to Outlook Therapeutics: Eye Drug Approval for Wet AMD Declined

In a significant setback that sent its shares tumbling, Outlook Therapeutics announced that the U.S. Food and Drug Administration (FDA) has declined to approve ONS-5010 (Lytenava), its eagerly anticipated drug for the treatment of wet age-related macular degeneration (wet AMD). This decision marks a critical moment for the biotechnology firm and casts a shadow over the future of a treatment many hoped would offer a new alternative in the ophthalmic market.

The FDA's Complete Response Letter (CRL) outlined several key concerns.

Primarily, the agency cited issues with the manufacturing process, specifically pointing to problems with one of Outlook Therapeutics' contracted manufacturers. Furthermore, the FDA indicated that there was a lack of substantial evidence of effectiveness from an additional clinical trial, a detail that adds another layer of complexity to the drug's path forward.

The market reaction was swift and brutal, with Outlook Therapeutics' shares plummeting by over 70% in early trading following the news.

Investors quickly recalibrated their expectations for the company, which had been banking on ONS-5010 as its flagship product. The rejection underscores the high-stakes nature of drug development and the stringent requirements for FDA approval, particularly for biologics.

ONS-5010, which uses a proprietary formulation of bevacizumab, was intended to be a new and differentiated treatment option for wet AMD, a leading cause of vision loss among older adults.

It aimed to provide a direct competitor to existing treatments such as Roche's Lucentis (ranibizumab), by offering a potential alternative for patients and physicians.

This isn't the first time Outlook Therapeutics has faced scrutiny from the FDA regarding ONS-5010. The company had previously received feedback from the agency noting an insufficient number of clinical sites and certain analytical issues that needed to be addressed.

While Outlook Therapeutics had worked to resolve these prior concerns, the latest CRL indicates that the new issues, particularly regarding manufacturing and additional trial data, have proven to be significant hurdles.

Despite the severe blow, Outlook Therapeutics is not abandoning ONS-5010. The company has stated its intent to immediately request a Type A meeting with the FDA to discuss the specific deficiencies outlined in the CRL.

Their plan involves addressing the identified manufacturing issues and potentially conducting further studies to provide the required evidence of effectiveness. Outlook Therapeutics expressed optimism that, after resolving these matters, it could resubmit its Biologics License Application (BLA) as early as possible in 2024.

The landscape for wet AMD treatments is competitive, with established players like Regeneron's Eylea and Roche's Vabysmo dominating the market.

ONS-5010's approval would have offered patients and clinicians another choice, but this delay means existing treatments will continue to hold their ground. The future of ONS-5010 now hinges on Outlook Therapeutics' ability to swiftly and effectively address the FDA's concerns, a process that will require significant resources and strategic execution.