Inovio Pharmaceuticals Inc. (NASDAQ: INO) experienced a significant drop in its share value on Monday after the U.S. Food and Drug Administration (FDA) announced its acceptance of the company's Biologics License Application (BLA) for INO-3107. This drug candidate is targeted as a potential treatment for adults suffering from Recurrent Respiratory Papillomatosis (RRP), a rare condition characterized by noncancerous growths in the respiratory tract caused by the Human Papillomavirus (HPV), predominantly affecting the voice box or larynx.
The FDA categorized the review of this application as standard, meaning it will follow the usual regulatory timeline. The Prescription Drug User Fee Act (PDUFA) goal date for the review has been established as October 30, 2026. The agency has also indicated that it does not currently intend to convene an advisory committee meeting to consider this application.
Inovio submitted the BLA for INO-3107 under the accelerated approval regulatory pathway, which is designed to expedite the review process for therapies that address unmet medical needs based on surrogate endpoints. However, in its acceptance letter, the FDA raised a preliminary concern that the submitted data do not adequately support eligibility for this accelerated pathway.
Specifically, the FDA's communication highlighted that the information provided so far has yet to meet the criteria required to justify the use of accelerated approval. This indicates that the regulatory body is seeking more robust evidence or possibly a reevaluation of the application's parameters.
Reacting to the FDA’s signals, Inovio maintains a firm stance on the therapeutic promise of INO-3107. The company asserts that this investigational treatment offers significant benefits over existing options and fulfills the criteria underpinning accelerated approval. To address the FDA’s concerns, Inovio intends to request a meeting with the agency to clarify the path forward to retain eligibility within the accelerated approval framework.
Importantly, Inovio has stated that it does not plan to pursue approval for INO-3107 through the traditional regulatory pathway at this time, emphasizing their focus on resolving the current review issues related to accelerated approval eligibility.
The BLA is supported primarily by clinical data stemming from a combined Phase 1/2 trial that evaluated INO-3107 in adult patients diagnosed with RRP. Eligible participants included those who had undergone two or more surgeries in the year preceding treatment initiation. Further bolstering the application, the company included long-term durability data from a retrospective analysis of these trial participants. This follow-up data showed that the majority of patients who could be evaluated continued to experience clinical benefits throughout the second year after treatment, and notably, without receiving additional dosing of INO-3107.
Despite the FDA's acceptance of the filing, the stock market's reception was negative, reflecting investor concerns over the regulatory challenges cited in the review process. At the time of reporting on Monday, shares of Inovio Pharmaceuticals were down 24.38%, trading at $1.73, according to Benzinga Pro data.
RRP is a relatively rare, benign disease, yet it imposes significant clinical burden due to the recurrent growth of papillomas in the respiratory tract. This therapeutic area has a limited number of treatment options, which underscores the potential significance of INO-3107 if approved. However, Inovio’s current regulatory hurdle regarding the accelerated approval pathway eligibility introduces uncertainty about the timing and eventual outcome of the review.
In this context, the next critical step for Inovio will likely be the planned engagement with the FDA aimed at resolving the agency’s concerns and realigning the submission with regulatory expectations. The result of these discussions could critically influence both the drug’s approval trajectory and investor sentiment in the coming months.
Given the situation, stakeholders will be closely watching both Inovio’s strategic responses and the FDA’s subsequent actions. While the standard review timeline extends to late 2026, the company’s ability to maintain its position within the accelerated approval program could potentially affect regulatory speed and market prospects.