Eton Pharmaceuticals Announces Extension of PDUFA Goal Date for ET-400

Rhea-AI Summary

Eton Pharmaceuticals (Nasdaq: ETON) announced that the FDA has extended the PDUFA goal date for its New Drug Application (NDA) for ET-400 to May 28, 2025, from the original date of February 28, 2025. The three-month extension was implemented to allow the FDA additional time to review supplemental information provided in December in response to their request.

The company states it has fully addressed all FDA questions with no outstanding requests. CEO Sean Brynjelsen expressed confidence in the NDA package and indicated that this standard extension is not expected to significantly impact their 2025 revenue projections. The drug is intended as a therapy for pediatric rare diseases.

Positive

• Company confirms all FDA questions have been fully addressed
• Management states the delay won't significantly affect 2025 revenue projections

Negative

• PDUFA date delayed by 3 months to May 28, 2025
• FDA requires additional review time for supplemental information

Insights

Pharmaceutical Industry Analyst

The FDA's three-month extension of Eton's ET-400 PDUFA date warrants careful analysis within the context of regulatory dynamics and market implications. This type of extension, while adding 90 days to the review timeline, is relatively routine when the FDA requires additional time to review supplemental information. The key positive signal here is that Eton has confirmed there are no outstanding FDA requests, suggesting this is primarily a procedural delay rather than a reflection of significant concerns with the application.

The company's maintained revenue projections for 2025 despite the delay is particularly noteworthy. This indicates either: 1) Conservative initial forecasting that already accounted for potential delays, or 2) The majority of projected 2025 revenue is expected to come in the latter half of the year, making a Q2 versus Q1 approval less impactful to annual figures.

For the pediatric rare disease market, where ET-400 is positioned, timing can be critical. However, the May 28 timeline still positions Eton to potentially capture most of the 2025 market opportunity, assuming approval. The pediatric rare disease space typically sees less immediate competitive pressure and more stable long-term revenue streams once products are established, making a three-month delay less significant compared to other therapeutic areas.

Investors should focus on two key aspects: First, Eton's clear communication about having addressed all FDA questions suggests a constructive regulatory dialogue. Second, the company's financial runway through this extended review period appears secure given their $476M market capitalization and existing commercial portfolio. This positions them to maintain operational momentum through the new PDUFA date without immediate capital concerns.

DEER PARK, Ill., Feb. 06, 2025 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) goal date for the New Drug Application (NDA) for ET-400. The new PDUFA goal date is May 28, 2025.

The Company was notified that the FDA requires additional time to conduct a full review of supplemental information that was provided in December in response to an FDA request. The FDA communicated that it has applied a standard three-month extension from the original goal date of February 28, 2025. Eton believes it has fully addressed all questions from the FDA and there are no outstanding requests.

“We are confident in the strength of our NDA package and look forward to ensuring access to patients in need of this important pediatric rare disease therapy immediately after FDA approval this year. We do not expect this standard extension to significantly affect our internal 2025 revenue projections,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals.

About Eton Pharmaceuticals

Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has seven commercial rare disease products: INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has four additional product candidates in late-stage development: ET-400, ET-600, Amglidia®, and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at www.etonpharma.com.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Eton to undertake certain activities and accomplish certain goals and objectives. These statements include but are not limited to statements regarding Eton’s business strategy, Eton’s plans to develop and commercialize its product candidates, the safety and efficacy of Eton’s product candidates, Eton’s plans and expected timing with respect to regulatory filings and approvals, and the size and growth potential of the markets for Eton’s product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Eton’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Eton’s development programs and financial position are described in additional detail in Eton’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Eton undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Investor Relations:
Lisa M. Wilson, In-Site Communications, Inc.
T: 212-452-2793
E: lwilson@insitecony.com

Source: Eton Pharmaceuticals, Inc.

FAQ

What is the new PDUFA date for Eton Pharmaceuticals' ET-400 drug application?

The new PDUFA date for Eton Pharmaceuticals' ET-400 is May 28, 2025, extended from the original date of February 28, 2025.

Why did the FDA extend ETON's PDUFA date for ET-400?

The FDA extended the PDUFA date to conduct a full review of supplemental information that Eton provided in December in response to an FDA request.

How long is the PDUFA extension for ETON's ET-400 application?

The FDA has applied a standard three-month extension to the PDUFA date for ETON's ET-400 application.

Will the PDUFA extension affect Eton Pharmaceuticals' 2025 revenue projections?

According to the company, the standard extension is not expected to significantly affect their internal 2025 revenue projections.

What type of treatment is Eton's ET-400 being developed for?

ET-400 is being developed as a therapy for pediatric rare diseases.