FDA cancels panel for Applied's rare disease drug before November decision

The FDA has confirmed that the Nov. 28 deadline for its approval decision on Applied Therapeutics' metabolic disorder drug, govorestat, remains firm. Initially, the decision was expected by the end of August, but it was postponed to provide the FDA with additional time to review supplemental data that had been submitted. This additional data was considered a major amendment to the new drug application, thus necessitating the delay.

Recently, the FDA notified Applied Therapeutics that an advisory committee meeting to discuss the application, which was tentatively scheduled for October 9, will no longer be necessary. This information was shared with the company during a late-cycle review meeting. This decision is seen as a positive development by the company and its stakeholders. Shoshana Shendelman, Ph.D., CEO of Applied Therapeutics, expressed satisfaction with the collaborative efforts during the new drug application review process and emphasized the company's commitment to bringing the first potential treatment for classic galactosemia to patients. Shendelman also highlighted the company's dedication to the galactosemia community through commercial preparations, including establishing an effective patient access program, raising physician awareness, and engaging with payors.

Analysts at William Blair considered the FDA's decision to forego the advisory committee meeting as a positive sign. They interpreted it as an indication that the regulatory body is satisfied with the clinical data available to make a decision by the November 28 PDUFA (Prescription Drug User Fee Act) date. This viewpoint was articulated in a note dated September 18.

The journey of govorestat has been challenging. Last year, a phase 3 clinical trial revealed that the drug did not outperform a placebo in improving a composite of four measures, including language skills and self-care capabilities among children with galactosemia. This rare disease can result in developmental delays, speech issues, and motor function abnormalities. Despite these setbacks, Applied Therapeutics argued that the trial data demonstrated a consistent and sustained clinical benefit in activities of daily living, behavioral symptoms, cognition, adaptive behavior, and tremor. Consequently, the company proceeded with filing a new drug application with the FDA.

Initially, Applied Therapeutics intended to seek approval based on biomarker data alone. However, the FDA indicated that it would require evidence showing that the drug improves clinical outcomes to consider a positive decision. The phase 3 trial provided the necessary evidence of govorestat's, also known as AT-007, effect on clinical outcomes.

In summary, the FDA's decision to keep the November 28 approval deadline and cancel the advisory committee meeting is seen as a favorable development for Applied Therapeutics and its drug candidate, govorestat. The company remains optimistic about the potential approval and is actively preparing to support the classic galactosemia community through effective commercial strategies.