Applied Therapeutics Updates on Govorestat FDA Response

Applied Therapeutics Faces FDA Reaction to Govorestat NDA

In a recent announcement, Applied Therapeutics, Inc. (Nasdaq: APLT), a biopharmaceutical company focused on developing treatments for rare diseases, has reported a significant update regarding their New Drug Application (NDA) for govorestat. This innovative medication is aimed at treating Classic Galactosemia, a rare genetic disorder.

Understanding the Complete Response Letter

The U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) after reviewing the NDA for govorestat. This letter signifies that the FDA found certain deficiencies in the NDA that prevent it from being approved in its current form. Although this news is disappointing for the company and the Galactosemia community, it provides a pathway for feedback and further discussion.

Next Steps for Applied Therapeutics

In response to the CRL, Applied Therapeutics plans to engage with the FDA to clarify the specific requirements needed for a potential resubmission of the NDA. The company is eager to address the FDA's concerns and continues its mission to bring govorestat to those in need, as noted by Dr. Shoshana Shendelman, the Founder and CEO.

Govorestat's Trial Results and Potential

The clinical trials conducted have shown promising results. Govorestat has demonstrated a rapid and sustained reduction in galactitol levels, which is a significant concern for patients suffering from Classic Galactosemia. Data from the Phase 3 ACTION-Galactosemia Kids study indicated that treatment can improve various aspects of daily life for pediatric patients, including cognition and behavioral symptoms.

Importance of Community Engagement

Dr. Shendelman expressed gratitude to all the patients, families, and healthcare professionals who participated in the clinical studies. The commitment of the community is crucial for the ongoing development of effective therapies for Classic Galactosemia, a disease with presently no existing treatment options.

Broader Applications of Govorestat

Interestingly, govorestat is also being explored for its efficacy in treating Sorbitol Dehydrogenase (SORD) Deficiency, another rare disease. The company anticipates submitting a separate NDA for this indication early in the first quarter of the upcoming year. The FDA’s review of govorestat for SORD will proceed independently of the current challenges faced with its application for Galactosemia.

What Makes Govorestat Unique?

Govorestat stands out as a central nervous system penetrant Aldose Reductase Inhibitor (ARI), putting it in a unique position for the treatment of multiple rare diseases. Notably, it has garnered Orphan Medicinal Product Designation from the European Medicines Agency (EMA) and Orphan Drug Designation from the U.S. FDA for both Galactosemia and SORD Deficiency.

Understanding Classic Galactosemia

Classic Galactosemia is a rare genetic metabolic disorder where the body cannot metabolize galactose properly. When galactose accumulates, it converts to galactitol, which can lead to severe neurological complications, including cognitive and motor skill deficiencies. With an estimated 3,300 patients in the U.S. alone, the condition represents a critical area of unmet medical need.

The Need for New Treatment Options

Given that around 80 to 100 new cases are diagnosed annually in the U.S., the urgency for effective treatment options becomes even clearer. The mandatory newborn screenings help diagnose this condition early, but without effective treatment options, the implications for many families remain daunting.

Applied Therapeutics' Commitment

As a clinical-stage biopharmaceutical company, Applied Therapeutics remains deeply committed to developing transformative treatments for rare diseases. Their flagship drug, govorestat, represents hope not only for patients with Galactosemia but also for other rare metabolic disorders.

Frequently Asked Questions

What is govorestat?

Govorestat is an innovative drug aiming to treat Classic Galactosemia and potentially other rare diseases by inhibiting aldose reductase in the central nervous system.

What did the FDA’s Complete Response Letter mean?

The Complete Response Letter indicates that the FDA found issues in the NDA for govorestat that need to be addressed before approval can be granted.

How did govorestat perform in clinical trials?

Clinical trials revealed that govorestat reduced galactitol levels in patients and improved their daily life activities, cognitive functions, and behavioral symptoms.

Are there other conditions govorestat is being developed for?

Yes, besides Classic Galactosemia, govorestat is also being studied for Sorbitol Dehydrogenase (SORD) Deficiency and PMM2-CDG.

What steps will Applied Therapeutics take next?

Applied Therapeutics plans to engage with the FDA to discuss the points raised in the CRL and outline the next steps towards a potential NDA resubmission.

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