This section highlights FDA-related milestones and regulatory updates for drugs developed by Applied Therapeutics (APLT).
Over the past two years, Applied Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
AT-007, AT-007, and govorestat. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
AT-007 - FDA Regulatory Timeline and Events
AT-007 is a drug developed by Applied Therapeutics for the following indication: Galactosemia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- AT-007
- Announced Date:
- May 9, 2025
- Indication:
- Galactosemia
Announcement
Applied Therapeutics, Inc announced that it will present full 12-month clinical results from the INSPIRE Phase 3 trial of govorestat (AT-007) in a late-breaking abstract selected for oral presentation at the Peripheral Nerve Society (PNS) 2025 Annual Meeting, being held May 17-20, 2025 in Edinburgh, Scotland.
AI Summary
Applied Therapeutics, Inc. will present its full 12-month clinical results from the INSPIRE Phase 3 trial of govorestat (AT-007) at the Peripheral Nerve Society (PNS) 2025 Annual Meeting in Edinburgh, Scotland. The late-breaking abstract is scheduled for oral presentation on May 18, 2025, from 12:25 to 12:40 p.m. BST.
The INSPIRE trial is a double-blind, placebo-controlled study evaluating the effectiveness of govorestat in patients with Sorbitol Dehydrogenase (SORD) Deficiency, a subtype of Charcot-Marie-Tooth disease. In addition to the 12-month data, the presentation will feature new topline results from 18-month and 24-month assessments before transitioning to an open-label extension study. These findings aim to guide the design of the next randomized study for govorestat.
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AT-007 (SORD) - FDA Regulatory Timeline and Events
AT-007 (SORD) is a drug developed by Applied Therapeutics for the following indication: SORD Deficiency.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- AT-007 (SORD)
- Announced Date:
- May 18, 2025
- Indication:
- SORD Deficiency
Announcement
Applied Therapeutics, Inc. today presented full 12-month clinical results and new topline data from 18 and 24 months from the INSPIRE Phase 2/3 trial of govorestat (AT-007) for the treatment of Sorbitol Dehydrogenase (SORD) Deficiency, a subtype of Charcot-Marie-Tooth (CMT) disease, in an oral presentation at the Peripheral Nerve Society (PNS) 2025 Annual Meeting, being held May 17-20, 2025 in Edinburgh, Scotland.
AI Summary
Applied Therapeutics, Inc. presented new clinical data from its INSPIRE Phase 2/3 trial at the PNS 2025 Annual Meeting in Edinburgh, Scotland. The oral presentation highlighted full 12-month clinical results along with topline data from 18- and 24-month patient follow-ups for govorestat (AT-007), an investigational treatment for Sorbitol Dehydrogenase (SORD) Deficiency—a subtype of Charcot-Marie-Tooth (CMT) disease.
Key findings included a significant, sustained reduction in sorbitol levels, improvements in clinical outcomes, and evidence from 24-month MRI scans suggesting a slowing of disease progression. Govorestat was shown to be generally safe and well tolerated over the treatment period. These promising long-term results support the company’s commitment to advancing govorestat and inform future discussions with the FDA regarding a potential New Drug Application submission in 2025.
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govorestat - FDA Regulatory Timeline and Events
govorestat is a drug developed by Applied Therapeutics for the following indication: For the Treatment of Classic Galactosemia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- govorestat
- Announced Date:
- November 27, 2024
- Indication:
- For the Treatment of Classic Galactosemia
Announcement
Applied Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for the New Drug Application (NDA) for govorestat, a novel, central nervous system (CNS)-penetrant aldose reductase inhibitor (ARI), for the treatment of Classic Galactosemia.
AI Summary
Applied Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for its New Drug Application (NDA) for govorestat. Govorestat is a novel, central nervous system-penetrant aldose reductase inhibitor intended for the treatment of Classic Galactosemia. The CRL indicates that, in its current form, the application has deficiencies in its clinical data, preventing the FDA from approving it at this time. The company is reviewing the FDA's feedback and plans to request a meeting to discuss the steps needed for a possible resubmission or appeal of the decision. Despite the setback, Applied Therapeutics remains committed to addressing the high unmet medical need in the Galactosemia community and plans to work closely with the FDA to bring this promising treatment to patients.
Read Announcement- Drug:
- govorestat
- Announced Date:
- September 18, 2024
- Indication:
- For the Treatment of Classic Galactosemia
Announcement
Applied Therapeutics, Inc. announced an update on the ongoing New Drug Application (NDA) review of govorestat for the treatment of Classic Galactosemia.
AI Summary
Applied Therapeutics, Inc. announced an important update on its New Drug Application review with the FDA for govorestat, a promising treatment for Classic Galactosemia. In a recent late-cycle review meeting with the agency, the FDA decided that an Advisory Committee meeting would no longer be necessary. This decision helps streamline the review process, as the FDA confirmed that the NDA review is continuing as planned.
The FDA has set the Prescription Drug User Fee Act (PDUFA) target action date for November 28, 2024, and expects to align on the post-marketing requirements by October 2024. Applied Therapeutics is encouraged by the collaborative discussions with the FDA and remains focused on bringing this transformative treatment to patients suffering from Classic Galactosemia.
Read Announcement- Drug:
- govorestat
- Announced Date:
- September 18, 2024
- Estimated Event Date Range:
- November 28, 2024 - November 28, 2024
- Target Action Date:
- November 28, 2024
- Indication:
- For the Treatment of Classic Galactosemia
Announcement
Applied Therapeutics, Inc. announced that FDA Review of NDA continues to proceed as planned with PDUFA target action date of November 28, 2024
AI Summary
Applied Therapeutics, Inc. announced that the FDA’s review of its New Drug Application for govorestat is proceeding on schedule. The agency has confirmed that the NDA review is moving forward as planned, with a Prescription Drug User Fee Act (PDUFA) target action date set for November 28, 2024. This update reinforces the company’s optimistic progress in the process of seeking approval for govorestat, a potential treatment for Classic Galactosemia.
The FDA’s decision to continue with its review without scheduling an advisory committee meeting highlights the streamlined nature of the current evaluation. Applied Therapeutics remains hopeful that the ongoing collaborative dialogue with the FDA will address any additional post-marketing requirements in the coming weeks, positioning govorestat to become a critical option for patients with this rare condition.
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