AT-001
- Generic Name
- AT-001
- Drug Type
- Small Molecule
- Chemical Formula
- C17H10F3N5O3S
- CAS Number
- 1355612-71-3
- Unique Ingredient Identifier
- E8I3O45DDU
- Background
AT-001 is under investigation in clinical trial NCT03062384 (AT-001 for Long-term Preservation of Brain Health in Aging).
Icelandic Biotech ATx Secures €26.5M to Advance Novel Dementia Treatment
Arctic Therapeutics (ATx) has raised €26.5 million in Series A funding to advance its innovative protein-targeting dementia treatments. The company's lead candidate AT-001, which targets amyloid-induced angiopathy, shows promise in disrupting harmful protein clusters in brain arteries, potentially preventing, delaying, or reversing dementia progression.
Govorestat NDA for Classic Galactosemia Faces No FDA Advisory Committee, PDUFA Date on Track
• Applied Therapeutics' govorestat, a CNS penetrant Aldose Reductase Inhibitor, is under NDA review for treating Classic Galactosemia, a rare metabolic disorder.
• The FDA has communicated that an Advisory Committee meeting, previously scheduled for October 9, 2024, will no longer be required for the govorestat NDA review.
• The FDA's Priority Review of the govorestat NDA is proceeding as planned, with alignment on post-marketing requirements expected in October 2024.
• The PDUFA target action date for the FDA's decision on govorestat remains on track for November 28, 2024, signaling potential approval by the end of the year.
FDA Extends Review of Govorestat for Classic Galactosemia Treatment
• The FDA extended the review period for Applied Therapeutics' govorestat NDA by three months, setting a new PDUFA target action date for November 28, 2024.
• The extension allows the FDA to review supplemental analyses of previously submitted data, which the FDA considers a Major Amendment to the NDA.
• Govorestat, a novel Aldose Reductase Inhibitor, aims to be the first approved medication for Galactosemia, addressing neurological complications and toxic metabolite build-up.
• Applied Therapeutics will maintain its expanded access program for govorestat, ensuring continued treatment access for Galactosemia patients during the review period.
Applied Therapeutics Advances Govorestat for Galactosemia with FDA and EMA
• Applied Therapeutics will hold a pre-NDA meeting with the FDA this summer to discuss a potential NDA submission for govorestat (AT-007) for treating classic Galactosemia.
• The company plans to submit a Marketing Authorization Application (MAA) to the EMA in the fall of 2023 for govorestat to treat classic Galactosemia.
• Govorestat has shown clinical benefits in children with Galactosemia, improving daily living activities, behavior, cognition, motor skills, and reducing plasma galactitol levels.
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