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What additional regulatory designations will PTC's AADC gene therapy receive by end of 2025?
2
Resolution / Starting Odds
Breakthrough Therapy Designation • 25%
Orphan Drug Designation • 25%
Priority Review • 25%
No additional designations • 25%
FDA and other regulatory agency announcements
Story
FDA Approves PTC Therapeutics' Gene Therapy for AADC Deficiency
Nov 13, 2024, 11:18 PM
The U.S. Food and Drug Administration (FDA) has approved PTC Therapeutics' gene therapy for Aromatic L-Amino Acid Decarboxylase (AADC) deficiency, a potentially fatal enzyme deficiency disorder. This therapy is administered directly in the brain, marking a significant advancement in the treatment of neurological conditions. The approval has positively impacted PTC Therapeutics' stock, which saw a 2% increase in aftermarket trading.
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Yes • 50%
No • 50%
Yes • 50%
No • 50%
BLA submission • 25%
Phase III trial initiation • 25%
Conditional approval • 25%
No progress • 25%
Breakthrough Therapy • 25%
Fast Track • 25%
Orphan Drug • 25%
None • 25%
Only in the US • 25%
Only in the EU • 25%
In both US and EU • 25%
In US, EU, and Japan • 25%
Yes • 50%
No • 50%