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Ultragenyx Pharmaceutical (RARE) FDA Events

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FDA Events for Ultragenyx Pharmaceutical (RARE)

This section highlights FDA-related milestones and regulatory updates for drugs developed by Ultragenyx Pharmaceutical (RARE). Over the past two years, Ultragenyx Pharmaceutical has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as DTX401, GTX-102, UX111, and UX143. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.

Ultragenyx Pharmaceutical's Drugs in FDA Review

DTX401 - FDA Regulatory Timeline and Events

DTX401 is a drug developed by Ultragenyx Pharmaceutical for the following indication: Glycogen Storage Disease Type Ia (GSDIa). 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.

GTX-102 - FDA Regulatory Timeline and Events

GTX-102 is a drug developed by Ultragenyx Pharmaceutical for the following indication: Angelman Syndrome. 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.

UX111 - FDA Regulatory Timeline and Events

UX111 is a drug developed by Ultragenyx Pharmaceutical for the following indication: For Sanfilippo syndrome type A (MPS IIIA). 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.

UX143 (setrusumab) - FDA Regulatory Timeline and Events

UX143 (setrusumab) is a drug developed by Ultragenyx Pharmaceutical for the following indication: Osteogenesis Imperfecta (OI). 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.

Ultragenyx Pharmaceutical FDA Events - Frequently Asked Questions

However, the company does has had drugs under review or in active clinical development.

In the past two years, Ultragenyx Pharmaceutical (RARE) has reported FDA regulatory activity for the following drugs: GTX-102, UX111, UX143 (setrusumab) and DTX401.

The most recent FDA-related event for Ultragenyx Pharmaceutical occurred on July 9, 2025, involving UX143 (setrusumab). The update was categorized as "evaluation," with the company reporting: "Ultragenyx Pharmaceutical announced that the randomized, placebo-controlled Phase 3 portion of the Orbit study evaluating UX143 (setrusumab) in pediatric and young adult patients with osteogenesis imperfecta (OI) is progressing toward a final analysis consistent with the original plan, around the end of the year."

Current therapies from Ultragenyx Pharmaceutical in review with the FDA target conditions such as:

  • Angelman Syndrome - GTX-102
  • For Sanfilippo syndrome type A (MPS IIIA) - UX111
  • Osteogenesis Imperfecta (OI) - UX143 (setrusumab)
  • Glycogen Storage Disease Type Ia (GSDIa) - DTX401

More FDA Event Resources from MarketBeat

  • NDA: New Drug Application
  • ANDA: Abbreviated New Drug Application
  • sNDA: Supplemental New Drug Application
  • BLA: Biologics License Application
  • sBLA: Supplemental Biologics License Application
  • FDA Approved: Approved by the FDA
  • EMA: European Medicines Agency
  • CE Mark: European Union Certification
  • NMPA: China National Medical Products Administration
  • MHLW: Japanese Ministry of Health
  • FDA Meeting: Consultation with FDA
  • Pre-IND: Pre-Investigational New Drug Meeting
  • Breakthrough Therapy: Special FDA designation for promising therapies
  • Fast Track: Accelerated FDA approval pathway
  • Orphan Drug: Designation for rare disease treatments
  • RPD: Rare Pediatric Disease Designation
  • RMAT: Regenerative Medicine Advanced Therapy
  • DSMB Review: Data Safety Monitoring Board Review
  • IDMC Review: Independent Data Monitoring Committee
  • MAA: MHRA Marketing Authorization Application
  • RTF: Refusal to File (Rejected Application)
  • 510(k): FDA Clearance for Medical Devices
  • Rolling Submission: Staggered regulatory review process

This page (NASDAQ:RARE) was last updated on 7/10/2025 by MarketBeat.com Staff
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