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5 upcoming catalysts · 0 phase 2+ trials on file
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Ultragenyx expects to report results from its Phase 3 study of GTX-102 for Angelman syndrome (full maternal UBE3A deletion) in the second half of 2026 (no exact date yet). Phase 3 is the last stage before a drug can be approved, so this result usually decides the program’s future.
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GTX-102 Phase 2/3 Aurora enrollment completion (additional genotypes/ages), expected in the second half of 2026 (no exact date yet). A company milestone for Ultragenyx (GTX-102 for Angelman syndrome (additional genotypes and age groups)): a step in the program rather than a decision or a data readout.
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UX016 GNE myopathy externally funded Phase 1/2 study start, expected in the second half of 2026 (no exact date yet). A company milestone for Ultragenyx (UX016 for GNE myopathy): a step in the program rather than a decision or a data readout.
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Ultragenyx expects to report results from its Phase 3 study of UX701 for Wilson disease in Q4 2026 (Oct–Dec) (no exact date yet). Phase 3 is the last stage before a drug can be approved, so this result usually decides the program’s future.
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Ultragenyx expects to report results from its Phase 3 study of DTX301 for Ornithine transcarbamylase (OTC) deficiency in the first half of 2027 (no exact date yet). Phase 3 is the last stage before a drug can be approved, so this result usually decides the program’s future.
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The FDA’s decision on Ultragenyx’s UX111 (rebisufligene etisparvovec) for Sanfilippo syndrome type A (MPS IIIA) was due on Sep 19, 2026. The outcome isn’t recorded here yet — the linked source has the latest.
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The FDA’s decision on Ultragenyx’s DTX401 (pariglasgene brecaparvovec) for Glycogen storage disease type Ia was due on Aug 23, 2026. The outcome isn’t recorded here yet — the linked source has the latest.
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The FDA approved Ultragenyx’s GENGLYCOS (DTX401, pariglasgene brecaparvovec) for Glycogen storage disease type Ia on Aug 19, 2026. The company can now sell it in that market.
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FDA accepts resubmitted UX111 BLA for Sanfilippo A; PDUFA Sept 19, 2026, on Apr 2, 2026. A company milestone for Ultragenyx (UX111 (rebisufligene etisparvovec) for Sanfilippo syndrome Type A (MPS IIIA)): a step in the program rather than a decision or a data readout.
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Ultragenyx reported results from its Phase 3 study of DTX301 (avalotcagene ontaparvovec) for Ornithine transcarbamylase (OTC) deficiency on Mar 12, 2026. The study met its main goal.
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