FDA accelerated approval of GENGLYCOS (DTX401) for GSDIa
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.
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary
- Company
- Ultragenyx Pharmaceutical Inc. RARE
- Drug
- GENGLYCOS (DTX401, pariglasgene brecaparvovec)
- For
- Glycogen storage disease type Ia
- Stage
- Approved
- When
- Aug 19, 2026
- Source note
- The known RARE PDUFA (Aug 23, 2026) for DTX401 resolved early — Ultragenyx PR dated Aug 19, 2026 announced accelerated approval in GSDIa patients 8 years and older; PMR is two years of open-label commercial data in 50 treated and 20 control patients.
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- Source: biospace.com ↗ — where this entry was taken from
- SEC filings (8-K) ↗ — press releases are attached to 8-K filings
- ClinicalTrials.gov ↗ — trials of this drug
- Drugs@FDA ↗ — search the drug name for approval letters
- RARE headlines on this site
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.
More details
- Entry
- GTX-102 Phase 3 Aspire Angelman topline now guided to Sept/Oct 2026 · Clinical trial results (readout / topline data)
- Drug
- GTX-102
- For
- Angelman syndrome (full maternal UBE3A deletion)
- Stage
- Phase 3 · the last stage before a drug can be approved
- When
- H2 2026 · the company gave a window, not a day
- Source note
- Q2 2026 earnings PR (Aug 4, 2026) narrows the known H2 2026 Aspire topline to the September or October 2026 timeframe (still H2; straddles Q3/Q4).
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- Source: finance.yahoo.com ↗ — where this entry was taken from
- SEC filings (8-K) ↗ — press releases are attached to 8-K filings
- ClinicalTrials.gov ↗ — trials of this drug
- RARE headlines on this site
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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.
More details
- Entry
- GTX-102 Phase 2/3 Aurora enrollment completion (additional genotypes/ages) · Company milestone
- Drug
- GTX-102
- For
- Angelman syndrome (additional genotypes and age groups)
- Stage
- Phase 2/3 · a combined mid- to late-stage study
- When
- H2 2026 · the company gave a window, not a day
- Source note
- Q2 2026 earnings PR (Aug 4, 2026).
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- Source: finance.yahoo.com ↗ — where this entry was taken from
- SEC filings (8-K) ↗ — press releases are attached to 8-K filings
- ClinicalTrials.gov ↗ — trials of this drug
- RARE headlines on this site
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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.
More details
- Entry
- UX016 GNE myopathy externally funded Phase 1/2 study start · Company milestone
- Drug
- UX016
- For
- GNE myopathy
- Stage
- Phase 1/2 · an early study that checks safety and takes a first look at effect
- When
- H2 2026 · the company gave a window, not a day
- Source note
- Q2 2026 earnings PR (Aug 4, 2026); minor pipeline start.
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- Source: finance.yahoo.com ↗ — where this entry was taken from
- SEC filings (8-K) ↗ — press releases are attached to 8-K filings
- ClinicalTrials.gov ↗ — trials of this drug
- RARE headlines on this site
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page
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.
More details
- Entry
- Cyprus2+ dose-finding data for UX701 in Wilson disease · Clinical trial results (readout / topline data)
- Drug
- UX701
- For
- Wilson disease
- Stage
- Phase 3 · the last stage before a drug can be approved
- When
- Q4 2026 · the company gave a window, not a day
- Source note
- Ultragenyx Q2 2026 earnings release: data from the dose-finding stage (after fourth cohort completion) expected in the fourth quarter of 2026.
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- Source: ir.ultragenyx.com ↗ — where this entry was taken from
- SEC filings (8-K) ↗ — press releases are attached to 8-K filings
- ClinicalTrials.gov ↗ — trials of this drug
- RARE headlines on this site
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page
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.
More details
- Entry
- Enh3ance Phase 3 second primary endpoint data for DTX301 in OTC deficiency · Clinical trial results (readout / topline data)
- Drug
- DTX301
- For
- Ornithine transcarbamylase (OTC) deficiency
- Stage
- Phase 3 · the last stage before a drug can be approved
- When
- H1 2027 · the company gave a window, not a day
- Source note
- Ultragenyx Q2 2026 earnings release: data from the second primary endpoint (treatment-burden reduction after longer follow-up) expected in the first half of 2027.
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- Checked by a person when it was added
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- Source: ir.ultragenyx.com ↗ — where this entry was taken from
- SEC filings (8-K) ↗ — press releases are attached to 8-K filings
- ClinicalTrials.gov ↗ — trials of this drug
- RARE headlines on this site
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page