FDA accepts resubmitted UX111 BLA for Sanfilippo A; PDUFA Sept 19, 2026
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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- Company
- Ultragenyx Pharmaceutical Inc. RARE
- Drug
- UX111 (rebisufligene etisparvovec)
- For
- Sanfilippo syndrome Type A (MPS IIIA)
- Stage
- Regulatory (BLA resubmission acceptance)
- When
- Apr 2, 2026
- Source note
- FDA accepted for review Ultragenyx's resubmitted Biologics License Application seeking accelerated approval of UX111 (rebisufligene etisparvovec) AAV9 gene therapy for Sanfilippo syndrome Type A (MPS IIIA), and assigned a PDUFA action date of September 19, 2026.
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- Source: sec.gov ↗ — 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
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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- 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
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page
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
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page
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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- 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