Drugs / REBISUFLIGENE ETISPARVOVEC
REBISUFLIGENE ETISPARVOVEC
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Rejected (CRL) | US (FDA) | — | mucopolysaccharidosis type 3A | 2025-07-11 | “FDA has issued a Complete Response Letter (CRL) for its Biologics License Application (BLA) for UX111 (ABO-102) AAV gene therapy” ultragenyx.com ↗ |
| Filed | US (FDA) | — | mucopolysaccharidosis type 3A | 2024-12-19 | “submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA or the Agency) seeking accelerated approval for UX111 (ABO-102)” ultragenyx.com ↗ “In December 2024, Ultragenyx submitted a BLA to the FDA seeking accelerated approval for UX111 (formerly ABO-102)” abeonatherapeutics.com ↗ |
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT04088734 | Sep 2019 → Mar 2022 | mucopolysaccharidosis type 3A | Ultragenyx Pharmaceutical Inc | Terminated | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT02716246 | Apr 2016 → Mar 2029 expected | mucopolysaccharidosis type 3A | Ultragenyx Pharmaceutical Inc | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 31
| Date | Issuer | Release |
|---|---|---|
| 2026-02-12 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Reports Fourth Quarter and Full Year 2025 Financial Results and Corporate Update ultragenyx.com ↗
Earlier today the company received an Incomplete Response Letter (IRL) regarding its resubmitted BLA. |
| 2026-02-03 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Positive Longer-Term Data Demonstrating Treatment with UX111 Gene Therapy Results in Sustained, Significant Reductions in CSF-HS and Continued Meaningful Improvements in Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome (MPS IIIA) ultragenyx.com ↗
The results demonstrate substantial and durable biomarker improvements and meaningful functional benefits compared with natural history, with consistent and highly statistically significant results across age and disease severity. |
| 2026-01-30 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) to U.S. FDA ultragenyx.com ↗
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has resubmitted its Biologics License Application (BLA) seeking accelerated approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA) to the U.S. Food and Drug Administration (FDA or the Agency). |
| 2025-07-11 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) ultragenyx.com ↗
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for its Biologics License Application (BLA) for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). |
| 2025-02-18 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) ultragenyx.com ↗
The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (PDUFA) action date of August 18, 2025. |
| 2025-02-05 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces New Data Demonstrating that Treatment with UX111 AAV Gene Therapy Significantly Improved Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome Type A (MPS IIIA) Correlated with Sustained Reductions in CSF-HS ultragenyx.com ↗
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced new data demonstrating treatment with UX111 (ABO-102) AAV gene therapy led to a statistically significant improvement in the Bayley-III i raw scores for the subdomains of cognition, receptive communication and expressive communication in patients with Sanfilippo syndrome type A (MPS IIIA) compared to natural history data from untreated patients. |
| 2024-12-19 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA) ultragenyx.com ↗
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA or the Agency) seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). |
| 2024-02-06 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Data Demonstrating Treatment with UX111 Results in Significant Reduction in Heparan Sulfate Exposure in Cerebrospinal Fluid Correlated with Improved Long-term Cognitive Function in Patients with Sanfilippo Syndrome Type A (MPS IIIA) ultragenyx.com ↗
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced data demonstrating treatment with UX111 (ABO-102) AAV gene therapy resulted in rapid and sustained decreased levels of heparan sulfate (HS) in cerebrospinal fluid (CSF) in patients with Sanfilippo syndrome type A (MPS IIIA), and that sustained reduction in CSF HS exposure over time was correlated with improved long-term cognitive development. |
| 2021-11-17 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Announces Presentation on Pivotal Transpher A Study of ABO-102 in MPS IIIA at the 14th ICIEM Conference abeonatherapeutics.com ↗ |
| 2021-07-26 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Announces New MRI Data Showing Increased Brain Volume in Young Patients with Sanfilippo Syndrome Type A (MPS IIIA) After Treatment with ABO-102 Gene Therapy abeonatherapeutics.com ↗
The new MRI data shows the potential of ABO-102 to increase brain grey matter, corpus callosum and amygdala volumes and is consistent with previously reported results of preservation of neurocognitive development in these three young patients in the Transpher A study. |
| 2021-02-12 | Abeona Therapeutics, Inc | Results New Positive Phase 1/2 Interim Data Presented at WORLDSymposium™ Shows Neurocognitive Development of Young MPS IIIA Patients Preserved up to Three Years Following Treatment with Abeona’s ABO-102 Gene Therapy abeonatherapeutics.com ↗ |
| 2020-02-12 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Announces Positive Interim Data from MPS III Gene Therapy Programs Presented at WORLDSymposium™ abeonatherapeutics.com ↗
Results from the Transpher A study demonstrated that MPS IIIA patients younger than 30 months treated with ABO-102 in dose cohort 3 continue to show neurocognitive development 18 months to two years after treatment. |
| 2019-12-20 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Receives European Medicines Agency PRIME Designation for ABO-102 Gene Therapy in MPS IIIA abeonatherapeutics.com ↗ |
| 2019-07-25 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Announces Positive Interim Data from the ABO-102 Phase 1/2 Gene Therapy Clinical Trial in MPS IIIA abeonatherapeutics.com ↗
These new results showed that treatment of the youngest patients with ABO-102, all enrolled in the high-dose cohort 3, resulted in preservation of neurocognitive development 12-18 months post treatment. |
| 2019-01-31 | Abeona Therapeutics, Inc | Results Abeona Therapeutics to Present New Supportive Data for Novel Gene Therapies at WORLDSymposium™ abeonatherapeutics.com ↗
These new supportive data underscore the potential of our novel gene therapies for people living with lysosomal storage diseases. |
| 2018-05-18 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Provides Clinical Update on MPS IIIA Gene Therapy Trial at the 21st Annual ASGCT Meeting abeonatherapeutics.com ↗
The ongoing ABO-102 (AAV-SGSH) trial results demonstrate robust and durable clinical effects achieved throughout various timepoints post-administration. |
| 2018-04-23 | Abeona Therapeutics, Inc | Regulatory Abeona Announces FDA Grants RMAT Designation to ABO-102 Gene Therapy in MPS IIIA abeonatherapeutics.com ↗ |
| 2018-02-08 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Reports Top-Line Data from Phase 1/2 Gene Therapy Trial in MPS IIIA abeonatherapeutics.com ↗ |
| 2017-10-06 | Abeona Therapeutics, Inc | Results Abeona Announces Top-Line One Year Data from ABO-102 MPS IIIA Trial at ARM’s Cell & Gene Meeting on the Mesa abeonatherapeutics.com ↗
Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene and cell therapies for life-threatening rare diseases, announced one year data from Cohort 1 of the ongoing ABO-102 Phase 1/2 trial for Sanfilippo syndrome Type A (MPS IIIA). |
| 2017-05-12 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Announces Top-Line Data for ABO-102 Phase 1/2 MPS IIIA Gene Therapy Trial at ASGCT abeonatherapeutics.com ↗ |
| 2017-05-09 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Receives Regulatory Approval to Initiate Clinical Trial in Australia with ABO-102 Gene Therapy For Patients with MPS IIIA abeonatherapeutics.com ↗
today announced Australian regulatory approval to initiate a Phase 1/2 for the ABO-102 gene therapy program for patients with Sanfilippo syndrome type A (MPS IIIA). |
| 2017-02-17 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Provides Update from ABO-102 Phase 1/2 MPS IIIA Clinical Trial at the 13th Annual WORLDSymposium™ 2017 abeonatherapeutics.com ↗
ABO-102 gene therapy well-tolerated in 4 subjects (N=3 low dose, N=1 high dose) through 650 days follow up with no Serious Adverse Events |
| 2017-02-07 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Announces Presentations and Posters at the 13th Annual WORLDSymposium™ 2017 abeonatherapeutics.com ↗
Clinical data highlights include: CSF and urinary heparan sulfate GAG reduction, liver and spleen volume reduction, and neurological effects in pediatric subjects through six months post-injection |
| 2016-10-25 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Announces Fast Track Designation from FDA for ABO-102 in Sanfilippo Syndrome Type A abeonatherapeutics.com ↗ |
| 2016-10-20 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Provides Update from ABO-102 Phase 1/2 MPS IIIA Clinical Trial at Orphan Drugs & Rare Disease Conference, London UK abeonatherapeutics.com ↗
Observations 30 days post-injection for the low dose cohort demonstrated: ABO-102 is well-tolerated in subjects injected with the low dose of 5E13 vp/kg ABO-102 with no treatment related adverse events or serious adverse events (SAEs). |
| 2016-10-18 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-102 Gene Therapy in Sanfilippo Syndrome Type A abeonatherapeutics.com ↗ |
| 2016-09-15 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Announces Publication of Preclinical Data Supporting Clinical Translation of MPS IIIA Gene Therapy abeonatherapeutics.com ↗
Preclinical efficacy data demonstrated significant benefits leading to restoration of SGSH activity and reduction of glycosaminoglycans (GAG) throughout the central nervous system (CNS) and peripheral organs |
| 2016-08-04 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Announces European Regulatory Approval for Phase 1/2 Gene Therapy Clinical Study for Patients With Sanfilippo Syndrome Type A (MPS IIIA) abeonatherapeutics.com ↗
The clinical study was approved by the Agencia Espanola de Medicamentos y Productos Sanitarios, and the Company is conducting the Phase 1/2 clinical study at Cruces University Hospital (Bilbao, Spain). |
| 2016-08-02 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Provides Update on Initial Subjects in Sanfilippo Type A Gene Therapy Trial, Demonstrating Encouraging Early Biopotency Signals abeonatherapeutics.com ↗
Additionally, preliminary measures of clinically relevant biomarkers provide promising signals of potential systemic and CNS clinical benefits for patients suffering with MPS IIIA. |
| 2016-02-29 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Announces FDA Allowance of Investigational New Drug (IND) for Systemic AAV Phase 1/2 Clinical Study With ABO-102 Gene Therapy for Patients With Sanfilippo Syndrome Type A (MPS IIIA) abeonatherapeutics.com ↗ |
| 2016-01-11 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Announces Initial European Regulatory Approvals for Phase 1/2 Gene Therapy Clinical Studies for Patients With Sanfilippo Syndromes Type A (MPS IIIA) and Type B (MPS IIIB) abeonatherapeutics.com ↗
the Interministerial Council of Genetically Modified Organisms has approved the Genetically Modified Organism (GMO) Voluntary Release regulatory filings for both Phase 1/2 Gene Therapy Clinical Studies to treat patients with AB0-101 (AAV NAGLU) and ABO-102 (AAV SGSH) |
All press releases naming this drug 41 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | REBISUFLIGENE ETISPARVOVEC | ChEMBL registry synonym — accepted as the source's own label CHEMBL6068466 ↗ |
| Known as | ABO-102 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04088734 ↗ |
| Known as | SCAAV9.U1A.HSGSH | ChEMBL registry synonym — accepted as the source's own label CHEMBL6068466 ↗ |
| Known as | UX111 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02716246 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “Self-complementary adeno-associated virus serotype 9 carrying the human SGSH gene” NCT02716246 ↗ |
| Route | Intravenous | “injected intravenously through a peripheral limb vein” NCT02716246 ↗ |