Rebisufligene etisparvovec
- Generic Name
- Rebisufligene etisparvovec
- Drug Type
- Biotech
- CAS Number
- 1966978-67-5
- Unique Ingredient Identifier
- V28L3Q0LVZ
Vertex Pharmaceuticals Abandons AAV Vector Technology Amid Broader Industry Retreat from Gene Therapy
• Vertex Pharmaceuticals has discontinued all research on adeno-associated virus (AAV) vector technology, impacting partnerships with Affinia Therapeutics and Tevard Biosciences focused on Duchenne muscular dystrophy treatments.
• The retreat from AAV vectors follows a broader industry trend, with Pfizer, Roche, Takeda, and Biogen all scaling back gene therapy programs due to safety concerns, limited payload capacity, and high manufacturing costs.
• Despite industry pullback, companies like Affinia Therapeutics continue developing next-generation AAV vectors, while others explore alternative delivery systems such as Herpes simplex virus-1 vectors with larger genetic payloads.
Ultragenyx Announces Positive 2024 Revenue and Advances Rare Disease Pipeline
• Ultragenyx reported preliminary 2024 revenue of $555 million to $560 million, exceeding previous guidance, driven by strong sales of Crysvita and Dojolvi.
• The company anticipates total revenue between $640 million and $670 million in 2025, projecting continued growth and pipeline advancement.
• Key clinical programs are progressing, including setrusumab for osteogenesis imperfecta (Phase 3) and GTX-102 for Angelman syndrome (Phase 3).
• Ultragenyx expects a PDUFA decision for UX111 in Sanfilippo syndrome type A and plans to file a BLA for DTX401 in Glycogen Storage Disease Type Ia in 2025.
Ultragenyx Initiates Phase 3 Aspire Study of GTX-102 for Angelman Syndrome
• Ultragenyx has commenced the Phase 3 Aspire study to evaluate GTX-102, an experimental treatment for Angelman syndrome, in children aged 4 to 17.
• The Aspire study is a randomized, sham-controlled trial with a 48-week primary efficacy analysis period, measuring cognitive improvement via the Bayley-4 cognitive raw score.
• Ultragenyx is also anticipating an interim analysis from the Phase 3 Orbit study for setrusumab, expected around the end of 2024 or early 2025.
• Analysts maintain a strong buy consensus on Ultragenyx stock, with price targets ranging from $48 to $140 per share, reflecting optimism about the company's pipeline.
Ultragenyx's UX111 Gene Therapy for Sanfilippo Syndrome Type A Receives FDA Priority Review
• The FDA has granted priority review to Ultragenyx's Biologics License Application (BLA) for UX111, a gene therapy for Sanfilippo syndrome type A (MPS IIIA).
• The FDA's decision is expected by August 18, 2025, and the agency is not planning an advisory committee meeting for this application.
• UX111 has demonstrated statistically significant improvements in cognitive and communication skills, correlated with reduced heparan sulfate levels in cerebrospinal fluid.
• UX111, if approved, would be the first-ever treatment for Sanfilippo syndrome type A, addressing a critical unmet need for this rare, neurodegenerative disease.
Advancements in Gene and Cell Therapies Target Diverse Diseases
• Ultragenyx seeks accelerated FDA approval for UX111, a gene therapy for MPSIII, based on Phase 1/2/3 trial data.
• Arbor Biotechnologies' CRISPR-based therapy ABO-101 receives clearance for US trial in Primary Hyperoxaluria Type 1.
• Allogene Therapeutics' ALLO-329 cleared by FDA for Phase 1 trial in rheumatology indications including lupus.
• uniQure progresses in trial for SOD1-ALS gene therapy AMT-162, advancing to the second cohort enrollment.
Ultragenyx Reports Positive Q3 2024 Results and Clinical Updates
• Ultragenyx's Q3 2024 total revenue increased by 42% year-over-year to $139 million, driven by Crysvita and Dojolvi sales growth, reaffirming its 2024 revenue guidance of $530-$550 million.
• The FDA granted Breakthrough Therapy Designation for setrusumab (UX143) in osteogenesis imperfecta, based on promising Phase 2 clinical trial results showing a significant reduction in fracture rate.
• Phase 3 follow-up data for DTX401 in glycogen storage disease type Ia (GSDIa) demonstrated a substantial 62% mean reduction in cornstarch intake among crossover patients.
• Ultragenyx anticipates filing a J-NDA for Dojolvi in Japan by mid-2025 and a BLA submission for DTX401 in mid-2025, marking significant progress in expanding treatment options for rare diseases.
Cell Therapy and Targeted Therapies Dominate Oncology Advances in Early 2025
• The FDA issued a CRL for Atara Biotherapeutics' tabelecleucel due to third-party manufacturing issues, not efficacy or safety data, delaying potential approval for EBV+ PTLD.
• EsoBiotec dosed the first patient in a trial for ESO-T01, an in vivo BCMA-directed CAR-T therapy for multiple myeloma, aiming for lower costs and simplified administration.
• Obecabtagene autoleucel (obe-cel) gained FDA approval for relapsed/refractory B-cell precursor ALL, offering a less toxic CD19-directed CAR T-cell therapy option.
• Arlocabtagene autoleucel (arlo-cel) shows promise in heavily pretreated relapsed/refractory multiple myeloma, eliciting a 48% complete response rate in phase 1 studies.
Liso-cel Demonstrates Significant Response in Relapsed/Refractory Marginal Zone Lymphoma
• Bristol Myers Squibb's liso-cel achieved a statistically significant and clinically meaningful overall response rate in patients with relapsed or refractory marginal zone lymphoma.
• The Phase 2 trial results highlight the potential of liso-cel as an effective treatment option for this challenging patient population.
• The data were presented at the 2025 Tandem Meetings, showcasing advancements in cell therapy for hematological malignancies.
Abeona Therapeutics Resubmits BLA for Pz-cel in Recessive Dystrophic Epidermolysis Bullosa
• Abeona Therapeutics has resubmitted its Biologics License Application (BLA) to the FDA for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (RDEB).
• The resubmission addresses Chemistry Manufacturing and Controls (CMC) requirements requested by the FDA, with no concerns raised regarding clinical safety or efficacy.
• Pz-cel, an autologous cell-based gene therapy, has shown promising results in Phase 3 and Phase 1/2a trials, demonstrating wound healing and pain reduction.
• The FDA is expected to set a new target action date upon acceptance, with a review period of two to six months for the resubmitted BLA.
Ultragenyx's UX111 Gene Therapy Shows Promise in Sanfilippo Syndrome Type A
• UX111 (rebisufligene etisparvovec) is an investigational gene therapy for Sanfilippo syndrome type A (MPS IIIA), a rare and fatal lysosomal storage disease.
• The therapy uses an AAV9 vector to deliver a functional copy of the SGSH gene, addressing the underlying enzyme deficiency that causes heparan sulfate accumulation.
• UX111 has received multiple designations, including Regenerative Medicine Advanced Therapy and Orphan Drug status, highlighting its potential impact on this unmet medical need.
Ultragenyx's Setrusumab Receives FDA Breakthrough Therapy Designation for Osteogenesis Imperfecta
• Ultragenyx's setrusumab (UX143) has been granted Breakthrough Therapy Designation by the FDA to reduce fracture risk in osteogenesis imperfecta (OI) patients.
• The FDA's decision was based on positive data from the Phase 2 Orbit study and Phase 2b ASTEROID study, demonstrating a significant reduction in fracture rates.
• Setrusumab, a fully human monoclonal antibody, inhibits sclerostin to enhance bone formation, density, and strength in OI patients.
• The Breakthrough Therapy Designation aims to expedite the development and review of setrusumab, offering hope for an approved treatment option for OI.
Ultragenyx to Evaluate Higher Dose of UX701 in Wilson Disease Gene Therapy Trial
• Ultragenyx is amending its Phase 1/2/3 Cyprus2+ trial of UX701, a gene therapy for Wilson disease, to include a higher dose cohort.
• Initial results from the first three cohorts showed clinical activity, with six patients fully tapering off standard-of-care treatment.
• The new cohort will also evaluate an optimized immunomodulation regimen to improve the efficiency and efficacy of UX701.
• UX701 has demonstrated stabilization of non-ceruloplasmin bound copper levels and improved copper trafficking in treated patients.
Ultragenyx to Pursue Accelerated Approval for UX111 Gene Therapy in MPSIIIA
• Ultragenyx plans to seek accelerated approval for UX111 (ABO-102), an AAV-based gene therapy, for mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome.
• The FDA has aligned with Ultragenyx on using cerebral spinal fluid (CSF) heparan sulfate (HS) as a surrogate endpoint for accelerated approval.
• Phase 1/2/3 trial data showed a 63.3% reduction in CSF HS exposure with UX111, correlating with cognitive improvements in treated children.
• BLA submission is anticipated in late 2024 or early 2025, pending further discussions with the FDA on submission details.
Ultragenyx Announces Preliminary 2023 Revenue and 2024 Financial Guidance
Ultragenyx Pharmaceutical Inc. has reported its preliminary unaudited 2023 revenue results, with total revenue ranging between $430 million to $435 million. The company also provided its 2024 revenue guidance, expecting total revenue to be between $500 million to $530 million. Key highlights include significant revenue from Crysvita and Dojolvi, and a year-end 2023 cash balance of approximately $776 million. Ultragenyx anticipates several important clinical milestones in 2024, including advancements in its gene therapy programs and Phase 3 studies for various genetic diseases.
Ultragenyx's UX111 Gene Therapy Shows Sustained Efficacy in MPS IIIA Patients
• UX111, an AAV9-based gene therapy, demonstrates rapid and sustained reduction of cerebrospinal fluid biomarker levels in MPS IIIA patients treated early.
• A phase 1/2/3 clinical trial (Transpher A) showed dose-dependent efficacy, with the highest dose cohort (3 x 10^13 vg/kg) exhibiting the most significant biomarker reduction.
• The gene therapy has shown neurodevelopmental gains in treated patients, with no drug-related serious adverse events reported in the trial.
• UX111, acquired by Ultragenyx from Abeona Therapeutics, has received multiple designations, including regenerative medicine advanced therapy and orphan drug status.
Ultragenyx Acquires Global Rights to Abeona's Gene Therapy for Sanfilippo Syndrome Type A
• Ultragenyx acquired global rights to ABO-102 (UX111), an AAV gene therapy from Abeona Therapeutics, targeting Sanfilippo syndrome type A (MPS IIIA).
• Interim data from the Transpher A trial showed preserved neurocognitive development in children treated early with ABO-102, with stabilized cognitive function.
• ABO-102 demonstrated statistically significant reductions in liver volume and heparan sulfate levels in treated patients, showing enzyme activity replacement.
• Ultragenyx plans to leverage its expertise in MPS diseases and gene therapy to accelerate the development and potential commercialization of ABO-102.
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