MedPath

FDA Approves Autolus' Obecabtagene Autoleucel (Aucatzyl) for R/R B-cell ALL

• The FDA has approved obecabtagene autoleucel (obe-cel), a CD19-directed CAR-T therapy, for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-cell ALL). • Approval was based on the Phase 1b/2 FELIX trial, which demonstrated overall complete remission rates above 60% and durable remissions exceeding 12 months. • Clinical holds on CARsgen's BCMA-directed CAR-T therapy zevorcabtagene autoleucel and Claudin18.2-directed CAR-T satricabtagene autoleucel have been removed by the FDA.

The FDA has granted approval to Autolus Therapeutics' obecabtagene autoleucel (Aucatzyl), also known as obe-cel, a CD19-directed genetically modified autologous T-cell immunotherapy. This approval marks a significant advancement in the treatment of adult patients with relapsed or refractory (r/r) B-cell precursor acute lymphoblastic leukemia (ALL).
The approval was supported by data from the pivotal Phase 1b/2 FELIX clinical trial (NCT04404660). The trial demonstrated promising efficacy, with overall complete remission (CR) rates exceeding 60%. Furthermore, patients who achieved complete remission within three months exhibited a median duration of remission beyond 12 months, highlighting the potential for durable responses with obe-cel.

CARsgen's CAR-T Therapies Advance

The FDA has lifted clinical holds on multiple CARsgen Therapeutics' CAR-T therapies, including zevorcabtagene autoleucel (zevor-cel), a BCMA-directed CAR-T for relapsed/refractory multiple myeloma (MM), and satricabtagene autoleucel (satri-cel), a Claudin18.2-directed CAR-T for gastric/pancreatic cancers. This decision allows CARsgen to proceed with clinical trials evaluating these therapies, offering potential new treatment options for patients with these cancers.

Other Cell and Gene Therapy Updates

  • A patient death occurred in Beam Therapeutics' BEACON trial (NCT05456880) evaluating BEAM-101 for sickle cell disease (SCD). The death was attributed to the busulfan-conditioning regimen, not the BEAM-101 therapy itself.
  • Neurogene's NGN-401 for Rett syndrome showed positive efficacy in a Phase 1/2 trial (NCT05898620), but a serious adverse event was reported in the high-dose cohort.
  • Abeona Therapeutics' BLA for prademagene zamikeracel (pz-cel; EB-101) for recessive dystrophic epidermolysis bullosa has been accepted by the FDA, with a PDUFA date of April 29, 2025.
  • Atamyo Therapeutics' ATA-200 for LGMD2C/R5 has received IND clearance, enabling a Phase 1b trial (NCT05973630).
  • Nippon Shinyaku has acquired commercialization rights for Atsena Therapeutics’ ATSN-101 for Leber Congenital Amaurosis (LCA1) in the US and Japan.
Subscribe Icon

Stay Updated with Our Daily Newsletter

Get the latest pharmaceutical insights, research highlights, and industry updates delivered to your inbox every day.

Related Clinical Trials

Anti-GPRC5D CAR-T Cells (CT071) in Participants With RRMM or RRpPCL

NCT06333509Not Yet RecruitingPhase 1
CARsgen Therapeutics Co., Ltd.
Posted 4/15/2024

ATA-200 Dose-escalation Gene Therapy Trial in Patients With LGMDR5

NCT05973630RecruitingPhase 1
Atamyo Therapeutics
Posted 2/15/2025

Related Topics

Gene Therapy Advances: From FDA Clearances to Clinical Trial Progress in Early 2025

The FDA cleared United Therapeutics' UKidney for US trials, a gene-edited porcine kidney product intended for end-stage renal disease via xenotransplant.
uniQure received the green light to proceed with the second dose cohort in their Phase 1/2 trial of AMT-162 for SOD1-ALS gene therapy.

FDA Accepts Abeona's BLA Resubmission for Pz-Cel in Recessive Dystrophic Epidermolysis Bullosa

The FDA has accepted Abeona Therapeutics' resubmitted Biologics License Application (BLA) for pz-cel, a gene therapy for recessive dystrophic epidermolysis bullosa (RDEB).
The FDA set a PDUFA target action date of April 29, 2025, for the decision on pz-cel, an autologous, cell-based gene therapy.

FDA Accepts Abeona's Gene Therapy BLA Resubmission for Recessive Dystrophic Epidermolysis Bullosa

The FDA has accepted Abeona Therapeutics' resubmitted Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for treating recessive dystrophic epidermolysis bullosa (RDEB).
Pz-cel is an autologous cell-based gene therapy designed to deliver the COL7A1 gene to wound sites, promoting collagen VII expression in RDEB patients.

FDA Approves Autolus' Aucatzyl (obe-cel) for Relapsed/Refractory B-ALL in Adults

The FDA has approved Autolus Therapeutics' Aucatzyl (obe-cel) for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL).
The approval was based on the results of the FELIX clinical trial, which demonstrated the efficacy of obe-cel in this patient population.

Gene and Cell Therapy Updates: FDA Actions, Clinical Advancements, and Strategic Acquisitions

Abeona Therapeutics has resubmitted its BLA to the FDA for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (RDEB) treatment.
Nanoscope Therapeutics' MCO-010 gene therapy demonstrated improved visual acuity in retinitis pigmentosa (RP) patients in a Phase 2b trial compared to placebo.

Abeona Therapeutics Resubmits BLA to FDA for Gene Therapy Pz-Cel in 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 follows a Type A meeting with the FDA, addressing Chemistry, Manufacturing, and Controls (CMC) information requested in a Complete Response Letter (CRL).

Pharma Update: FDA Eases CGT Path, Tharimmune Surges on EMA Feedback, Lexeo Dips on Alzheimer's Data

The FDA is creating a more accessible regulatory environment for cell and gene therapy companies, according to executives at BioFuture 2024.
Tharimmune's stock soared following positive feedback from the EMA regarding its phase II study of TH-104 for pruritus associated with primary biliary cholangitis (PBC).

Gene Editing Advances: Intellia, Poseida, YolTech, and KSQ Therapeutics Report Clinical Trial Updates

Intellia Therapeutics has initiated a pivotal Phase 3 trial (HAELO) of NTLA-2002 for hereditary angioedema, evaluating its ability to reduce HAE attacks through plasma kallikrein activity reduction.
Poseida Therapeutics announced positive interim Phase 1 data for P-BCMA-ALLO1 in heavily pre-treated multiple myeloma patients, demonstrating a 91% overall response rate.

FDA Grants Fast Track Status to Treg Therapy for Multiple Sclerosis and Orphan Drug Designation to Gene Therapy for Retinitis Pigmentosa

Abata Therapeutics' ABA-101, an autologous Treg cell therapy, received FDA Fast Track designation for progressive multiple sclerosis, aiming to reduce inflammation in the central nervous system.
Orgenesis Inc. reported encouraging data from a real-world study of ORG-101, a CD19 CAR-T therapy, showing high complete response rates in both adult and pediatric patients with B-cell ALL.

CAR-T Therapies Gain Ground in China and Beyond: Regulatory Approvals and Clinical Advancements

Legend Biotech's Carvykti (cilta-cel) receives approval in China for relapsed or refractory multiple myeloma after multiple lines of prior therapy.
JW Therapeutics' Carteyva (relma-cel) gains approval in China for treating relapsed or refractory mantle cell lymphoma in adult patients.

Reference News

[1]
Around the Helix: Cell and Gene Therapy Company Updates – November 13, 2024
cgtlive.com · Nov 14, 2024

The FDA approved obe-cel for r/r B-cell precursor acute lymphoblastic leukemia, while clinical holds were removed for CA...

© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy