Abeona Therapeutics® Announces FDA Acceptance of BLA Resubmission of Pz-cel for the Treatment of Recessive Dystrophic Epidermolysis Bullosa
Abeona Therapeutics announced that the FDA has accepted its resubmitted Biologics License Application (BLA) for pz-cel, an investigational autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB). The FDA set a PDUFA target action date of April 29, 2025. The BLA resubmission includes clinical data from the Phase 3 VIITAL™ study and a Phase 1/2a study with up to 8 years of follow-up. If approved, pz-cel would be the first autologous, cell-based gene therapy for RDEB, designed to provide collagen VII expression through COL7A1 gene integration.
Abeona Therapeutics ha annunciato che la FDA ha accettato la sua richiesta di licenza biologica (BLA) ripresentata per pz-cel, una terapia genica cellulare autologa in fase di sperimentazione per l'epidermolisi bollosa distrofica recessiva (RDEB). La FDA ha fissato una data di azione obiettivo PDUFA per il 29 aprile 2025. La ripresentazione della BLA include dati clinici dello studio di Fase 3 VIITAL™ e di uno studio di Fase 1/2a con follow-up fino a 8 anni. Se approvato, pz-cel sarebbe la prima terapia genica basata su cellule autologhe per RDEB, progettata per fornire espressione di collagene VII attraverso l'integrazione del gene COL7A1.
Abeona Therapeutics anunció que la FDA ha aceptado su solicitud de licencia biológica (BLA) re presentada para pz-cel, una terapia génica autóloga basada en células en investigación para la epidermólisis bullosa distrófica recesiva (RDEB). La FDA estableció una fecha de acción objetivo PDUFA para el 29 de abril de 2025. La re presentación de la BLA incluye datos clínicos del estudio de Fase 3 VIITAL™ y un estudio de Fase 1/2a con un seguimiento de hasta 8 años. Si se aprueba, pz-cel sería la primera terapia génica basada en células autólogas para RDEB, diseñada para proporcionar expresión de colágeno VII a través de la integración del gen COL7A1.
Abeona Therapeutics는 FDA가 pz-cel에 대한 재제출된 생물학적 약품 허가 신청(BLA)을 승인했다고 발표했습니다. pz-cel은 열성형성 표피 수포증(RDEB) 치료를 위한 연구 중인 자가 세포 기반 유전자 요법입니다. FDA는 2025년 4월 29일을 PDUFA 목표 조치 날짜로 설정했습니다. BLA 재제출에는 VIITAL™ 3상 연구 및 최대 8년의 후속 연구를 포함하는 1/2a 단계의 임상 데이터가 포함되어 있습니다. 승인을 받으면 pz-cel은 RDEB를 위한 최초의 자가 세포 기반 유전자 치료제가 되며, COL7A1 유전자 통합을 통해 콜라겐 VII 발현을 제공하도록 설계되었습니다.
Abeona Therapeutics a annoncé que la FDA a accepté sa demande de licence biologique (BLA) révisée pour pz-cel, une thérapie génique autologue à base de cellules en cours d'investigation pour l'épidermolyse bulleuse dystrophique récessive (RDEB). La FDA a fixé une date d'action cible PDUFA au 29 avril 2025. La nouvelle soumission de la BLA comprend des données cliniques de l'étude de phase 3 VIITAL™ et d'une étude de phase 1/2a avec un suivi allant jusqu'à 8 ans. Si elle est approuvée, pz-cel serait la première thérapie génique autologue à base de cellules pour RDEB, conçue pour fournir une expression de collagène VII par le biais de l'intégration du gène COL7A1.
Abeona Therapeutics hat angekündigt, dass die FDA den erneut eingereichten Antrag auf biologische Lizenz (BLA) für pz-cel akzeptiert hat, eine experimentelle autologe zellbasierte Gentherapie für die rezessive dystrophische Epidermolysis bullosa (RDEB). Die FDA setzte ein PDUFA-Zielaktionsdatum auf den 29. April 2025. Die erneute Einreichung der BLA umfasst klinische Daten aus der Phase-3-Studie VIITAL™ sowie eine Phase-1/2a-Studie mit bis zu 8 Jahren Nachbeobachtung. Bei Genehmigung wäre pz-cel die erste autologe zellbasierte Gentherapie für RDEB, die darauf abzielt, eine Kollagen-VII-Expression durch die Integration des COL7A1-Gens bereitzustellen.
- FDA acceptance of BLA resubmission for pz-cel
- Potential to be first autologous cell-based gene therapy for RDEB
- Company may be eligible for Priority Review Voucher upon approval
- Long-term clinical data available with up to 8 years of follow-up
- Extended timeline to potential approval with PDUFA date of April 29, 2025
Insights
FDA assigns PDUFA target action date of April 29, 2025
CLEVELAND, Nov. 12, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review Abeona’s resubmission of its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel), its investigational autologous cell-based gene therapy, as a potential new treatment for recessive dystrophic epidermolysis bullosa (RDEB). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 29, 2025.
“The FDA acceptance of our BLA resubmission moves us one step closer to providing pz-cel as a differentiated treatment option to address the persistent unmet needs of people with RDEB in the U.S.,” said Vish Seshadri, Chief Executive Officer of Abeona. “We look forward to continuing to work with the FDA to finalize the review of the pz-cel application.”
The BLA resubmission is supported by clinical efficacy and safety data after a one-time administration of pz-cel from the pivotal Phase 3 VIITAL™ study (NCT04227106) and a Phase 1/2a study (NCT01263379) with up to 8 years of follow-up. If approved, pz-cel would be the first autologous, cell-based gene therapy for RDEB, and the first RDEB treatment designed to provide collagen VII expression at wound sites via a stably integrated copy of the COL7A1 gene.
The Company’s BLA for pz-cel was previously accepted for Priority Review by the FDA for patients with RDEB. Abeona may be eligible for a Priority Review Voucher (PRV) should pz-cel be approved.
About prademagene zamikeracel (pz-cel)
Prademagene zamikeracel (pz-cel), Abeona’s investigational autologous, COL7A1 gene therapy, is currently being developed for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin disease caused by a mutation in both copies of the COL7A1 gene. As a result of this defect, cells are unable to express functional collagen VII protein which is needed to form anchoring fibrils that bond the epidermis to the dermis. Lack of anchoring fibrils leads to fragile skin that blisters easily and patients suffer from years of painful wounds, itch and increased risk of infection and squamous cell carcinoma. Pz-cel is made from patients' own skin cells that are genetically corrected with a functional COL7A1 gene integrated into the skin cells’ genome to express collagen VII. These gene-corrected cells are expanded to form keratinocyte sheets to cover wound areas in a single surgical application. Pz-cel has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations by the U.S. FDA.
About Abeona Therapeutics
Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Prademagene zamikeracel (pz-cel) is Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa. The Company’s fully integrated cell and gene therapy cGMP manufacturing facility served as the manufacturing site for pz-cel used in its Phase 3 VIITAL™ trial, and is capable of supporting commercial production of pz-cel upon FDA approval. The Company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. For more information, visit www.abeonatherapeutics.com.
Forward-Looking Statements
This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attempted to identify forward-looking statements by such terminology as “may,” “will,” “believe,” “anticipate,” “expect,” “intend,” “potential,” and similar words and expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to, the timing and outcome of the FDA’s review of our BLA resubmission for pz-cel; the FDA’s grant of a Priority Review Voucher upon pz-cel approval; continued interest in our rare disease portfolio; our ability to enroll patients in clinical trials; the outcome of future meetings with the FDA or other regulatory agencies, including those relating to preclinical programs; the ability to achieve or obtain necessary regulatory approvals; the impact of any changes in the financial markets and global economic conditions; risks associated with data analysis and reporting; and other risks disclosed in the Company’s most recent Annual Report on Form 10-K and subsequent periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.
FAQ
When is the PDUFA date for Abeona's (ABEO) pz-cel BLA?
What clinical studies support Abeona's (ABEO) pz-cel BLA resubmission?
What is unique about Abeona's (ABEO) pz-cel treatment for RDEB?
