Abeona Therapeutics® Completes Pz-cel Biologics License Application Resubmission to U.S. Food and Drug Administration
Abeona Therapeutics has resubmitted its Biologics License Application (BLA) to the FDA for pz-cel, its investigational gene therapy for recessive dystrophic epidermolysis bullosa (RDEB). The resubmission addresses Chemistry Manufacturing and Controls items identified in the previous Complete Response Letter (CRL) from April 2024. The CRL did not identify any clinical efficacy or safety concerns, and no new clinical trials were requested. The BLA is supported by data from the Phase 3 VIITAL™ study and a Phase 1/2a study. The FDA's review period will be either 2 or 6 months, depending on classification as Class 1 or Class 2.
Abeona Therapeutics ha presentato nuovamente la sua Domanda di Licenza Biologica (BLA) alla FDA per pz-cel, la sua terapia genica investigativa per l'epidermolisi bollosa distrofica recessiva (RDEB). La nuova presentazione affronta gli elementi di Chimica, Produzione e Controlli identificati nella precedente Lettera di Risposta Completa (CRL) dell'aprile 2024. La CRL non ha evidenziato preoccupazioni relative all'efficacia clinica o alla sicurezza, e non sono stati richiesti nuovi studi clinici. La BLA è sostenuta da dati dello studio di Fase 3 VIITAL™ e di uno studio di Fase 1/2a. Il periodo di revisione della FDA sarà di 2 o 6 mesi, a seconda della classificazione come Classe 1 o Classe 2.
Abeona Therapeutics ha volvido nuevamente su Solicitud de Licencia Biológica (BLA) a la FDA para pz-cel, su terapia génica en investigación para la epidermólisis bullosa distrofica recesiva (RDEB). La nueva presentación aborda elementos de Química, Fabricación y Control identificados en la anterior Carta de Respuesta Completa (CRL) de abril de 2024. La CRL no identificó preocupaciones de eficacia o seguridad clínica, y no se solicitaron nuevos ensayos clínicos. La BLA está respaldada por datos del estudio de Fase 3 VIITAL™ y un estudio de Fase 1/2a. El período de revisión de la FDA será de 2 o 6 meses, dependiendo de la clasificación como Clase 1 o Clase 2.
아베오나 테라퓨틱스는 FDA에바이오로직스 라이센스 신청서(BLA)를 재제출했습니다라며, 이는 열성형 피부 수포증(RDEB)을 위한 조사 중인 유전자 치료제인 pz-cel과 관련이 있습니다. 이번 재제출은 2024년 4월의 이전 완전 응답서(CRL)에서 확인된 화학, 제조 및 관리 항목을 다루고 있습니다. CRL은 임상적 효능이나 안전성에 대한 우려를 제기하지 않았습니다, 새 임상 시험은 요청되지 않았습니다. BLA는 VIITAL™ 연구의 3상 데이터와 1/2a상 연구의 데이터를 바탕으로 하고 있습니다. FDA의 검토 기간은 클래스 1 또는 클래스 2로 분류에 따라 2개월 또는 6개월이 될 것입니다.
Abeona Therapeutics a soumis à nouveau sa Demande de Licence Biologique (BLA) à la FDA pour pz-cel, sa thérapie génique expérimentale pour l'épidermolyse bulleuse dystrophique récessive (RDEB). La nouvelle soumission traite des éléments de chimie, fabrication et contrôle identifiés dans la précédente Lettre de Réponse Complète (CRL) d'avril 2024. La CRL n'a pas soulevé de préoccupations concernant l'efficacité ou la sécurité clinique, et aucun nouvel essai clinique n'a été requis. La BLA est soutenue par des données de l'étude de Phase 3 VIITAL™ et d'une étude de Phase 1/2a. La période d'examen de la FDA sera de 2 ou 6 mois, selon la classification en Classe 1 ou Classe 2.
Abeona Therapeutics hat seinen biologischen Lizenzantrag (BLA) erneut bei der FDA eingereicht für pz-cel, seine experimentelle Gentherapie für die rezessive dystrophe Epidermolysis bullosa (RDEB). Die Neubeantragung behandelt chemische, herstellungs- und kontrollelemente, die in dem vorherigen vollständigen Antwortschreiben (CRL) vom April 2024 identifiziert wurden. Die CRL hat keine Bedenken hinsichtlich der klinischen Wirksamkeit oder Sicherheit identifiziert, und es wurden keine neuen klinischen Studien angefordert. Der BLA wird durch Daten aus der Phase-3-Studie VIITAL™ und einer Phase-1/2a-Studie unterstützt. Der Prüfungszeitraum der FDA beträgt entweder 2 oder 6 Monate, abhängig von der Klassifizierung als Klasse 1 oder Klasse 2.
- No clinical efficacy or safety concerns identified by FDA in previous CRL
- No additional clinical trials required for approval
- BLA supported by completed Phase 3 and Phase 1/2a study data
- Previous BLA rejection in April 2024 due to CMC requirements
- Uncertain timeline for FDA review (2-6 months pending classification)
Insights
The BLA resubmission for pz-cel represents a significant regulatory milestone for Abeona Therapeutics. The key positive aspects are that the FDA did not identify any clinical efficacy or safety concerns in the previous CRL and no additional clinical trials were requested. The resubmission addresses only Chemistry Manufacturing and Controls (CMC) requirements, which are typically more straightforward to resolve than clinical issues.
The timeline implications are favorable - if classified as a Class 1 resubmission, FDA review could complete within 2 months; even with a Class 2 designation, the 6-month review period is relatively short. The acceptance decision expected within 14 days will be a important near-term catalyst.
With Phase 3 VIITAL study data supporting the application and previous regulatory alignment through the Type A meeting, the probability of approval appears stronger than the first submission. For RDEB patients who currently lack approved treatments, pz-cel could become a first-in-class therapy, potentially capturing significant market share.
CLEVELAND, Oct. 29, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that the Company has resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA or Agency) for prademagene zamikeracel (pz-cel), its investigational autologous cell-based gene therapy, as a potential new treatment for patients with recessive dystrophic epidermolysis bullosa (RDEB).
“We have worked closely with the FDA in preparing for the pz-cel BLA resubmission and thank the Agency for their ongoing guidance,” said Vish Seshadri, Chief Executive Officer of Abeona. “We have incorporated the Agency’s feedback and are confident that our resubmission package addresses all the Chemistry Manufacturing and Controls items identified in the Complete Response Letter, including observations from the completed Pre-License Inspection of our manufacturing facility. Upon acceptance of the BLA, we look forward to assisting the FDA in the completion of its review.”
The BLA resubmission follows the Company’s Type A meeting in August 2024, where Abeona aligned with the FDA on the content of the resubmission, including additional information to satisfy all Chemistry Manufacturing and Controls (CMC) requirements outlined in the Complete Response Letter (CRL) received in April 2024. In the CRL, the FDA required that certain additional information needed to satisfy CMC requirements be provided before the application could be approved. The CRL did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA, and the FDA did not request any new clinical trials or clinical data to support the approval of pz-cel. The BLA resubmission is supported by clinical efficacy and safety data from the pivotal Phase 3 VIITAL™ study (NCT04227106) and a Phase 1/2a study (NCT01263379).
Upon acceptance of the BLA resubmission, Abeona expects the FDA to set a new Prescription Drug User Fee Act (PDUFA) target action date for pz-cel. The review period for a resubmitted BLA is either two or six months from the resubmission date depending on the FDA's classification of a resubmission as Class 1 or Class 2, respectively. The FDA’s decision on acceptance of a BLA resubmission is typically made within 14 calendar days following the resubmission.
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
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