Abeona Therapeutics Advances pz-cel Toward 2025 Commercialization

Abeona Therapeutics Moves Forward with pz-cel for RDEB

Abeona Therapeutics Inc. is diligently working towards the commercialization of its promising gene therapy, pz-cel, aimed at treating recessive dystrophic epidermolysis bullosa (RDEB). With the U.S. Food and Drug Administration's (FDA) acceptance of its Biologics License Application (BLA) resubmission, the company is on track to potentially launch this innovative treatment by 2025.

Key Developments and PDUFA Timeline

Recently, the FDA set a target action date of April 29, 2025, under the Prescription Drug User Fee Act (PDUFA) for pz-cel's BLA resubmission review. Abeona has been proactive, engaging with potential treatment centers and payors to facilitate patient access while ramping up its commercial readiness.

Critical Alignments with the FDA

In August 2024, Abeona held a significant Type A meeting with the FDA, resulting in a clear understanding of the necessary components for its BLA resubmission. The FDA did not request new clinical trials or additional data, indicating confidence in the existing clinical efficacy and safety data supporting pz-cel's application.

Financial Position and Cash Runway

As of September 30, 2024, Abeona reported $110 million in cash, cash equivalents, and other liquid assets. This financial stability provides a solid runway as the company gears up for commercial operations beyond 2026. The increment of cash following recent successes contributes towards funding the necessary operational expenses and clinical advancements.

Payer Engagement and Reimbursement Strategies

Abeona's engagement with the Centers for Medicare and Medicaid Services (CMS) has been fruitful, seeing approval for a product-specific procedure code for pz-cel, alongside favorable reimbursement outcomes. These decisions are expected to streamline patient access and provide essential support for hospital billing related to cell and gene therapies.

Investment in Facilities and Manufacturing Capabilities

In line with its growth strategy, Abeona secured a lease for additional manufacturing space. This expansion is poised to enhance the production capabilities of pz-cel to meet anticipated demand, showcasing the company's commitment to ensuring scalable operations.

Strengthening Intellectual Property

October also brought news of newly issued patents from the United States Patent and Trademark Office, protecting pz-cel's technology. The recently issued U.S. Patent No. 12,110,504 and other pending patents solidify Abeona's position in the gene therapy arena, allowing it to safeguard its innovative approach to treating RDEB.

Pipeline and Collaborative Ventures

Abeona is not halting its progress with pz-cel but is actively expanding its pipeline. The company announced a non-exclusive agreement with Beacon Therapeutics aimed at advancing its AAV204 capsid for ophthalmological applications, further diversifying its research portfolio.

Focus on Rehabilitation and Overall Strategy

The roadmap ahead includes not only the anticipated approval and launch of pz-cel but also evolving partnerships and development programs. Abeona is keen on capitalizing on its expertise in cell and gene therapies to address critical unmet medical needs across various pathologies.

Frequently Asked Questions

What is pz-cel and its significance?

Pz-cel is an autologous cell-based gene therapy developed by Abeona Therapeutics for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin disorder.

What is the PDUFA action date for pz-cel?

The FDA has set a target action date of April 29, 2025, for the resubmission review of the BLA for pz-cel.

How is Abeona preparing for the commercial launch of pz-cel?

Abeona is onboarding high-volume treatment centers, engaging with payors for reimbursement strategies, and educating stakeholders to ensure a successful launch of pz-cel.

What recent financial results has Abeona disclosed?

Abeona reported $110 million in cash, cash equivalents, and short-term investments as of September 30, 2024, sufficient to support operations into 2026.

Are there any recent collaborations involving Abeona?

Abeona has entered a non-exclusive collaboration with Beacon Therapeutics to evaluate new gene therapies for select ophthalmology indications.

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