AscellaHealth, a global specialty pharmaceutical partner, has announced the successful launch of a comprehensive support program for ZEVASKYN™ (prademagene zamikeracel), the first FDA-approved autologous cell sheet-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB). The collaboration with Abeona Therapeutics Inc. (Nasdaq: ABEO) demonstrates how tailored infrastructure can enable successful commercialization of complex cell and gene therapies.
Groundbreaking Partnership Model
The partnership between AscellaHealth and Abeona Therapeutics represents a new approach to launching first-in-class gene therapies. Working as a collaborative partner, AscellaHealth designed and executed patient-centric, end-to-end solutions to address unique clinical, operational and reimbursement needs for this new-to-world autologous cell-based gene therapy.
"Abeona's groundbreaking cell-based gene therapy demanded a HUB partner who could build tailored infrastructure from the ground up," said William White, Vice President of Market Access at Abeona Therapeutics. "AscellaHealth's comprehensive expertise in navigating this complex journey, from patient and provider engagement to optimizing reimbursement for this first-of-its-kind treatment, made them the unequivocal partner of choice."
AbeonaAssist: Customized Patient Support Program
The collaboration led to the creation of AbeonaAssist™, a highly customized patient support program that creates a seamless experience for patients, caregivers and healthcare providers. This bespoke program was built from scratch to address the complex logistics required for personalized, autologous therapy that demands tight coordination throughout the treatment process.
Bill Oldham, Chairman and President of AscellaHealth, emphasized the strategic nature of the partnership: "We value our strategic partnership with Abeona which is built upon an aligned vision, a foundation of shared goals and a clear understanding of what can be achieved through collaboration. Together, we have created synergies that not only benefit both organizations, but most importantly, patient access to treatment and optimized clinical outcomes."
Measurable Clinical Outcomes
AscellaHealth's integrated patient services and HUB model are proving instrumental in achieving optimal results for the launch of ZEVASKYN. The partnership has demonstrated enhanced compliance, retention and satisfaction rates, underscoring the effectiveness of tailored programs that consistently deliver measurable outcomes for patients with complex, chronic conditions and rare disease.
The results highlight several key benefits:
- Higher patient compliance and satisfaction through personalized support
- Improved retention rates during critical therapy windows
- Efficient reimbursement navigation that reduces approval delays and billing friction
ZEVASKYN: First-in-Class Gene Therapy
ZEVASKYN is the first and only autologous cell sheet-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a severe skin disease caused by a defect in both copies of the COL7A1 gene resulting in the inability to produce functional type VII collagen.
Without functional type VII collagen and anchoring fibrils, the skin is fragile and blisters easily, leading to wounds that continually open and close, or fail to heal altogether. Patients often have large open wounds that can lead to serious life-threatening complications.
ZEVASKYN incorporates the functional type VII collagen-producing COL7A1 gene into a patient's own skin cells, ex vivo, using a replication-incompetent retroviral vector to produce functional type VII collagen in treated wounds. The therapy has demonstrated clinically meaningful wound healing and pain reduction with a single surgical application.
Safety Profile and Monitoring Requirements
While ZEVASKYN shows significant therapeutic promise, it comes with specific safety considerations that require ongoing monitoring. Serious allergic reactions can occur, with symptoms including itching, swelling, hives, difficulty breathing, runny nose, watery eyes, or nausea. In rare cases, severe anaphylaxis may occur.
There is a potential risk that treatment with ZEVASKYN may contribute to the development of cancer because of how the therapy works, requiring patients to be monitored for the rest of their lives to check for any signs of cancer. Additionally, since ZEVASKYN is made using human and animal materials, although these materials are tested before use, the risk of passing on infections cannot be eliminated.
The most common side effects reported are pain from the procedure and itching at the treatment site.
Manufacturing and Development Pipeline
Abeona Therapeutics operates a fully integrated cGMP cell and gene therapy manufacturing facility in Cleveland, Ohio, which serves as the manufacturing site for ZEVASKYN commercial production. The company's development portfolio also features adeno-associated virus (AAV)-based gene therapies for ophthalmic diseases with high unmet medical need, utilizing novel, next-generation AAV capsids to improve tropism profiles for various devastating diseases.
