FDA greenlights Abeona's Zevaskyn gene therapy for epidermolysis bullosa

The US Food and Drug Administration (FDA) has officially approved Abeona Therapeutics' Zevaskyn (prademagene zamikeracel), marking a significant advancement in the treatment of recessive dystrophic epidermolysis bullosa (RDEB). This approval signifies a monumental first as Zevaskyn becomes the inaugural autologous cell-based gene therapy to receive the green light in the United States for addressing wounds in both adult and pediatric sufferers of this rare skin disorder.

RDEB is a debilitating genetic condition that disrupts the connective tissue, resulting in extremely fragile skin prone to extensive blistering and severe wound formation. In many cases, these wounds cover more than 30% of an individual's body. At the core of Zevaskyn's therapeutic strategy is the use of a patient’s own skin cells, which are genetically altered to produce type VII collagen. This is a crucial protein that individuals with RDEB cannot naturally produce due to mutations in both copies of the COL7A1 gene. The treatment is delivered surgically in the form of a sheet applied directly to the patient's wounds, with availability in the US anticipated by the third quarter of this year.

Vish Seshadri, the CEO of Abeona, emphasized the transformative nature of this approval in the landscape of RDEB treatment. He noted that through a single surgical intervention, Zevaskyn offers those afflicted by RDEB the chance for significant wound healing and pain relief, even in the most challenging cases.

The FDA's decision to approve Zevaskyn was heavily supported by results from the phase 3 VIITAL study. This study underscored the therapy’s effectiveness, showing remarkable wound healing and pain reduction after just one application. Specifically, in the trial, 81% of 43 large and chronic wounds treated with Zevaskyn demonstrated at least 50% healing at the six-month mark. This is a stark contrast to the 16% healing rate observed in 43 matched control wounds that received standard care.

Further reinforcing Zevaskyn’s efficacy, a phase 1/2a study conducted on 38 chronic wounds in seven patients revealed that a single application of the therapy led to sustained improvements at the treated sites, with observations extending over a median follow-up period of nearly seven years. Importantly, Zevaskyn exhibited a favorable safety profile in both studies, with no serious adverse events related to the treatment reported.

Abeona’s chief commercial officer, Madhav Vasanthavada, expressed confidence in the strength of the clinical trial data, highlighting Zevaskyn’s potential to deliver enduring positive outcomes with just one treatment session. He emphasized Abeona’s commitment to collaborating with both commercial and government healthcare payers to establish outcome-based agreements. This approach aims to support the delivery of Zevaskyn to patients more swiftly, ensuring that those in need can access the therapy based on its promising benefits.