Abeona Surges After FDA Clears First Gene Therapy For Severe Genetic Skin Disease

Abeona Therapeutics Inc.'s (NASDAQ:ABEO) stock traded higher on Wednesday.

The Food and Drug Administration (FDA) on Tuesday approved Abeona’s Zevaskyn (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, as the first and only autologous cell-based gene therapy for wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB).

Zevaskyn is the only FDA-approved product to treat RDEB wounds with a single application.

Zevaskyn consists of a patient's skin cells (keratinocytes) genetically modified to produce functional Type VII collagen. Zevaskyn sheets are surgically applied to the patient's wounded areas. In a single application, up to 12 credit card-sized sheets can be combined to cover large areas or applied to multiple distinct wounds, allowing for significant coverage of affected body areas.

HC Wainwright writes the approval was granted on time, showing that the agency can still meet deadlines despite recent disruptions. Analyst Raghuram Selvaraju on Wednesday wrote that pz-cel is well-positioned to become the leading treatment for the most severe cases of recessive dystrophic epidermolysis bullosa (RDEB).

It may also be used on its own or alongside other approved treatments like Krystal Biotech, Inc.’s (NASDAQ:KRYS) Vyjuvek (beremagene geperpavec) in certain patients.

On Monday, the European Commission approved Vyjuvek for wounds in patients with dystrophic epidermolysis bullosa who have mutations in the collagen type VII alpha 1 chain (COL7A1) gene, starting from birth.

• The timing for the availability of VYJUVEK in individual countries will depend on multiple factors, including the completion of reimbursement procedures.
• The company is planning for its first European launch in Germany in mid-2025.

The list price for Zevaskyn has been revealed at $3.1 million per treatment, much higher than the $800,000 we previously assumed, the analyst said.

Based on this new price, the analyst estimates that treating 10–15 patients by the end of 2025 could bring Abeona over $30 million in sales.

HC Wainwright’s updated revenue forecast for 2025 is now $31.6 million, increasing to $130.4 million in 2026.

HC Wainwright continues to project peak U.S. sales of around $600 million. Abeona believes there are about 750 potential patients in the target population, which could mean a market opportunity of over $2 billion if all were treated with Zevaskyn.

Analyst Selvaraju maintains the Buy rating and raise the price target from $15 to $20 per share, reflecting increased confidence in the drug.

Price Action: ABEO stock was up 23.77% to $6.56 on Wednesday.

Read Next:

• Fresh Del Monte Stock Falls As Q1 Sales, Earnings Miss Expectations

Photo: Shutterstock