Resubmission of BLA for pz-cel Accepted
The FDA accepted the resubmission for review and set a PDUFA date of April 29, 2025, indicating progress towards potential approval.
New U.S. Patents Issued
A new U.S. patent for pz-cel was issued with an expiration date of 2037, and another patent will issue relating to its packaging and transport system, expiring in 2040.
Commercial Launch Preparations
A strong commercialization team has been assembled, and 5 EB centers are targeted for launch. Approximately 750 pz-cel-eligible U.S. RDEB patients have been identified.
Favorable Medicare Reimbursement
CMS granted a product-specific procedure code and favorable MS-DRG 018 assignment, supporting reimbursement for Medicare RDEB patients.
Financial Position
Cash resources of $110 million as of September 30, 2024, expected to fund operations into 2026.