CMS Assigns Reimbursement Code for Investigative Cell Therapy

The Centers for Medicare and Medicaid Services (CMS) has granted a product-specific procedure code ICD-10-PCS for prademagene zamikeracel (pz-cel), a cell therapy in development to treat patients with recessive dystrophic epidermolysis bullosa (RDEB).

RDEB is rare connective tissue disorder that causes severe and painful skin wounds and can lead to systemic complications. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning Type VII collagen.

Developed by Abeona Therapeutics, pz-cel is a cell therapy designed to incorporate the functional collagen-producing COL7A1 gene into a patient’s own skin cells.

If pz-cel receives U.S. marketing approval, the CMS code will allow for documentation, billing, and analysis of inpatient hospital procedures using pz-cel. The code will go into effect on Oct. 1, 2024.

Madhav Vasanthavada

“The favorable Medicare decisions complement the positive feedback we have heard from commercial payers, giving us confidence as we seek to secure strong access and reimbursement for all RDEB patients. The CMS coding assignment is a major step in simplifying inpatient hospital billing across all payers and increasing speed to access of pz-cel after its potential FDA approval,” Madhav Vasanthavada, chief commercial officer of Abeona, said in a news release.

In April 2024, the FDA has issued a complete response letter for the biologics license application (BLA) for pz-cell, asking for additional information for the Chemistry Manufacturing and Controls (CMC) requirements. Abeona officials said at the time that the information requested by the FDA pertains to validation requirements for certain manufacturing and release testing methods.

The CRL did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA. Regulators did not request any new clinical trials or clinical data to support the approval of pz-cel. Abeona officials said they are on target for resubmitting the BLA in the second half of 2024 with the required information.

The BLA is supported by clinical efficacy and safety data from the pivotal phase 3 VIITAL study and confirmatory evidence from a phase 1/2a study. Both studies revealed that a one-time application of pz-cel on large and chronic wounds delivered sustained wound healing and pain reduction.

Data from the VIITAL study were presented during the inaugural International Societies for Investigative Dermatology (ISID) Meeting in May 2023. Long-term follow up data up to eight years and quality of life data from the phase 1/2a study were published in Orphanet Journal of Rare Diseases.

In May 2024, long-term safety data from the VITAL trial with up to 11 years of follow-up were presented during a late-breaker session at the Society for Investigative Dermatology (SID) Annual Meeting.

Additionally, in July 2024, data on wound healing at various anatomical sites after pz-cel treatment were presented at the Society for Pediatric Dermatology (SPD) Annual Meeting.