J&J Multiple Myeloma Drug Regimen Receives Proactive National Priority Review Voucher from the FDA
Expedited Review Aims to Accelerate Decision for Combination Therapy with Breakthrough Efficacy Data
The U.S. Food and Drug Administration (FDA) has “proactively” awarded Johnson & Johnson (J&J) a Commissioner’s National Priority Voucher (CNPV) for a multiple myeloma combination therapy. This voucher speeds up the review process, targeting a decision one to two months following application submission, instead of the standard timeframe of up to 10 months.
The announcement follows the presentation of Phase 3 clinical trial results (MajesTEC-3) for the combination at the American Society of Hematology annual meeting. J&J, which had described the results as “unprecedented,” had already submitted an application for the two-drug combination intended for multiple myeloma patients who had received one to three prior lines of therapy.
FDA Commissioner, Dr. Martin Makary, stated that the FDA leadership read the study shortly after the results were published and proactively contacted the company about the CNPV because “When a treatment demonstrates outstanding trial results, we have a duty to patients to move swiftly.”
The MajesTEC-3 trial showed that the new combination regimen reduced the risk of death by 54% compared to traditional regimens. The progression-free survival (PFS) advantage of the new cocktail reached an impressive 83%. The overall survival (OS) curve for the combination suggests its potential as a functional cure for patients.
Analysts called the MajesTEC-3 results “exceptional.” However, a perceived weak point in the data was the exclusion of patients who were refractory to Darzalex, a drug that has become a standard first-line therapy. Analysts expressed concern that only 5% of the MajesTEC-3 patients had previously received Darzalex, potentially impacting the generalizability of the treatment’s efficacy to the refractory group.
The CNPV granted to J&J’s regimen is the 16th since the pilot program’s inception. The program prioritizes drugs aligning with U.S. national priorities, such as delivering innovative cures and supporting domestic drug manufacturing. Despite the priority status, questions have been raised regarding the legality of the initiative and the rigor of the FDA’s evaluation process for these drugs.
