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News
Pierre Fabre Pharmaceuticals has resubmitted its biologics license application (BLA) for tabelecleucel to the FDA — its third attempt — for treating EBV-positive post-transplant lymphoproliferative disease (PTLD). The resubmission follows two prior rejections and includes updated trial data showing a 50.7% response rate. Patients with this ultra-rare cancer face survival measured in weeks to months after initial treatment fails.
Pierre Fabre Pharmaceuticals (PFP) has resubmitted its BLA to the FDA for tabelecleucel (Ebvallo), an off-the-shelf EBV-specific T-cell immunotherapy, targeting adults and children (2+) with EBV-positive post-transplant lymphoproliferative disease (PTLD) who've failed at least one prior therapy. This is the third submission after two complete response letters (CRLs) — the first over a manufacturing facility inspection issue, the second because the FDA questioned whether the single-arm ALLELE trial was sufficient for approval.
The path forward was cleared after a Type A meeting in April 2026, where the FDA agreed that a single-arm study paired with a prespecified historical control could meet the bar for an adequate and well-controlled study. The updated ALLELE dataset — now covering 75 patients — anchors this submission, alongside data from expanded access programs and commercial experience in Europe, where tabelecleucel is already approved.
By the Numbers:
Why it matters: EBV+ PTLD is an ultra-rare and deadly cancer that strikes transplant recipients at their most vulnerable. With no FDA-approved options and survival often measured in weeks, tabelecleucel's resubmission represents a critical lifeline — and a test case for how regulators handle single-arm trial evidence in rare diseases.
