Biologics

FDA Accepts Pierre Fabre BLA Resubmission for Review

FDA has accepted for review a Pierre Fabre Pharmaceuticals BLA resubmission with a proposed indication as monotherapy for treating adult and pediatric patients 2 years of age and older with Epstein-Barr virus-positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. The agency has set a user fee review action target date of 3/31/2027.

The company resubmitted the BLA based on FDA input from an April Type A meeting that was held to discuss a recent complete response letter. According to the company, FDA agreed that a single-arm study using an appropriate historical control, if conducted in a prespecified manner, could serve as an “adequate and well-controlled” study capable of supporting a future marketing application for the therapy.

In January, FDA issued the companies a second complete response letter on the BLA, citing the need for additional clinical evidence. That application had been resubmitted after what the companies described as alignment with FDA on the criteria for resubmission following an earlier complete response letter issued 1/2025, which identified a single good manufacturing practice deficiency and raised no concerns about safety, efficacy, or trial design, according to the companies.

“The resubmission includes an updated dataset with additional patients and longer follow up from the ongoing pivotal Phase 3 single arm ALLELE study of tabelecleucel in adults and children two years of age and older with relapsed/refractory EBV+ PTLD following hematopoietic cell transplant (HCT) or solid organ transplant (SOT),” the company says. It also includes additional supplemental data from expanded access programs, a separate clinical study, and commercial experience in Europe.

Under a global partnership, Pierre Fabre handles the development, manufacturing, and commercialization of Atara's T-cell therapy tabelecleucel, in most markets, including the U.S. The therapy is intended to treat patients with Epstein-Barr virus–positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have failed standard treatment options.

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Bioresearch Monitoring Issue at Lovelace Institute