Biologics

Pierre Fabre Resubmits Tabelecleucel BLA

Pierre Fabre Pharmaceuticals (PFP) says its tabelecleucel BLA has been resubmitted 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.

PFP 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, PFP 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.

Multiple Bentley Laboratories Violations

A 3/16-3/27 FDA inspection at Bentley Laboratories, a contract drug manufacturing firm in Edison, NJ, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs and other violations. A 9/17 Warning Letter says the specific CGMP violations were: · failure to thoroughly investigate any

6 Observations on Right Value FDA-483

FDA has released the Form FDA-483 issued following an 8/24-9/8 inspection at Right Value Drug Stores, an Irving, TX outsourcing facility. The six inspection observations were: * failure to follow procedures designed to prevent microbiological contamination of drug products purporting to be sterile; * failure of laboratory controls

HHS Initiative Aimed at Cutting Drug Development to Under 4 Years

HHS’ Advanced Research Projects Agency for Health has launched a funding initiative aimed at dramatically shortening clinical development of drugs and biologics through adaptive platform trials, artificial intelligence, and real-time data analysis. The “Simulation-augmented, Real-time Platform Adaptive Seamless Trials (SURPASS)” program is seeking technologies and trial approaches

Roche Wins Priority Review for Multiple Sclerosis Drug

FDA has accepted for priority review a Roche NDA for fenebrutinib, an investigational non-covalent Bruton’s tyrosine kinase (BTK) inhibitor for treating relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS). The submission is based on data from the Phase 3 FENhance 1 and 2 RMS studies and

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Multiple Bentley Laboratories Violations

6 Observations on Right Value FDA-483

HHS Initiative Aimed at Cutting Drug Development to Under 4 Years

Roche Wins Priority Review for Multiple Sclerosis Drug