Quality/GMPs

FDA leadership initiated rapid drug review programs, enhanced transparency by publishing decision letters in real time, and strategically integrated AI into agency operations.

FDA’s approval of the first Wiskott-Aldrich gene therapy signals a shift in manufacturing and regulatory standards for rare-disease CGTs.

FDA’s clearance of a chemically enhanced stem cell therapy for severe aplastic anemia drives forward cell therapy development and manufacturing innovation.

Priority review, RMAT, and breakthrough status were key regulatory actions intensely accelerating advanced next-gen therapy development in 2025.

Manufacturing biologics is a complex task. Mitigating risk early in the design of manufacturing facilities and the qualification of equipment minimizes future complications.

FDA’s new-indication approval for lisocabtagene maraleucel in marginal zone lymphoma boosts engineered T-cell treatment for relapsed lymphoma.

FDA outlines reduced primate testing for monoclonal antibodies, signaling a shift toward modern evaluation tools that may influence drug development practices.

FDA’s internal findings raise new concerns over pediatric vaccine safety and signal potential regulatory shifts affecting future COVID-19 vaccine development.

Regulators and manufacturers face new uncertainty as shifting CDC vaccine language raises concerns for evidence-based oversight and biopharma compliance.

Microfluidic rapid-testing advances enhance real-time QC, strengthen compliance, and speed contamination detection across modern biomanufacturing.

The EC approves the world's first liquid IV infliximab biosimilar, which cuts preparation time by 51% and offers up to €2.6 million (US$3.0 million) in potential annual savings.

Biogen reports CHMP support for an enhanced nusinersen regimen, signaling potential shifts in SMA treatment strategies and global regulatory decisions.

FDA’s newly introduced review route enables faster clearance of custom genetic and cell treatments, reshaping rare-disease development and manufacturing strategies.

This second part of a two-part article provides a clear understanding of microbiological load reduction during cleaning processes in the non-sterile pharmaceutical manufacturing.

The regulatory progress for AAVantgarde’s AAVB-039 underscores advances in dual-vector gene therapy, as well as scalable manufacturing and efficient rare-disease clinical development.

This new draft guidance mandates efficient, long-term CGT postapproval monitoring using real-world evidence, registries, and decentralized models, all critical for biopharma.

This innovative trial guidance accelerates cell and gene therapy development for rare diseases, boosting biopharma efficiency and data maximization.

FDA's Center for Biologics Evaluation and Research has released updated draft recommendations for sponsors of cell therapies, gene therapies, and tissue products.

ACIP votes to separate MMR and varicella vaccines (MMRV) for children to cut febrile seizure rates.

Misleading promotions of GLP-1 and compounded semaglutide products are drawing renewed regulatory scrutiny over risk disclosure and safety messaging.

Optimizing FDA 483 responses with strategic CAPA creates resilient quality compliance in biopharma manufacturing.

The agency says it will now publish complete response letters promptly, offering developers early insight into regulatory deficiencies to improve development planning.

The nonprofit is calling upon its community of experts in both artificial intelligence and machine learning to continue building support for responsible adoption of AI in the bio/pharmaceutical industry.

FDA’s fast track designation may speed VMX-C001’s application review, addressing urgent surgery needs in patients on Factor Xa anticoagulants.

The authors examine common FDA 483 findings and outline strategies to address and prevent regulatory non-compliance.