Quality/GMPs

The final guidance provides specific recommendations for CMC, pharmacology, toxicology, and clinical study design for CAR-T cell products.

Validating computerized systems is required to demonstrate adherence to data integrity, says Susan J. Schniepp, distinguished fellow at Nelson Laboratories, LLC.

The immune-resetting peptide, developed by Revolo Biotherapeutics, received positive results from its Phase IIb study.

The final guidance provides recommendations for developing gene therapy products incorporating genome editing of human somatic cells.

Takeda has received FDA approval for HYQVIA, a subcutaneous immunoglobulin for the maintenance treatment of chronic inflammatory demyelinating polyneuropathy.

The agency recommended 77 drugs for marketing authorization in 2023, including 39 new APIs.

FDA’s Center for Drug Evaluation and Research released its report on new drugs approved in 2023.

The agency is reviewing data on secondary malignancies related to T-cells for approved CAR T-cell medicines.

The European Medicines Agency safety committee says there is a potential risk of neurodevelopment disorders in children born to men treated with valproate.

Revolutionary therapies restructure pharmaceutical manufacturing.

Reviewing regulatory documents, such as 483 forms and Warning Letters, can aid in determining a facility’s inspection readiness, says Siegfried Schmitt, vice president, Technical at Parexel.

Clinician-Scientists are vital innovation spark plugs, accounting for 40% of Nobel awards in Physiology or Medicine, and two thirds of Big Pharma CSOs.

The midterm report of the European Medicines Agencies Network Strategy finds that the network has strengthened and is on track to achieve its objectives.

WHO provides a look at the world’s health as the agency turns 75.

The final guidance document provides recommendations for designing a new registry or using an existing registry to support decisions regarding safety and effectiveness of a drug.

The agencies published a joint guidance document regarding quality development for breakthrough therapies and PRIME programs.

The Multi-annual AI Workplan creates a strategy to maximize the benefits and manage the risks of artificial intelligence.

The guidance document discusses the impact of clinical pharmacology considerations on peptide drug development.

The US District Court for the District of Massachusetts entered a consent decree of permanent injunction against Pharmasol Corporation for distributing adulterated drugs.

RoslinCT will manufacture Vertex Pharmaceuticals’ CRISPR-based gene therapy, Casgevy, recently approved for treating sickle cell disease and β thalassemia.

The new advisory committee will provide the agency with technical and scientific advice and recommendations on potential treatments for genetic metabolic diseases.

Sanofi has issued a statement expressing its disappointment with FTC’s decision to block an exclusive licensing agreement with Maze Therapeutics for a rare disease drug candidate.

Regulators in the European Union have published the first version a list of critical medicines in the EU to avoid potential shortages.

Biologics have specific quality considerations that make fast tracking these drugs more difficult.

Pharmaceuticals are entering a transition into a far more complex era of patient therapies, and patient segmentation.

Final product inspection results should be included as part of the batch release documentation, says Susan J. Schniepp, distinguished fellow at Nelson Laboratories, LLC.

Exagamglogene autotemcel seen as synechdoche.

Adzynma provides a replacement for low levels of the ADAMTS13 enzyme in patients with congenital thrombotic thrombocytopenic purpura.

Recommended medicines include treatments for generalized myasthenia gravis, glioma, neovascular age-related macular degeneration, and more.

Electronic product information for selected human medicines that have been harmonized across the European Union have been published for the first time.