FDA’s Center for Biologics Evaluation and Research approved six original BLAs in 2012, a solid if unspectacular showing. But some of the center’s most interesting approvals are products that do not appear on the original BLA count: quadrivalent flu vaccines (supplements to predecessor trivalent products) and hemapoietic cord blood cells (not included under the PDUFA umbrella).
As FDA’s biologics center closes in on a decade since responsibility for therapeutic biologics was transferred to CDER, CBER posted a solid, if undramatic, year for approvals in 2012, with six original BLA approvals under the PDUFA framework.
Six approvals puts the 2012 CBER novel biologic class at the mid-point of CBER BLA activity in recent years, which has ranged
Plozasiran, Arrowhead Pharmaceuticals' treatment for familial chylomicronemia syndrome, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.
The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.
Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.
The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.
The Oncologic Drugs Advisory Committee will meet for two days in mid-May, followed by a Vaccines and Related Biological Products Advisory Committee’s review of the 2025-2026 COVID-19 vaccine formulation.
The European Medicines Agency, like its counterpart in the US, is increasingly focusing on the use of alternatives to animal testing.
Some advisory committee members said adding quantitative data on misuse, abuse, opioid use disorder and overdose to labeling would be helpful, but others worried the two epidemiological studies were not sufficiently generalizable to a broader population.
