US FDA advisory committee members questioned how potential off-target effects with Vertex’s gene therapy for sickle cell disease should be conveyed to patients and families, and whether labeling should say anything about these theoretical risks.
The first US Food and Drug Administration advisory committee called upon to review a therapeutic incorporating genome editing found itself navigating new terrain befitting this cutting-edge technology, including practical implications of theoretical risks.
Specifically, panelists questioned how potential off-target effects with Vertex Pharmaceuticals Incorporated’s exa-cel should be conveyed to patients and their families, and what
Advisory committee for Capricor’s deriamocel planned, regenerative medicine advanced therapy designations proliferate for osteoarthritis, and the ranks of dual RMAT/breakthrough therapy designation holders grow.
The 31 May goal date for Moderna’s next-generation COVID-19 vaccine looms over the month, but the US FDA's decision lineup includes three more novel agents, including AbbVie's Teliso-V antibody-drug conjugate, as well as supplemental indications like GSK's bid for Nucala in COPD.
Antabio’s next-generation, broad-spectrum antibacterial, MEM-ANT3310, has made it onto the European Medicines Agency’s priority medicines scheme. The company says its product is designed to make a significant impact on the growing problem of antimicrobial resistance.
Organizational turmoil has not yet affected reviews of novel agent applications, with Q1 2025 approvals coming in low, but the first quarter share of the entire year is variable.
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Organizational turmoil has not yet affected reviews of novel agent applications, with Q1 2025 approvals coming in low, but the first quarter share of the entire year is variable.
This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.
