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First Gene Editing Therapy Among Seven Drugs On Track For EU-Wide Approval

CHMP Confirms Non-Renewal Of Conditional Authorization For GSK’s Multiple Myeloma Drug

 
• By Vibha Sharma

The European Medicines Agency has recommended seven new drugs for EU-wide approval, and OKd two others under its EU-M4all procedure that is meant for high-priority human medicines intended for markets outside the EU.

Casgevy Is A Personalized One-Off Treatment • Source: Shutterstock

The first CRISPR/Cas9 gene-editing therapy, Casgevy (exagamglogene autotemcel), is on track for pan-EU marketing approval as a treatment for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TBT) after receiving a thumbs up at the December meeting of the European Medicines Agency’s human medicines committee, the CHMP.

Casgevy is Vertex Pharmaceuticals/CRISPR Therapeutics’ cell-based gene therapy medicinal product that uses the novel CRISPR/Cas9 technology to edit...

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More from Approvals

EU Biosimilar Filings, Opinions And Approvals

 
• By Neena Brizmohun

A list of EU biosimilar filings, CHMP opinions and EU marketing authorizations, including details of the biosimilar company, the brand name/INN, indication(s), the reference product/company, and the date and type of event.

Japan Gives Global-First Recommendations To Taisho’s Vornorexant, Santen’s Sepetaprost

 
• By Lisa Takagi

Japan gives world-first approval recommendations to two domestically originated drugs, for insomnia and glaucoma/ocular hypertension.

New EU Filings

 
• By Neena Brizmohun

Arimoclomol, Zevra Therapeutics's treatment for Niemann-Pick disease Type C, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

Sanofi Secures EU Fast-Track Status For ‘Game-Changing’ Sleeping Sickness Drug

 
• By Neena Brizmohun

If approved, Sanofi’s acoziborole could become the first single-dose oral treatment for sleeping sickness.

More from Product Reviews

EU CHMP Opinions And MAA Updates

 
• By Neena Brizmohun

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.

Japan Gives Global-First Recommendations To Taisho’s Vornorexant, Santen’s Sepetaprost

 
• By Lisa Takagi

Japan gives world-first approval recommendations to two domestically originated drugs, for insomnia and glaucoma/ocular hypertension.

August Brings Fresh Faces And Tough Calls To US FDA User Fee Calendar

 
• By Bridget Silverman

The FDA could act on 17 applications, including 10 novel agents, during August. Only one would be a second-cycle review.