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Luxturna's EU Approval Highlights Gene Therapy Pricing Challenges

 
• By Francesca Bruce

Novartis/Spark Therapeutics’ transformative gene therapy Luxturna has just been approved in the EU. Like other gene therapies, it comes with a heavy price tag. So how will Novartis convince payers to pay, and how will payers cope with expensive, game changing therapies in the future? 

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Novartis's gene therapy Luxturna has been approved in the EU • Source: Shutterstock

Novartis AG/Spark Therapeutics Inc.'s Luxturna (voretigene neparvovec) has been granted marketing authorization in the EU, the first gene therapy to be approved for the treatment of retinal disease. The Pink Sheet takes a look at how this innovative one-off treatment might now get to market and how Europe’s payers might cope with the growing numbers of high-cost, game-changing therapies that are set to hit the market.

Luxturna has been approved for children and adults with inherited retinal dystrophy caused by RPE65 gene mutations. This rare genetic disorder causes vision loss and usually leads to blindness, and...

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New EU Approvals

 
• By Neena Brizmohun

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include two new products, one of which is Vyjuvek, Krystal Biotech's advanced therapy for treating dystrophic epidermolysis bullosa.

Autolus’s Aucatzyl And GSK’s Blenrep Among 10 New Drugs To Get EMA Nod

 
• By Vibha Sharma

The European Medicines Agency has OKd 10 new medicines for EU-wide approval, including a CAR-T therapy for treating acute lymphoblastic leukemia. Two drugs were, however, rejected.

EU Authorization Before US? Biologics Manufacturing Is Likely To Blame

 
• By Bridget Silverman

In the infrequent cases when EU authorization precedes US FDA approvals of new products, biologics manufacturing issues are usually to blame, a Pink Sheet analysis found.

Novavax’s COVID-19 Vaccine Label Narrowed After Political Officials Intervened

 
• By Sarah Karlin-Smith

The label that Novavax's COVID-19 vaccine was slated to receive before political officials held up the approval was broader than was granted 16 May.

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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.

HHS Rescinds COVID-19 Vaccine Advice, Usurping US CDC Role

 
• By Sue Sutter

By stepping into the role of the Centers for Disease Control and Advisory Committee on Immunization Practices, HHS Secretary Robert F. Kennedy Jr. is causing further confusion and uncertainty about vaccine policy, experts say.

EMA: ‘Sad To See’ Member States Against Patient Engagement Under EU Pharma Reform

 
• By Eliza Slawther

The European Medicines Agency’s chief medical officer Steffen Thistrup argued that patients are able to understand the “complex regulatory and scientific issues” that the EMA committees discuss when they assess drugs.