1. Scrip
  3. >>Gene Therapies

Bluebird Could Find US Tailwind For Zynteglo After ICER Report

 
• By Alaric DeArment

The cost-effectiveness watchdog now assumes an upfront $2.1m payment instead of five annual payments. The company switched gears to the US after the TDT gene therapy stumbled in Europe.

ICER released a revised version of its report on bluebird bio's Zynteglo • Source: Shutterstock

The Institute for Clinical and Economic Review (ICER) is pulling back from a recommendation for using five annual payments to cover bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) in transfusion-dependent beta-thalassemia (TDT) and assuming a single upfront payments of $2.1m, but is still linking that to an outcomes-based agreement.

The cost-effectiveness watchdog group published a revised evidence report on 2 June assessing the comparative clinical effectiveness and value of...

More from Gene Therapies

More from Advanced Therapies

Removal Of REMS For CAR-Ts Could Greatly Expand Their Reach

 
• By Alaric DeArment

The FDA eliminated REMS requirements for approved CAR-T cell therapies that have limited their availability, a move that could enable increased use in the community setting.

Can Japan’s Richest Person Save Cell Therapy’s Future?

 
• By Lisa Takagi

With financial help from the founder of Uniqlo, Japan’s leading facility for induced pluripotent stem cells is aiming to automate production to slash costs and kick-start R&D in the field.

Positive Fabry Data Should Smooth Way For Sangamo’s Partnering Efforts

 
• By Alaric DeArment

The biotech announced results from the pivotal trial of isaralgagene civoparvovec and plans to file for approval in 2026 as it looks for a commercialization partner.