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UniQure/CSL Set To Launch First Hemophilia B Gene Therapy Next Year At Record Price

With $3.5m Price Tag

Executive Summary

The firms’ gene therapy Hemgenix has won a US thumbs up for the treatment of hemophilia B and plans are underway for a US launch with a big price tag that experts agree should pay off in the long-term.

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Pfizer’s Gene Therapy Asset Chases UniQure/CSL’s Hemgenix After Pivotal Hemophilia B Success

Pfizer’s fidanacogene elaparvovec has hit the mark in a Phase III hemophilia B trial just a month after rival UniQure’s Hemgenix won US approval, with Pfizer’s plans for regulatory action raising the stakes for Hemgenix.

Hemophilia B Gene Therapy Among Five Drugs On Track For EU-Wide Approval

Alongside CSL/Behring/uniQure’s Hemgenix (etranacogene dezaparvovec), the European Medicines Agency has given the green light to new drugs for liver cancer, NSCLC, Pompe disease and multiple sclerosis. But it turned down omburtamab for treating the rare cancer, neuroblastoma.  

Value-Based Agreements On The Cards For UniQure/CSL’s $3.5m Hemophilia B Gene Therapy

While Hemgenix has courted criticism since its $3.5m price tag was unveiled, a drug pricing expert has told Scrip discounts and value-based agreements could make the therapy more palatable to payers.

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