On Tuesday, the U.S. Food and Drug Administration (FDA) approved uniQure (NASDAQ:QURE) and Australia-based CSL’s unit CSL Behring’s Hemgenix for adults with hemophilia B who are currently undergoing Factor IX prophylaxis therapy or who have experienced recurrent bleeding episodes or fatal hemorrhages.
However, Cowen analyst noted that that the medicine was priced higher than the firm’s estimated $1.9M but its consultants were optimistic about the treatment’s uptake, the report added.
In May 2021, uniQure and CSL completed their licensing deal for the gene therapy. Under the agreement, uniQure has received payments from CSL totaling ~$500M and is eligible to receive up to an additional $1.5B in commercial milestone payments, plus royalties.
A few months ago, the FDA already approved two other expensive gene therapies, Zynteglo and Skysona, both from bluebird bio (BLUE).
Skysona, which was approved in September to treat a rare neurodegenerative disease called cerebral adrenoleukodystrophy, was priced at $3M. Whereas, Zynteglo to treat patients with beta-thalassemia is priced at $2.8M.