Biotech Values

[DewDiligence]

CSL.AX/QURE—FDA approves first gene-therapy for hemophilia B:

https://www.prnewswire.com/news-releases/us-food-and-drug-administration-approves-csls-hemgenix-etranacogene-dezaparvovec-drlb-the-first-gene-therapy-for-hemophilia-b-301685654.html

CSL today announced that the U.S. Food and Drug Administration (FDA) approved HEMGENIX (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy for appropriate adults with hemophilia B.

HEMGENIX is approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes. In the ongoing clinical trial, HEMGENIX reduced the rate of annual bleeds and 94 percent of patients discontinued factor IX prophylaxis and remained prophylaxis-free.

…The multi-year clinical development program for HEMGENIX was led by uniQure (Nasdaq: QURE), and sponsorship of the clinical trials transitioned to CSL after it acquired global rights to commercialize the treatment.

QURE will manufacture the product. The US list price is $3.5M for a single treatment, which the companies hope will provide a functional cure for a lifetime.

Hemophilia B, in which patients produce an insufficient amount of of Factor IX, comprises about 15% of all hemophilia cases.