Bio­Marin spells out a trou­bling slide in Fac­tor VI­II lev­els for its big block­buster con­tender val­rox

We al­ready knew from Bio­Marin’s state­ment a few days ago that their gene ther­a­py for he­mo­phil­ia A con­tin­ued to pro­tect pa­tients against the bleed­ing episodes that can threat­en their lives. Now, 4 years in, we’re get­ting the hard da­ta on dura­bil­i­ty for val­rox — and here the biotech faces con­sid­er­able po­ten­tial trou­ble.

As Stifel’s Paul Mat­teis not­ed re­cent­ly, at the end of the first, sec­ond and third years, post-in­fu­sion, mean Fac­tor VI­II ac­tiv­i­ty lev­el of the high dose 6e13 vg/kg co­hort was 64.3, 36.4 and 32.7 IU/dL re­spec­tive­ly as mea­sured by the CS as­say.

Endpoints News

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.