The whole haemophilia gene therapy field will be hoping that a case of liver cancer in etranacogene dezaparvovec’s pivotal trial is not treatment related.
Early 2021 will see rare diseases dominate for biotech, with Sarepta revealing key data on its gene therapy play.
And bleeds seen with the “transformative” project were played down.
Waning factor VIII levels with giroctocogene fitelparvovec once again raise the spectre of Biomarin’s valrox.
Other groups with large disconnects between sellside and investor expectations include Beigene, Alnylam and Acceleron.
An analysis of FDA complete response letters does not reveal a big uptick – but the year is not over yet.
Could Pfizer/Sangamo’s project be facing the same durability issues as Biomarin’s Roctavian?
Valrox leaves a disproportionate hole in Biomarin’s valuation, but gene therapy competitors shouldn’t celebrate just yet.