FDA blocks much-anticipated BioMarin hemophilia gene therapy
Companies, agencies, institutions, etc
AP
BioMarin
The U.S. Food and Drug Administration’s
FDA
Biomarin
Novartis AG’s
Zolgensma
Pfizer Inc.
Sangamo Therapeutics
Biomarin Pharmaceutical Inc.
People
Roctavian
Generation Bio
SVB Leerink
Joseph Schwartz
Linda A. Johnson
Groups
Swiss
Physical locations
No matching tags
Places
No matching tags
Locations
FAIRLESS HILLS
PA
U.S.
San Rafael
California
Events
No matching tags
Summary
The company added that FDA concluded differences between the results of a small, early-stage study and interim data from the late-stage study left unclear how long the therapy’s effect would last.Roctavian was meant to free patients with severe hemophilia A from 100 to 150 IV infusions of Factor VIII per year to prevent or at least reduce painful, spontaneous bleeding into joints and muscles, which can cause permanent damage to them.Also known as valoctocogene roxaparvovec or valrox for short, it would have been the first gene therapy approved in the U.S. for any type of hemophilia.
As said here by LINDA A. JOHNSON