It’s been 15 years since Genzyme began rationing the Fabry disease treatment Fabrazyme after a shortage caused by contamination at a manufacturing site, marking the start of many years of litigation.
Genzyme`s Biologic Fabrazyme (agalsidase beta) Receives Approval in the U.S.
BRISBANE, Calif.--(BUSINESS WIRE)--Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced updated preliminary results from the Phase 1/2 STAAR clinical study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for the treatment of Fabry disease. These latest data show that, as of the November 9, 2021 cutoff date, the investigational treatment continued to be well tolerated and that the four longest treated patients continued to exhibit elevated alpha-galactosidase A (?-Gal A) activity. These data are being presented at the 18th Annual WORLDSymposiumTM in a platform presentation on February 8, 2022 during the 1:30 p.m. Eastern Time session and in a poster presentation available on February 7, 2022 at 6:00 p.m. Eastern Time. These data are available on the Events & Presentations page of Sangamo’s website.
The FDA has started its review of Israeli biotech Protalix BioTherapeutics and partner Chiesi’s Fabry disease therapy pegunigalsidase alfa, setting up a possible approval by 27 January.
In 2010, people with a rare enzyme disorder called Fabry found supply of the only treatment rationed. Those taking Genzyme's drug Fabrazyme could only get a third of their normal dose, and new patients couldn't get any.
Genzyme`s Fabrazyme (Agalsidase Beta) Receives Supplemental Approval in US
When Protalix BioTherapeutics reported its 2018 results this March, it spent much of its time talking up the potential of nabbing an accelerated approval for its Fabry disease treatment pegunigalsidase alfa.