FDA blocks much-anticipated BioMarin hemophilia gene therapy

  • 📰 CTVNews
  • ⏱ Reading Time:
  • 21 sec. here
  • 2 min. at publisher
  • 📊 Quality Score:
  • News: 12%
  • Publisher: 99%

Canada Headlines News

Canada Latest News,Canada Headlines

FDA rejects potentially game-changing hemophilia A gene therapy BioMarin over concerns it might not really be a one-and-done lifetime treatment

The U.S. Food and Drug Administration's rejection late Tuesday means the San Rafael, California-based company will have to complete an ongoing late-stage patient study, likely delaying possible approval till late in 2022.

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. The gene therapy works by using an inactivated virus, created in a lab, to deliver to liver cells a working gene via a one-time IV infusion meant to enable the body to produce FVIII on its own.

 

Thank you for your comment. Your comment will be published after being reviewed.
Please try again later.

'...Over Financial Concerns Regarding This One-and-Done Treatment.'

This is why we must pass the Promising Pathway Act

Its time to stop playing god. We dont know the effects of this stuff. How about starting at curing the common cold first...

Gene therapy are we better than nature ?

We have summarized this news so that you can read it quickly. If you are interested in the news, you can read the full text here. Read more:

 /  🏆 1. in CA

Canada Latest News, Canada Headlines

Similar News:You can also read news stories similar to this one that we have collected from other news sources.

Toronto protesters block sheriffs from conducting evictions during COVID-19Dozens of demonstrators occupy two ramps leading to the parking garage at the Superior Court of Ontario Good The two groups telling people not to pay their rent you mean. Just another bunch of low life's exploiting people for their own agendas Pathetic
Source: globeandmail - 🏆 5. / 92 Read more »

Tiger Woods' 11-year-old son wins junior golf tournamentTiger Woods' 11-year-old son Charlie is a chip off the old block when it comes to golf. Congratulations Kid.. Apple doesn’t fall too far...... WTG, Mr. Junior Woods!!! 👊💕
Source: CTVNews - 🏆 1. / 99 Read more »

2021 NBA All-Star weekend in Indy unlikely on current dates - TSN.caThe NBA’s 2021 All-Star weekend in Indianapolis is unlikely to take place as currently scheduled, with the league revealing Monday that large blocks of hotel rooms that had been reserved for the event have been released. LMAO F-O-R-E-V-E-R 14DAYSTOSLOWSPREAD CHINA WHILE WUHAN IS OPEN
Source: TSN_Sports - 🏆 80. / 51 Read more »