$3 million per person price tag set on first gene therapy for hemophilia expected ready this fall

c d a e d eeb b c source
Credit: Stuart Ritchie/Getty Images

“It’s been absolutely brilliant and life-changing for me,” says [Jack] Grehan, 26, of Billinge in North West England. He received an experimental gene therapy in 2017 that, at least for now, has eliminated his need for regular [blood clotting] injections. “I can just go about my day and not have to worry.”

Based on experiences like Grehan’s, the company that developed the therapy is seeking approval in Europe and the United States to start selling the first gene therapy for hemophilia. That’s generating excitement among patients, patient advocates and doctors.

Others are more cautious.

“This is really exciting, but also raises a lot of questions,” says Meg Bradbury, director of research at the Hemophilia Federation of America, a patient advocacy group.

One of the biggest questions is the possible cost. BioMarin Pharmaceutical Inc. of San Rafael, Calif., the company that developed the gene therapy, says the treatment could cost as much as $3 million per patient, which would make it the most expensive drug ever approved.

Related article:  Podcast: Earth's largest mammals were 'downsized' by human hunting
Follow the latest news and policy debates on agricultural biotech and biomedicine? Subscribe to our newsletter.

“It’s just outrageous,” says Peter Bach, who studies drug prices at Memorial Sloan Kettering Cancer Center in New York.

Company officials defend the possible price, however. It currently costs hundreds of thousands of dollars a year to treat each hemophilia patient. The gene therapy would, researchers hope, be a one-time treatment that lasts a lifetime.

Read the original post

Outbreak
Outbreak Daily Digest
Biotech Facts & Fallacies
Talking Biotech
Genetics Unzipped
can you boost your immune system to prevent coronavirus spread x

Video: How to boost your immune system to guard against COVID and other illnesses

Scientists have recently developed ways to measure your immune age. Fortunately, it turns out your immune age can go down ...
mag insects image superjumbo v

Disaster interrupted: Which farming system better preserves insect populations: Organic or conventional?

A three-year run of fragmentary Armageddon-like studies had primed the journalism pumps and settled the media framing about the future ...
dead bee desolate city

Are we facing an ‘Insect Apocalypse’ caused by ‘intensive, industrial’ farming and agricultural chemicals? The media say yes; Science says ‘no’

The media call it the “Insect Apocalypse”. In the past three years, the phrase has become an accepted truth of ...
globalmethanebudget globalcarbonproject cropped x

Infographic: Cows cause climate change? Agriculture scientist says ‘belching bovines’ get too much blame

A recent interview by Caroline Stocks, a UK journalist who writes about food, agriculture and the environment, of air quality ...
organic hillside sweet corn x

Organic v conventional using GMOs: Which is the more sustainable farming?

Many consumers spend more for organic food to avoid genetically modified products in part because they believe that “industrial agriculture” ...
benjamin franklin x

Are most GMO safety studies funded by industry?

The assertion that biotech companies do the research and the government just signs off on it is false ...
gmo corn field x

Do GMO Bt (insect-resistant) crops pose a threat to human health or the environment?

Bt is a bacterium found organically in the soil. It is extremely effective in repelling or killing target insects but ...
favicon

Environmental Working Group: EWG challenges safety of GMOs, food pesticide residues

Known by some as the "Environmental Worrying Group," EWG lobbies for tighter GMO legislation and famously puts out annual "dirty dozen" list of fruits and ...
m hansen

Michael Hansen: Architect of Consumers Union ongoing anti-GMO campaign

Michael K. Hansen (born 1956) is thought by critics to be the prime mover behind the ongoing campaign against agricultural biotechnology at Consumer Reports. He is an ...
News on human & agricultural genetics and biotechnology delivered to your inbox.
Optional. Mail on special occasions.
Send this to a friend