#HEMGENIX
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jeffyguy.bsky.social
If you're headed to work, you must love your job now that drugs are discounted 1500% Instead of working, you can get prescriptions and let the government pay you 14 times the original cost. Hemgenix costs $3.5 million per dose. If you can fake hemophilia, you can pull down $49M for just one ailment.
August 4, 2025 at 11:58 AM
proflhunter.bsky.social
Totally correct.

And if it's a single dose that cures the disease and not a lifetime course? Not a problem for capitalism! www.fiercepharma.com/pharma/csl-a...
Sporting a $3.5M price tag, CSL and uniQure's hemophilia B gene therapy crosses FDA finish line
Move over, bluebird bio: There's a new most expensive drug in the world. | CSL Behring and uniQure's newly approved hemophilia B gene therapy, Hemgenix, comes with an eye-popping price tag of $3.5 mil...
www.fiercepharma.com
February 18, 2026 at 6:48 AM
samelsonjones.bsky.social
More good news about gene therapy for #hemophilia:

www.linkedin.com/posts/the-ch...
Children's Hospital of Philadelphia on LinkedIn: Meet Dominic, our first patient at CHOP outside a clinical trial to… | 12 comments
Meet Dominic, our first patient at CHOP outside a clinical trial to receive Hemgenix, an FDA approved one-time gene therapy for severe hemophilia B, an… | 12 comments on LinkedIn
www.linkedin.com
November 9, 2024 at 3:48 PM
samelsonjones.bsky.social
Rate of immunosuppression after #genetherapy for #hemophilia B with #Hemgenix in the real-world seems higher than the ~20% rate reported in clinical trial. Though small numbers here, also consistent with anecdotal results.

We will need more rigorous real-world data.

#ISTH2025 #hemesky
June 21, 2025 at 6:59 PM
denkstrom.bsky.social
Statt bis zu 150 Spritzen jährlich: Eine einzige Infusion schützt Hämophilie-B-Patienten dauerhaft. Kanada setzt erstmals die neue Gentherapie ein.
Hämophilie B: Eine Infusion statt 150 Spritzen pro Jahr
Am 20. Mai 2026 hat Kanada erstmals einen Patienten mit Hemgenix behandelt, der weltweit einzigen zugelassenen Gentherapie gegen Hämophilie B. Statt bis zu 156 Faktor-IX-Infusionen pro Jahr reicht eine einmalige Behandlung, die in 94 Prozent der Fäll
denkstrom.org
August 10, 2026 at 8:01 AM
drtimos.bsky.social
Thoughts on this? >> Hemgenix hemophilia B gene therapy reimbursable in Germany -
Hemophilia News Today >> Comment below! #strategy #competitiveintelligence #marketing #healthcare #pharma #pharmaceutical #competitivemarketing #biotech
Hemgenix hemophilia B gene therapy reimbursable in Germany - Hemophilia News Today
Hemgenix hemophilia B gene therapy reimbursable in Germany  Hemophilia News Today
dlvr.it
April 4, 2025 at 6:30 PM
drtimos.bsky.social
Thoughts on this? >> First hemophilia B patients in Denmark treated with Hemgenix
–... - Hemophilia News Today: First hemophilia B patients in Denmark treated with… >> Comment below! #strategy #competitiveintelligence #marketing #pharma #biotech #pharmaceutical #healthcare #competitivemarketing
First hemophilia B patients in Denmark treated with Hemgenix –... - Hemophilia News Today
First hemophilia B patients in Denmark treated with Hemgenix –...  Hemophilia News Today
dlvr.it
January 25, 2025 at 6:00 AM
profmakris.bsky.social
The German authorities have today concluded their negotiations with CSL Behring and agreed the price they will pay for the #hemophilia B gene therapy (Hemgenix). Does anybody know how much they will pay? tinyurl.com/mpnc9kk7
CSL Behring and GKV-Spitzenverband Agree on Reimbursement Price for HEMGENIX® – Europe's First Gene Therapy for Haemophilia B
Marburg, Germany, 2 April 2025 – CSL Behring and the GKV-Spitzenverband (National Association of Statutory Health Insurance Funds) have successfully concluded negotiations on the reimbursement p...
tinyurl.com
April 3, 2025 at 7:12 PM
drtimos.bsky.social
Thoughts on this? >> 2 new US patients given hemophilia B gene therapy Hemgenix:
Reports - Hemophilia News Today: 2 new US patients given hemophilia B gene therapy… >> Comment below! #strategy #competitiveintelligence #marketing #competitivemarketing #pharmaceutical #pharma #biotech #healthcare
2 new US patients given hemophilia B gene therapy Hemgenix: Reports - Hemophilia News Today
2 new US patients given hemophilia B gene therapy Hemgenix: Reports  Hemophilia News Today
dlvr.it
January 11, 2025 at 4:01 AM
mb81089900.bsky.social

Teuerstes #Medikament in der #Schweiz zugelassen – das wirft ethische Fragen auf
#Hemgenix

das «teuerste Medikament der Welt», die Zahl 3.5 Millionen Dollar pro Infusion
Jetzt wurde das Mittel auch von @Swissmedic_ zugelassen.

www.watson.ch/wissen/leben...
Teuerstes Medikament in der Schweiz zugelassen – das wirft ethische Fragen auf
Hemgenix gilt als das teuerste Medikament der Welt, nun ist es auch in der Schweiz zugelassen. Solche Arzneien stellen die solidarische Gesundheitsversorgung vor eine heikle Frage: Wie viel ist ein kr...
www.watson.ch
January 24, 2024 at 10:39 AM
philippbeck.bsky.social
One might see a parallel in Beam/WVE vs Hemgenix;Roctavian;Beqvez/Hympavzi I.e. non-permanent approach was preferred by the market.
March 10, 2025 at 12:57 PM
pharmaphorum.bsky.social
NEWS: Germany has okayed the use of a pricey haemophilia B gene therapy with a 'unique' outcomes-based payment model.

pharmaphorum.com/news/germany...
Germany will reimburse haemophilia gene therapy Hemgenix
Germany has agreed to reimburse CSL Behring's haemophilia B gene therapy Hemgenix with a unique outcomes-based payment model
pharmaphorum.com
April 3, 2025 at 12:57 PM
stanforddeptmed.bsky.social
Katherine High delivered this week's Stanley Schrier Lecture at Medicine Grand Rounds. The Spark Therapeutics co-founder & 2026 Breakthrough Prize in Life Sciences winner led the team behind Luxturna, the first FDA-approved gene therapy for an inherited disease.
Katherine High is presenting to an audience in a conference room, with a screen displaying slides featuring various pharmaceutical logos and drug names, including Pfizer, Wyeth, and Hemgenix. The room is well-attended, and the audience is focused on the presentation. Katherine High on stage in front of a large screen, gesturing and wearing a brown jacket. A group of diverse people standing on a stage, posing for a group photo. Some individuals are holding an award. The background features a plain wall and overhead lighting.
September 25, 2026 at 4:32 PM
christianbisti.bsky.social
Ordino subito un paio di confezioni di Hemgenix.
A 3,5 milioni di $ a dose, dovrei sistemarmi per un po'.
Mi accontento anche di uno sconto del "solo" 200%
July 23, 2025 at 6:41 PM
endpts.com
Multimillion-dollar gene therapies weren’t supposed to face real price competition. China’s pipeline suggests otherwise. @jaredwhitlock.bsky.social explores the shift in the latest Endpoints In Focus.
Multimillion-dollar gene therapies weren’t supposed to face competition. China has other plans
Chinese firm Belief BioMed launches $350K hemophilia B gene therapy, challenging $3.5M Hemgenix by CSL Behring/uniQure, as China leads global race for cheaper gene therapies.
endpoints.news
March 5, 2026 at 5:06 PM
denkstrom.bsky.social
Statt bis zu 150 Spritzen jährlich: Eine einzige Infusion schützt Hämophilie-B-Patienten dauerhaft. Kanada setzt erstmals die neue Gentherapie ein.
Hämophilie B: Eine Infusion statt 150 Spritzen pro Jahr
Am 20. Mai 2026 hat Kanada erstmals einen Patienten mit Hemgenix behandelt, der weltweit einzigen zugelassenen Gentherapie gegen Hämophilie B. Statt bis zu 156 Faktor-IX-Infusionen pro Jahr reicht eine einmalige Behandlung, die in 94 Prozent der Fäll
denkstrom.org
May 29, 2026 at 4:16 PM
endpts.com
Pfizer is discontinuing Beqvez, its $3.5M hemophilia B gene therapy, due to limited patient demand. The move marks another challenge for hemophilia gene therapies amid slow adoption.
Pfizer ends rollout of Beqvez, putting another dent in hemophilia gene therapy field
Pfizer ends commercialization of Beqvez hemophilia B gene therapy. CEO Kim Phelan of Coalition for Hemophilia B expresses disappointment, notes CSL Behring's Hemgenix remains available.
endpts.com
February 21, 2025 at 2:59 PM
checkrare.bsky.social
Steven Pipe, MD, Professor of Pediatrics and Pathology at the University of Michigan, discusses positive follow-up results in patients with hemophilia B treated with Hemgenix (etranacogene dezaparvovec-drlb).
checkrare.com/positive-fol...

#CheckRare #HemophiliaB #RareHematology #RareDisease
March 19, 2025 at 4:46 PM
futharkvillage.bsky.social
Hospitals with machines similar to the ones used to make synthetic diamonds, programmed to print Hemgenix or Skysona, would not be saving money over just compounding the drugs in the usual way... Even before the lawsuits drive those hospitals into bankruptcy.
May 25, 2025 at 7:18 PM
toughsf.bsky.social
The most expensive medicine in the US is $4.25m for Lenmeldy, to treat children with metachromatic leukodystrophy.
Then come $3.5m Hemgenix for hemophilia B, $3.2m Elevidys for DMD and $3m Skysona for Cerebral Adrenoleukodystrophy.
All are one-time gene therapy doses.
June 17, 2024 at 2:38 PM
jdevos.bsky.social
China approved a gene therapy at 1/10 the price of its U.S. rival. China is poised to rapidly disrupt the gene and cell therapy field.

buff.ly/CfZDNgS
Multimillion-dollar gene therapies weren’t supposed to face competition. China has other plans
Chinese firm Belief BioMed launches $350K hemophilia B gene therapy, challenging $3.5M Hemgenix by CSL Behring/uniQure, as China leads global race for cheaper gene therapies.
endpoints.news
March 5, 2026 at 6:52 PM
jaredwhitlock.bsky.social
Multimillion-dollar gene therapies weren’t supposed to face competition. China has other plans

Latest feature + data analysis

endpoints.news/china-is-tak...
Multimillion-dollar gene therapies weren’t supposed to face competition. China has other plans
Chinese firm Belief BioMed launches $350K hemophilia B gene therapy, challenging $3.5M Hemgenix by CSL Behring/uniQure, as China leads global race for cheaper gene therapies.
endpoints.news
March 5, 2026 at 10:10 PM
nordiclifescience.org
“This is what scientific progress in medicine is intended to achieve: improving the life of patients, in particular those with rare diseases, by providing innovative therapeutic options.” – Fredrik Sjöö Nordic Region CSL Behring #GeneTherapy
First Danish patients treated with gene therapy for haemophilia B - NLS
CSL Behring Denmark has announced that the first Danish patients with haemophilia B were treated with the gene therapy HEMGENIX.
nordiclifescience.org
January 21, 2025 at 8:41 AM
ruthcoxeter.bsky.social
“An Endpoints News analysis of clinical trial databases shows that China accounts for 48 of the 77 programs targeting this group of ultra-expensive gene therapies. China’s total is more than double the number in the US and seven times as many as Europe.”
Multimillion-dollar gene therapies weren’t supposed to face competition. China has other plans
Chinese firm Belief BioMed launches $350K hemophilia B gene therapy, challenging $3.5M Hemgenix by CSL Behring/uniQure, as China leads global race for cheaper gene therapies.
endpoints.news
March 5, 2026 at 5:39 PM
619sdld.bsky.social
UC San Diego Health First to Offer Novel Gene Therapy for Hemophilia B

UC San Diego Health is the first in San Diego to offer Hemgenix, an FDA-approved gene therapy for hemophilia B. This one-time infusion enhances clotting factor IX production, reducin…
UC San Diego Health First to Offer Novel Gene Therapy for Hemophilia B
University of California San Diego Health now offers a new gene therapy for hemophilia B, a bleeding disorder that prevents blood from clotting properly.
buff.ly
January 9, 2025 at 10:05 PM