1. Scrip
  2. >>New Products

UniQure/CSL Set To Launch First Hemophilia B Gene Therapy Next Year At Record Price

With $3.5m Price Tag

 
• By Ayisha Sharma

The firms’ gene therapy Hemgenix has won a US thumbs up for the treatment of hemophilia B and plans are underway for a US launch with a big price tag that experts agree should pay off in the long-term.  

Hands woman hold drop of blood on blue background.
Hemophilia B Is A Rare Clotting Disorder That Mostly Affects Males • Source: Shutterstock

uniQure N.V./CSL Limited’s Hemgenix (tranacogene dezaparvovec-drlb) has been approved for the treatment of adults with moderately severe to severe hemophilia B by the US Food and Drug Administration, making it the first gene therapy for the rare bleeding disorder and the most expensive drug to date.

The Australian firm revealed the treatment would cost $3.5m in the US, edging past the $3m price tag for

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from New Products

Insmed Targets $5bn-Plus Market With Brinsupri For Bronchiectasis

 
• By Mandy Jackson

The US FDA approved Insmed’s second product, the DPP1 inhibitor Brinsupri (brensocatib) for non-cystic fibrosis bronchiectasis. The company launched the drug immediately with a list price of $88,000 per year.

Boehringer Scores First Approval For Targeted Oral Drug In HER2-Mutant NSCLC

 
• By Alaric DeArment

The US FDA approved Hernexeos for previously treated HER2-mutant NSCLC, making it the first oral option in a setting where drugs like AstraZeneca/Daiichi Sankyo’s Enhertu dominate.

Pipeline Watch: Five Approvals And Two Phase III Readouts

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

PTC Projects Confidence In Sephience Launch With Royalty Buyback

 
• By Manas Mishra

The company will start shipping its newly launched phenylketonuria drug within two weeks and has bought back global net sales obligations for the treatment.

More from Scrip

Xenon Nears Phase III Readout For Potential Epilepsy And Depression Blockbuster

 
• By Andrew McConaghie

Leading a field of rival potassium channel modulators, azetukalner will have a pivotal epilepsy readout early next year, with Phase III trials in depression and biopolar disorder also underway.

Stock Watch: Sanofi And Merck Temper Big Pharma Q2 Earnings Cheer

 
• By Andy Smith

The investor enthusiasm that greeted initial second-quarter pharma earnings announcements was dampened by the results from Sanofi and Merck & Co. Sanofi was particularly punished by investors despite overall growth, while Merck’s revenue fall was probably baked into expectations.

Japan Results Roundup: Reduced Forex Impact, Uncertain US Tariff Outlook

 
• By Lisa Takagi

Most major Japanese firms report generally positive fiscal first quarter results, led by mainstay growth and despite lower currency effects. But the future impact of any US pharma tariffs remains an overhang.