The FDA has approved Beqvez, a Pfizer gene therapy developed for moderate-to-severe hemophilia B. The one-time treatment carries a $3.5 million price tag, the same as a CSL Behring gene therapy already available for treating the inherited bleeding disorder.
Pfizer already reported its experimental drug for hemophilia A and B met a pivotal study’s goals of reducing bleeding episodes. During the annual meeting of the American Society of Hematology, the company presented a full look at the results, which are expected to support regulatory submissions soon.
Munck Wilson Mandala Partner Greg Howison shared his perspective on some of the legal ramifications around AI, IP, connected devices and the data they generate, in response to emailed questions.
By selling partial royalties to approved hemophilia gene therapy Hemgenix, uniQure gains immediate cash to support its pipeline, including a Huntington’s disease gene therapy already in the clinic . The royalty deal extends uniQure’s cash runway into 2026.
CSL Behring's hemophilia B gene therapy, Hemgenix, carries a $3.5 million list price. But the company contends that by reducing economic burdens associated with bleeding and prophylactic infusions, the one-time treatment will save money over time.
A uniQure gene therapy for hemophilia B in on track for an FDA submission in the first quarter of 2022. Meanwhile, uniQure added to its pipeline with the acquisition of preclinical epilepsy gene therapy biotech Corlieve Therapeutics for €46.3 million upfront.
A uniQure gene therapy for hemophilia B that has been under a clinical hold for four months can now resume testing. The hold was lifted after uniQure answered the FDA's questions about whether the gene therapy caused the liver cancer diagnosed in one patient who received the therapy.
At the Abarca Forward conference earlier this year, George Van Antwerp, managing director at Deloitte, discussed how social determinants of health and a personalized member experience can improve medication adherence and health outcomes.
UniQure is making a case for lifting a clinical hold on its hemophilia B gene therapy, reporting Monday results of an independent investigation that appear to clear the experimental treatment as the cause of a patient’s cancer.
The therapy, etranacogene dezaparvovec, is currently in Phase III clinical development for hemophilia B. Shares of uniQure were down nearly 20%, but an investment bank analyst wrote that the financial terms were favorable for the company.
After 22 years, gene editing pioneer Sangamo Therapeutics is taking the plunge with three new in vivo gene editing clinical trials. If successful, the studies could serve as a template for myriad other genetic disorders.