Decades ago most hemophilia patients died young. Advances in treatment have led to them living about as long as the general population, which means that for the first time, doctors are facing the challenge of caring for aging hemophiliacs. A key problem is that in the days before treatment breakthroughs,…
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Novo Nordisk’s recombinant coagulation factor IX nonacog beta pegol (N9-GP), under the U.S. trade name Rebinyn, has been approved by the U.S. Food and Drug Administration (FDA) to treat adults and children with hemophilia B. The company expects to launch this new treatment in the United States early in 2018. The…
Cellphones are a good way for hemophiliacs receiving care at home to relay information that doctors need to stay on top of their treatment. But a lot of patients have yet to embrace the service — and a recent online survey of 181 hemophiliacs shed some light on why. Spanish…
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Denver’s Hemophilia Hero Chris Bombardier Becomes First with the Disease to Climb Mount Everest
Chris Bombardier, the author of the “Adventures of a Hemophiliac” blog, has become the first person with hemophilia to climb Mount Everest, the world’s highest peak. That achievement on May 22 means the 31-year-old Denver native has only one mountain to go toward his goal of scaling the tallest peaks…
The Phase 1/2 trial looking at the Factor VIII protein SHP656 as a long-acting therapeutic to treat hemophilia A did not meet a pre-defined once-weekly dosing criterion, according to Xenetic Biosciences and Shire, which are jointly developing the drug. The study demonstrated SHP656’s efficacy and pharmacokinetic data (the drug’s behavior once…
The National Organization for Rare Disorders (NORD) has recognized pharmaceutical company CSL Behring for pioneering Idelvion (rIX-FP), its therapeutic medicine against hemophilia B. “We are proud to present the team at CSL Behring with a 2017 Industry Innovation Award for the important work they are doing to address the needs of patients…
The U.S. Food and Drug Administration (FDA) has granted fast-track status to SB-525, a clinical gene therapy for hemophilia A developed by Sangamo Therapeutics in partnership with Pfizer. SB-525 delivers a human factor VIII cDNA construct and synthetic liver-specific promoter to the cell’s nucleus by using recombinant adeno-associated virus (rAAV). The…
Dimension Therapeutics will discontinue clinical development of DTX101, a gene therapy intended to treat moderate/severe-to-severe hemophilia B. The decision comes after a review of data from a Phase 1/2 clinical trial (NCT02618915), which assessed and the safety and dosing of DTX101. The trial was a…
Sangamo Therapeutics and Pfizer have entered a global collaboration and license agreement to develop and market gene therapy programs for hemophilia A, including SB-525. SB-525 was cleared by the U.S. Food and Drug Administration (FDA) in January as an investigational new drug (IND) for the…
UniQure has taken major steps this year toward U.S. and European regulatory approval of AMT-060, its gene-therapy treatment for hemophilia B. In January, the U.S. Food and Drug Administration (FDA) gave AMT-060 breakthrough therapy designation for patients with severe hemophilia B. In April the European Medicines Agency (EMA) gave it PRIME designation. The designations…
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