News

Hemophilia patients who develop inhibitors against treatment face markedly higher healthcare costs — about three times those of other patients — and experience poorer quality of life, according to a team of scientists in Italy. Alternatives are available and should be considered for these patients, they write. The study, titled…

Express Scripts has teamed up with PinnacleCare to include in its new Rare Conditions Care Value (RCCV) program a service that provides patients with rare diseases like hemophilia a second opinion from a specialist care provider. The initiative, simply called Second Opinion, is designed to give patients an additional…

Since every hemophilia A patient responds differently to therapies, researchers are working to develop personalized preventive (prophylaxis) strategies with Nuwiq (antihemophilic factor [recombinant].) Octapharma, the treatment’s manufacturer, presented data showing that an individual’s pharmacokinetic (PK) profile (how the therapy works once inside the body) can be used to possibly…

Preliminary results from the U.S. part of Bayer’s global HemACTIVE patient survey show that a majority of hemophilia patients want to be more active but are limited in their capacity to perform outdoor activities. The data was shared by Bayer at the World Federation of Hemophilia 2018…

US Bioservices, a specialty pharmacy, announced that it is working with the pharmacy benefit management company MedImpact to dispense prescriptions and select services through the MedImpact Direct Specialty Program for the hemophilia community. Under the collaboration, US Bioservices will offer hemophilia patients who are MedImpact members the opportunity to…

A gene therapy approach acquired by uniQure was recently covered in two new patents, one issued in the United States and one in Canada, broadly covering ways of treating bleeding disorders that include hemophilia B, the company announced in a press release. The therapy has a nucleic acid encoding the…

Hemlibra (emicizumab-kxwh) was recently granted priority review status by the U.S. Food and Drug Administration following the agency’s acceptance of Genentech’s supplemental biologics license application (sBLA) for the treatment for people with hemophilia A without factor VIII inhibitors. Priority review status is granted to therapies that could…

As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third time, 9-year-old Jordan finally got the hug he wanted — as well as a kiss on the forehead. The video of…

The U.S. Food and Drug Administration has approved a larger vial size — 3,500 IU — of CSL Behring’s Idelvion (albutrepenonacog alfa) for the treatment of hemophilia B patients. Idelvion is supplied as a powder for intravenous use and must be reconstituted, or mixed with a liquid (called…

SPK-9001, an investigational gene therapy being developed by Spark Therapeutics and Pfizer, continues to demonstrate great potential to treat hemophilia B patients in an ongoing Phase 1/2 clinical trial. The latest results showed that the annualized bleeding rates of all treated patients were reduced by 98%. In…