FDA Approves Adynovate to Treat Children and for Surgeries with Hemophilia A
The FDA has approved Adynovate for children under 12 with Hemophilia A. The Antihemophilic Factor (Recombinant) has also been approved for use in surgical settings for both adult and pediatric patients. Adynovate was previously approved for prophylactic and on-demand treatment of adults. See full article at raredr.com.
Roche's ACE-910 Succeeds in First of Three Final Stages of Clinical Trial
Roche's experimental hemophilia treatment succeeded in the first of three final-stage clinical trials. Patients receiving the drug, known as ACE-910 or emicizumab, showed a statistically significant reduction in bleeds over time. Previous adverse events in earlier trials initially concerned researchers. See full article at bloomberg.com.
Biogen Approves Spin-off Hemophilia Business Bioverativ
Biogen announced that its board had finalized approval of a spin-off of its hemophilia business, Bioverative Inc., into a publicly traded company. The company initially announced plans to spin of its hemophilia business in May of 2016. See full article at thestreet.com.
NovoSeven Resolved 96.5% of Bleeds in Hemophilia A and B Patients
Novo Nordisk announced that their NovoSeven, a portable, room temperature stable recombinant factor VIIa, resolved 96.5% of bleeds when initiated within one hour after onset of bleeding. This demonstrates efficacy of early treatment in people with hemophilia A or B with inhibitors. See full article at europeanpharmaceuticalreview.com.
Vonvendi, Treatment for von Willebrand Disease, Meets Endpoints in Study
Shire's von Willebrand factor treatment Vonvendi has met the primary endpoint in a Phase 3 study controlling bleeding and blood loss during major, minor, and oral elective surgeries. The results were shared at the annual American Society of Hematology meeting held in San Diego. See full article at biopharmadive.com.
CRISPR Used for the First Time to Correct Hemophilia B in Mice
CRISPR/Cas9, a powerful genome editing tool, is showing promise for efficient correction of disease-causing mutations. The approach was used for the first time to target mice with hemophilia B. The gene therapy method is being tested at the University of Pennsylvania's Perelman School of Medicine. See full article at pennmedicine.org.
Biogen Presents Updated Data for Eloctate and Alprolix at ASH
Data that reinforced the long-term safety and efficacy of extended half-life hemophilia therapies Eloctate and Alprolix were shared at the 58th Annual meeting of the American Society of Hematology. The new data showed low target joint annual bleeding rates and effective target joint resolution in patients of all ages. See full article at businesswire.com.
uniQure Announces Clinical Data from AMT-060 Phase I/II Trial
New results from uniQure's AMT-060 Phase I/II trial of their gene therapy treatment were shared at the Annual meeting of the American Society of Hematology held in San Diego. Sustained levels of Factor IX and cessation of prophylaxis were positive outcomes of the yearlong study with small group of hemophilia B patients. See full article at globenewswire.com.
CSL Behring Shares New Phase III Analysis of Idelvion for Hemophilia B
CSL Behring presented a data analyis of clinical trial results and factor level results for those treated with Idelvion, a prophylaxis therapy with longer time between treatment. Results suggest that keeping levels above 5 and 10 percent significantly reduced bleeding risk. The data was shared at the Annual meeting of the American Society of Hematology in San Diego. See full article at prnewswire.com.
Gene Transfer in Spark's Study Sustains Stable Factor Levels in Hemophilia B
Spark Therapeutics' SPK-9001, a gene therapy treatment undergoing trials, achieved sustained mean Factor IX levels greater than 30% without immunosuppression according to data presented at the Annual meeting of the American Society of Hematology. The treatment led to the termination of prophylaxis and nearly complete cessation of Factor use in patients. See full article at healio.com.
Alnylam Shares Positive Data on Fitusiran for Hemophilia Patients w/Inhibitors
Alnylam Announced Positive Results from Part D of its Phase I study of Fitusiran in patients with hemophilia and inhibitors. The once monthly treatment has proven to be well tolerated and is on track for a Phase 3 trial in 2017. Results were shared at the Annual meeting of the American Society of Hematology (ASH) held Dec 3-6, 2016 in San Diego, California. See full article at pipelinereview.com.
UT Austin Develops First Capsule to Treat Hemophilia
Engineers at the University of Texas at Austin have developed a biodegradable, oral delivery system, via a capsule, to provide protein therapy to hemophilia patients. Researchers sought ways to reduce the number of injections to treat hemophilia easier for children and for patients in countries where accessibility to the injected factor is difficult. See full article at news.utexas.edu.
NHF Addresses Post-Election Questions on Affordable Care Act and Advocacy
Val Bias, CEO of the NHF, posted a positive letter about potential changes to the Affordable Care Act in the wake of the Presidential Election results. The article explains the need for both sides to work together for positive change. He notes that issues that affect the bleeding disorder community have found victories with both Democrat and Republican administrations. See full article at hemophilia.org.
HFA Produces a Podcast Aimed at Young Adults with Hemophilia
The Hemophilia Federation of America has started a podcast specifically directed at young adults to encourage wellness in this growing demographic. The first podcast became available on October 26, 2016 and began with an interview on using exercise such as weight training for those with bleeding disorders. Listen and learn more at buzzsprout.com.
Adverse Events in Trial Dent Hopes for Roche Hemophilia Drug
Four patients in a clinical trial of an experimental hemophilia medicine from Roche have suffered serious adverse events. Problems related to blood clots appeared and patients were still experiencing breakthrough bleeding while on ACE910, the new medication under investigation, also known as emicizumab. See full article at reuters.com.
To Treat One Rare Blood Disorder, Scientists Exploit Another
In a study published in Blood, the Journal of the American Society of Hematology (ASH), researchers report that they have developed a new therapy for hemophilia based on research of a common clotting disorder, factor V Leiden. Research on the treatment is still in the early stages. See full article at medicalexpress.com.
Voices of 5000+ Patients: 1st National HTC Survey Experience
The CDC and the Hemophilia Federation of America hosted a webinar with our HTC's Brenda Riske and Rick Shearer along with UCLA's Judith Baker, leaders in creating the first national survey of HTC patients. They discuss methods and results from this survey of patients and the care they receive at their HTCs. Access this webinar at YouTube courtesy Hemophilia Federation of America.
Prescription Benefit Program Changes for Recombinant Clotting Factor Product
Patients who are using CVS/Caremark and have the "Advanced Control Formulary" plan may need to check their prescription coverage. Helixate FS, a product to treat hemophilia A, is being removed from the prescription benefit program for a limited number of patients. See full article at hemophiliafed.org.
FDA Warns of Risk of Hepatitis B Reactivating in Patients Treated for Hepatitis C
The US Food and Drug Administration has issued a warning about the risk of hepatitis B becoming an active infection again in patients who have had a previous infection and are treated with certain antiviral medicines for hepatitis C. In a few cases reactivation has resulted in serious problems. See full article at fda.gov.
Virtual Reality Games Make Infusions Easier on Young Patients
A pilot study at Nationwide Children's Hospital in Ohio, with a grant from the NHF, is currently testing the feasibility of integrating virtual reality technology into clinics. The feedback on the headset to help children reduce fear and anxiety during infusions has so far been positive. See full article at newswise.com.