Plant Based Hemophilia Therapy Shows Promise to Reduce Inhibitors
Research at the University of Pennsylvania and University of Florida has led to new potential therapies to prevent inhibitors by using lettuce cells to teach the body to tolerate factor. The positive results, previously tested in mice, and now tested in dogs have shown encouraging results. See full article at news.upenn.edu.
New Drugs to Treat Hepatitis C Under Priority Review at FDA
The FDA has accepted under priority review AbbVie's new drug application seeking approval of its once-daily regimen of medications to treat all major genotypes of chronic hepatitis C. The review will reduce the review time frame from ten months to six to potentially move the possible cure to market faster. See full article at pharmaceutical-businessreview.com
Dimension Announces Results from Phase 1/2 Trial for DTX101-AAV
Dimension Therapeutics announced early results of their phase 1/2 study of DTX101 for the treatment of adult patients with moderate to severe hemophilia B. The AAV gene therapy trial in a clinical setting is designed to evaluate the safety, dose and efficacy of DTX101 in adult patients. See full article at satprnews.com.
uniQure's AMT-060 Gains Breakthrough Designation from FDA for Hemophilia B
The FDA has granted breakthrough therapy designation to AMT-060 for the treatment of severe hemophilia B. The treatment is an investigational gene therapy program. Results of a dose-ranging phase 1/2 study showed sustained increases in Factor IX, reduction in factor replacement needed, and a near cessation of spontaneous bleeding at 12 months. See full article at healio.com.
FDA Clears Sangamo's SB-525 Gene Therapy for Investigational New Drug
Sangamo BioSciences announced that the US FDA has cleared the company's Investigational New Drug Application for its SB-525 gene therapy program to treat Hemophilia A. The approval allows Sangamo to begin clinical trails evaluating the adeno-associated virus (AAV2/6) cDNA human Factor 8 treatments. See full article at pipelinereview.com.
NBC Checks Back in with Real Life Super Hero Kid 'Iron Max'
Max Levy earned the name "Iron Max" following a port placement surgery due to his severe hemophilia. Marvel Comics featured him in an Iron Man Comic-his favorite, and made him a super hero kid. NBC visited Iron Max after a year to see how he was doing and what he has been up to. See full article at nbcnews.com.
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.