FDA Accepts Beyer's Biologics License Application for BAY 94-9027
The FDA has accepted Bayer's Biologics License Application for BAY94-9027, an extended half life recombinant Factor VIII compound to treat hemophilia A in adults and adolescents. The formula is engineered to potentially prolong FVIII activity and may result in reduced frequency of infusions. See full article at prnewswire.com.
FDA Grants BioMarin Investigational New Drug Status to BMN 270
BioMarin's Investigational New Drug application for its gene therapy candidate BMN 270 to treat hemophilia A has been approved by the FDA. The decision was supported by clinical data of a Phase 1/2 trial showing five of six patients reaching low range of normal amounts of factor. See full article at hemophilianewstoday.com.
FDA Grants Orphan Drug Designation to Shire's SHP654 To Treat Hemophilia A
The US FDA has granted Shire with orphan drug designation for its gene therapy candidate SHP654 to treat hemophilia A. The therapy hopes to use a virus stereotype vector that is delivered to the liver that will help the body produce factor, reducing bleeds and the need to infuse factor. See full article at raredr.com.
UniQure Announces New Study for Hemophilia B Gene Therapy Program
UniQure announced their plans to advance AMT-061, which combines an AAV5 vector with the FIX Padua mutant, into a pivotal study in 2018 for patients with severe and moderately severe hemophilia B. Ongoing trials of similar AMT-061 already have shown promise. See full article at inpublic.globenewswire.com.
Hemophilia A Study Shows Potential to Reduce Inhibitors
A study on mice that was published in the journal Blood outlines findings that show potential in targeting ways to reduce inhibitors in Hemophilia A patients. The MZ B-cells found in the spleen were part of the focus in finding ways to prevent inhibitors from developing in patients. See full article at hemophilianewstoday.com.
BloodStream Podcast Presents Factor Revolution: 60 Years of Hemophilia
BloodStream Stories presents a new 3 part audio series detailing the last sixty years in hemophilia treatment. With over forty interviews the project gathers source material, news reports, and other original audio content to share the history of hemophilia treatment. Hear the podcast at bloodstream.libsyn.com.
How to Talk to Kids About Tragic Events
In the wake of numerous disasters and the violence of tragedies such as the shooting in Las Vegas, Children's Hospital Colorado has published an article to assist parents in finding ways to talk with their children about such tragedies. The article gives tips to help parents help their kids face tragedies and other events that could be frightening and stressful. See full article at childrenscolorado.org.
NHF Collects Funds to Support Puerto Rico Patients with Bleeding Disorders
The National Hemophilia Foundation has set up a relief fund for the bleeding disorders community in Puerto Rico following the damage caused to the island after hurricanes Irma and Maria. The NHF is working with the HTCs in Puerto Rico and the US to determine need and eligibility. To donate and for details see article at hemophilia.org.
Urgently Needed Hemophilia Drugs Delivered to Puerto Rico Pediatric Hospital
A shipment of life-saving Factor VIII and IX was delivered to University Pediatric Hospital in San Juan, Puerto Rico in the wake of the devastation from hurricane Maria. At the time of delivery the staff estimated they had between 24 and 72 hours until their supplies of the medication would be depleted. See full article at directrelief.org.
Catalyst Biosciences Granted FDA Orphan Drug Designation for CB2679/ISU304
Catalyst Biosciences announced that the US FDA has granted orphan drug designation for CB2679d/ISU304, their highly potent, subcutaneous, recombinant Factor IX variant for the treatment of hemophilia B. The variant is being evaluated in a Phase 1/2 clinical trial in South Korea. See full article at markets.businessinsider.com.
Seven Ways You Can Prepare for Emergencies
The National Hemophilia Foundation published an article especially for those with bleeding disorders to provide resources and suggestions on how to be prepared for emergencies. Links, phone numbers, and recommendations on how to prepare and how to keep supplies on hand and ready along with other items you might need during a disaster are listed. See full article at www.hemophilia.org.
New Section of Memorial Dedicated to People with Hemophilia who Died of AIDS
A new section of the National AIDS Memorial Grove has been dedicated to the lives of those with hemophilia who died of AIDS. The memorial is located in Golden Gate Park in San Francisco and hosted a ceremony to honor the lives of the estimated 10,000 hemophiliacs who died as a result of the virus in tainted blood supply products. See full article at sfchronicle.com.
"How do you thank someone for saving your life?"
Do you know the symptoms of thrombotic events? The Boston Globe wrote an article telling how a doctor who was attending a seminar recognized symptoms the speaker was experiencing as a potential pulmonary embolism. The doctor's knowledge and experience helped save a life. See full article at bostonglobe.com.
Alynlam's Hemophilia Program on Hold Following Patient Death
Alynlam's hemophilia clinical program that is testing fitusiran in patients with hemophilia A or B has been put on hold following the death of a patient in one of the company's trials. The company is suspending all clinical trials with fitusiran while they examine the evidence and determine whether or not the drug is associated with causing the death. See full article at raredr.com.
Catalyst Biosciences Announces Positive Clinical Data for Hemophilia B Trial
Catalyst Biosciences announced positive clinical data from the first Cohort of its ongoing Phase 1/2 proof-of-concept clinical trial in people with severe hemophilia B. The results support that CB 2679d has the potency advantage needed to be delivered as a convenient subcutaneous prophylactic therapy to prevent spontaneous bleeds. See full article at globenewswire.com.
Bioverativ and Bicycle Join to Develop Drugs for Hemophilia
Bicycle Therapeutics has partnered with Bioverativ to develop a new class of drugs for patients with rare blood disorders including hemophilia. The partnership will look to discover and select candidates for programs that will use the Bicycle Therapeutics technology called bicycles-inspired by their structure to optimize target specific therapies. See full article at labiotech.eu.
Bayer Submits BLA for Long-Acting Factor VIII for Hemophilia A
Bayer announced that they have submitted to the FDA a Biologics License Application (BLA) for BAY94-9027, a long-acting site-specifically PEGylated recombinant human Factor VIII designed to treat hemophilia A up to once every seven days. The BLA is supported by positive data from their Phase III PROTECT VIII trial. See full article at genengnews.com.
Pfizer Launches Innovative Technologies for Hemophilia Patients
Pfizer announced the launch of two first-of-its-kind innovative technologies for patients with hemophilia at the 69th Annual National Hemophilia Foundation Meeting in Chicago this August. Hemocraft, a modified version of the video game Minecraft, and HemMobile Striiv Wearable, a custom wristband designed to assist people with hemophilia, were on display during the meeting. See full article at raredr.com.
FDA Grants Priority Review of Genentech's Emicizumab for Hemophilia A
Genentech announced that the FDA has accepted their Biologics License Application (BLA) and granted Priority Review for emicizumab prophylaxis as a once-weekly subcutaneous treatment for adults, adolescents and children with hemophilia A with factor VIII inhibitors. See full article at businesswire.com.
NHF, HFA and Hemophilia of Iowa File Hemophilia Discrimination Complaint
Leading bleeding disorder organizations the National Hemophilia Foundation and the Hemophilia Federation of America joined Hemophilia of Iowa to file a legal complaint against Wellmark Blue Cross, Blue Shield. The action accuses Wellmark of violating the ACA and HIPAA laws and discriminating against patients in Iowa with hemophilia. See full article at prnewswire.com.
Hemophilia B Pediatric, Adult Patients Benefit from Idelvion, Studies Show
Results of studies CSL Behring's Idelvion shared at the 26th Biennial ISTH Congress in Berlin revealed positive findings for both adults and children with hemophilia B. The four studies cited indicated that Idelvion provided lower bleeding rates and higher quality of life for patients. See the full article at hemophilianewstoday.com.
Regis Jesuit's Elijah Warren Eager to Get Going at Arizona State
Elijah Warren, a local swimmer, and patient at our HTC, has signed to attend Arizona State University next fall. A senior this year at Regis Jesuit, Elijah has committed to swim for ASU and train under Bob Bowman, who trained Olympian Michael Phelps. Elijah has severe hemophilia and has worked hard to become a competitive swimmer on a national level. See the full article at aurorasentinel.com.
UniQure Presents Data on Hemophilia B Gene Therapy Research
Clinical data from uniQure was presented at the Congress of the International Society on Thrombosis and Hemostasis (ISTH) held in Berlin, Germany in early July. The presentation demonstrated that the presence of pre-existing anti-AAV5 neutralizing antibodies does not predict the potential efficacy of AAV5 mediated gene transfer in patients with hemophilia B. See the full article at pharmiweb.com.
BioMarin, Spark, Raising the Stakes for Hemophilia
While gene therapy is a promising new research area, BioMarin and Spark both presented data from trials at the recent International Society on Thrombosis and Hemostasis (ISTH) 2017 Congress in Berlin during July. Additional data on other upcoming treatments for hemophilia were also presented. See the full article at biopharmadive.com.
BioMarin Announces Results from Study of BMN 270 to Treat Hemophilia A
BioMarin announced an update to its previously reported interim results of a Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. The results show an annual bleed rate reduction of 97%. BioMarin plans to initiate a Phase 3 trial later this year. See the full article at streetinsider.com.
Promising New Data from Alnylam and Sanofi for Fitusiran
An experimental hemophilia drug from Alnylam that was developed in a partnership with Sanofi has promising new results. Data from a year long clinical trial show that 48% have not had any bleeding events. Before the study patients were reporting a median of 20 bleeding events per year. See the full article at statnews.com.
New England Journal of Medicine Publishes Latest Update on the HAVEN 1 Trial
The New England Journal of Medicine published an article reviewing results of a study of prophylactic treatment of hemophilia A patients with inhibitors with Emicizumab, also known as ACE910. The results showed those treated had a bleeding rate that was 87% lower than those with no treatment. See the full article at nejm.org.
Shire Submits Application to the FDA for Gene Therapy to Treat Hemophilia A
Biotech firm Shire announced that they have submitted an application to the FDA for a gene therapy drug that treats hemophilia A. SHP654, which was also designated as BAX 888, was submitted based on pre-clinical and Phase 1 studies that showed promise for both hemophilia A and B patients. See the full article at iii.co.uk.