Bleeding Disorders

Gene Therapy Shows Promise in Factor IX Coagulant Activity for Hemophilia B

December 07, 2017

In all participants, vector-derived factor IX coagulant activity was sustained, with a mean steady-state factor IX coagulant activity of 33.7 ± 18.5%.

ACC: New Guidance on Managing Bleeding in Patients on Oral Anticoagulants

December 04, 2017

At each step, patient specific factors should be considered.

Hemophilic Arthropathy Treatment Gets Orphan Drug Designation

By November 27, 2017

TRM-201 (rofecoxib) is a highly potent COX-2 selective non-steroidal anti-inflammatory drug (NSAID) with a well-established efficacy profile.

Alprolix Labeling Updated With Long-Term Data, Additional Adverse Reaction

By November 27, 2017

Alprolix is a recombinant clotting factor therapy developed using Fc fusion technology to prolong circulation in the body.

Hemlibra Approved to Treat Hemophilia A With Inhibitors

By November 16, 2017

Hemlibra is a bispecific factor IXa- and factor X-directed antibody indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors.

Effect of Treatment Delay With Tranexamic Acid Examined

November 08, 2017

Overall, 63% of bleeding deaths occurred within 12 hours of onset. There was a peak in deaths from postpartum hemorrhage at 2 to 3 hours after childbirth.

BLA for Investigational Hemophilia A Treatment Accepted

By October 31, 2017

BAY94-9027 is designed to prolong FVIII activity in the blood while preserving coagulation activity using site-specific technology in which the Polyethylene glycol molecule is consistently attached to the factor VIII protein at a specific site.

Voluntary Market Withdrawal of Octagam 10% Announced

By October 23, 2017

The Company has decided in conjunction with the FDA to suspend further administration of Octagam 10% from these specific production lots.

Anticoagulants Taken With Certain Meds May Up Bleeding Risk

October 05, 2017

And antithrobotic medications linked to higher rate of hematuria-related events in older adults

Prophylaxis vs. On Demand Tx in Patients with Severe Hemophilia

August 31, 2017

Overall, 35.7% of prophylaxis participants were bleed-free and 76.2% had fewer than 2 BEs per year.

BLA for Novel Hemophilia A Therapy Granted Priority Review

By August 24, 2017

The FDA has accepted and granted Priority Review to the Biologics License Application (BLA) of emicizumab (Genentech) for the prophylactic treatment of hemophilia A in adults, adolescents and children with factor VIII inhibitors.

FDA Approves Additional Vial Strengths of Nuwiq for Hemophilia A

By August 22, 2017

In addition to the currently available strengths (250, 500, 1000, 2000 IU), starting in September, Nuwiq will also be supplied in 2500, 3000, and 4000 IU vial strengths.

Caplacizumab Fast-Tracked for Acquired Thrombotic Thrombocytopenic Purpura

By July 26, 2017

The Phase 2 TITAN study (n=75) evaluated the safety and efficacy of caplacizumab with standard of care of plasma exchange and immunosuppression.

Idarucizumab Effective for Dabigatran Reversal in Emergency Situations

July 13, 2017

Effective for dabigatran reversal among patients with uncontrolled bleeding, undergoing urgent surgery

Emicizumab Beneficial in Hemophilia A Patients With Inhibitors to FVIII

By June 29, 2017

The primary analysis of the study arm showed a statistically and clinically significant reduction in bleeding by 87% (risk rate [RR] 0.13; P<0.0001] with emicizumab prophylaxis.

NDA Filed for Tavalisse for Immune Thrombocytopenia Treatment

By June 19, 2017

Tavalisse is an oral treatment designed to inhibit SYK kinase, a key signaling member in the immune process that leads to platelet destruction in ITP.

PPIs May Benefit Older Patients on Antiplatelet Therapy

June 15, 2017

Research suggests proton-pump inhibitors should be co-prescribed in patients age 75 years

Anticoagulation During Pregnancy Linked to Distinct Maternal, Fetal Risks

June 02, 2017

VKA, LMWH tied to lowest risk of adverse maternal, fetal outcomes, respectively

Rebinyn Approved to Treat Hemophilia B

By June 01, 2017

Rebinyn, a recombinant DNA-derived coagulation Factor IX concentrate, temporarily replaces the missing coagulation Factor IX in hemophilia B patients.

Novel Hemophilia A Gene Therapy Gets Fast Track Status

By May 16, 2017

The Food and Drug Administration (FDA) has granted Fast Track designation to SB-525 (Sangamo Therapeutics) for the treatment of hemophilia A.

FDA to Review Fostamatinib for Chronic, Persistent ITP

By April 18, 2017

Rigel announced its submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA) for fostamatinib for the treatment of patients with chronic and persistent immune thrombocytopenic purpura (ITP).

Emicizumab Evaluated in Children with Hemophilia A

By April 17, 2017

The interim results from HAVEN 2, after a median of 12 weeks of treatment, showed that prophylaxis with emicizumab is associated with clinically meaningful reduction in the number of bleeds over time.

Major Bleeding Risk Similar with Antiplatelets, OAs in Elderly AF Patients

April 17, 2017

Elderly taking aspirin, clopidogrel have major bleeding similar to that seen with oral anticoagulants

Is tPA Use Safe for Sickle Cell Patients in Acute Ischemic Stroke?

By April 11, 2017

Commenting on the study's results, lead author Robert J. Adams, MD, said, "Having sickle cell disease did not adversely affect any of the indicators we measured."

First Consumer Genetic Health Risk Tests Get FDA Approval

By April 06, 2017

While the GHR tests may provide genetic risk information, they cannot determine a person's overall risk for developing a disease, as other factors beyond genetics (ie, environment, lifestyle) may also influence risk.

Intracranial Bleed Risk Compared for Apixaban, Warfarin in A-fib Patients

By March 29, 2017

Patients taking apixaban showed significantly less intracranial hemorrhage (0.33% per year) vs. patients taking warfarin (0.80% per year) no matter the type and location.

CV, Bleeding-Related Mortality Examined Post-Coronary Stenting

By March 24, 2017

Fifty-two (10.9%) of the patients who experienced an ischemic event, and 41 (17.7%) who suffered experieced a bleeding event, died.

Nipride RTU Approved for Immediate BP Reduction

By March 10, 2017

Nipride RTU is approved for immediate blood pressure reduction and also carries a Boxed Warning describing the possibility of excessive hypotension and cyanide toxicity.

Investigational Hemophilia B Gene Therapy Granted Breakthrough Status

By January 30, 2017

The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to the investigational gene therapy, AMT-060 (uniQure), for the treatment of severe hemophilia B.

FDA Review Underway for Eptacog Beta in Hemophilia

By January 06, 2017

LFB S.A. announced that the FDA has accepted for review the Biologic License Application (BLA) of its recombinant Coagulation Factor VIIa (eptacog beta, activated) for the potential treatment of congenital hemophilia A or B in adolescents and adults with inhibitors.