FDA Approves Expanded Indication for Hemlibra in Hemophilia A

By October 04, 2018

The expanded approval was based on data from two Phase 3 clinical trials: HAVEN 3 and HAVEN 4.

Jivi With Step-Wise Dosing Approved for Management of Hemophilia A

By August 30, 2018

The approval was supported by data from the phase 2/3 international, open-label PROTECT VIII trial (N=126) that evaluated previously treated patients aged ≥12 years with severe hemophilia A.

Larger Vial Size Approved for Hemophilia B Treatment Idelvion

By May 31, 2018

The new 3500 IU vial size will help shorten the reconstitution time needed to prepare multiple vials for a similar dose.

BLA for Extended Half-Life Factor VIII Hemophilia A Tx Filed With FDA

By February 27, 2018

N8-GP (turoctocog alfa pegol) is a glycopegylated form of turoctocog alfa designed for prolonged half-life.

Rebinyn Now Available for the Treatment of Hemophilia B

By February 08, 2018

Rebinyn, a recombinant DNA-derived coagulation Factor IX concentrate with an extended half-life, was approved by the Food and Drug Administration (FDA) in May 2017.

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.

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.

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.

Hemophilia A Treatment Gains Expanded Approval

By December 27, 2016

Adynovate, an extended circulating half-life recombinant Factor VIII (rFVIII), was initially approved for use in patients aged ≥12 years with hemophilia A for on-demand treatment and control of bleeding episodes, and routine prophylaxis to reduce the frequency of bleeding episodes.

Emicizumab Prophylaxis Reduced Number of Bleeds in Hemophilia A Trial

By December 22, 2016

The primary endpoint was the number of bleeds over time among patients with emicizumab prophylaxis or no prophylaxis.