Hematologic Disorders

FDA Grants Zelboraf Priority Review for Rare Blood Disease

By August 07, 2017

The application includes data from the open-label, Phase 2 VE-BASKET study; non-randomized, basket study investigating the use of Zelboraf in patients with BRAF V600 mutation-positive cancers and other diseases, including 22 patients with ECD.

Novel Synthetic Bio Drug for Hyperammonemia Granted Fast Track

By June 26, 2017

Synthetic Biotic medicines utilize synthetic biology to reprogram probiotic bacteria to perform critical functions that compensate for those missing or damaged due to a particular disease.

First ESA Biosimilar Gets FDA Advisory Committee Nod

By May 26, 2017

The FDA will consider the Committee's recommendation before acting on the biologics license application (BLA) for the epoetin alfa biosimilar.

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.

Soliris Application Seeks Ultra-Rare Neuromuscular Disease Indication

By January 09, 2017

Alexion has submitted a marketing application to the Food and Drug Administration (FDA) to extend the indication for Soliris (eculizumab) to be a treatment for patients with refractory generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.

FDA Fast Tracks Rare Hematological Disorder Treatment

By December 20, 2016

Apellis has announced that the Food and Drug Administration (FDA) has granted Fast Track designation to APL-2, a novel drug candidate studied for the treatment of paroxysmal nocturnal hemoglobinuria (PNH)

ASH: Benefits of Xarelto in Cancer-Associated Thrombosis Presented

By December 06, 2016

At the 2016 American Society of Hematology (ASH) Annual Meeting, researchers from Janssen and Bayer presented data showing that cancer patients treated with Xarelto (rivaroxaban) for blood clots had significantly fewer emergency room (ER) visits and lower healthcare costs at start of therapy vs. patients given standard treatment.

Certain Antidepressants May Up Risk of Intracranial Hemorrhage

By December 05, 2016

The cohort included 1,363,990 incident users of antidepressants.

Platelet-Reducing Agent Available Again

By December 05, 2016

Following an 11-month shortage, the Food and Drug Administration (FDA) has announced that Agrylin (anagrelide HCl; Shire) 0.5mg capsules are now available.

New RBC Transfusion and Storage Recommendations Published

October 17, 2016

In a clinical practice guideline published online Octocber 12 in the Journal of the American Medical Association, recommendations are presented for the target hemoglobin level for red blood cell (RBC) transfusion and optimal duration of RBC storage.

Bacteriuria ID'd as Possible Trigger of Higher Edema Incidence

August 25, 2016

For patients with hereditary angioedema due to C1-inhibitor deficiency (C1-INH-HAE), those with bacteriuria have a higher number of edematous attacks, according to a study published online in Allergy.