Hematological Disorders

FDA Approves Epoetin Alfa Biosimilar Retacrit

By May 15, 2018

The approval of Retacrit was based on a review of evidence that included human pharmacokinetic/pharmacodynamic data and clinical immunogenicity data which demonstrated a high degree of similarity between Retacrit and its reference product.

First Generic Alternative for Mephyton Now Available

By May 15, 2018

Amneal's generic product is available in a 5mg strength tablet supplied in 100-count bottles.

FDA Approves Hemospray to Treat Non-Variceal GI Bleeds

By May 09, 2018

The product is a single-use device that delivers hemostatic powder through the channel of an endoscope.

Andexxa Approved for Reversing Anticoagulant Activity of Rivaroxaban, Apixaban

By May 04, 2018

Andexxa, a recombinant modified human Factor Xa (FXa) protein, was approved under the FDA's Accelerated Approval pathway.

FDA Committees Vote Against Drug Tx for Severe Neonatal Jaundice

By May 04, 2018

Stannsoporfin, a heme oxygenase inhibitor, works by inhibiting bilirubin production at its source.

Sickle cell trait not linked to increased risk of ischemic stroke

April 27, 2018

In this meta-analysis of prospective population-based studies, sickle cell trait (SCT) was not associated with any difference in risk of first ischemic stroke.

IBD and Anemia: Adverse Events After IV Iron Therapy Examined

By April 23, 2018

To examine the occurrence of adverse reactions in this patient population, researchers from John Hopkins University, Baltimore, Maryland, conducted a retrospective analysis of IBD patients who received IV iron infusions between 2010 and 2014 in the commercial claims database, MarketScan.

Gene Therapy Looks Promising for Transfusion-Dependent β-Thalassemia

By April 19, 2018

The safety and efficacy of LentiGlobin were evaluated in 2 independent 2-year clinical studies: HGB-204 (Northstar, N=18), an open-label, single-dose, nonrandomized, multicenter Phase 1/2 study in patients with TDT, and HGB-205, an ongoing open-label, single-dose, nonrandomized, single-center Phase 1/2 study in patients with TDT and severe sickle cell disease.

First-in-Class Treatment Approved for Chronic Immune Thrombocytopenia

By April 18, 2018

Tavalisse is an oral spleen tyrosine kinase (SYK) inhibitor.

Hemlibra Gets Breakthrough Tx Status for Hemophilia A Without Inhibitors

By April 18, 2018

The trial enrolled 152 patients with hemophilia A who were previously treated, either on-demand or as prophylaxis, with factor VIII therapy.

Praxbind Gains Full FDA Approval as Pradaxa Reversal Agent

By April 17, 2018

Complete reversal was seen within 4 hours in the majority of patients, as measured by ecarin clotting time (ECT 82%) or diluted thrombin time (dTT 99%). In addition, there was a low late of thrombotic events and no new safety events were reported.

Vonvendi Approved for Peri-Operative Management of Bleeding in VWD Patients

By April 17, 2018

Safety and efficacy of Vonvendi with or without recombinant factor VIII (FVIII) treatment in elective surgical procedures in patients aged ≥18 years diagnosed with severe von Willebrand disease, was established in the Phase 3 trial.

Iron Formulation and Cancer Risk: Is There a Link?

By April 12, 2018

In the U.S., ferric citrate is commonly found in dietary supplements and ferric EDTA is often used as a food additive.

Certain Lots of Kcentra Recalled Due to Potential Risk of Vial Breakage

By April 06, 2018

The recall notification is due to a potential risk of breakage of the glass vials during transport because of a change in the secondary packaging configuration.

FDA Approves Third Countermeasure for Acute Radiation Syndrome

By March 30, 2018

The FDA approval was supported by efficacy studies in animals as they could not be ethically conducted in humans.

Oral vs. Intramuscular Vitamin B<sub>12</sub>: Efficacy, Safety Compared

By March 16, 2018

Researchers from Beijing University of Chinese Medicine and the Catalan Health Institute, Spain sought to investigate the effects of oral vitamin B12 vs. IM vitamin B12 to determine whether outcomes for vitamin B12 deficient patients would differ based on the formulation.

Comparative Safety of NSAIDs Among Clopidogrel Users Investigated

By March 15, 2018

Using Medicaid claims from 5 states (1999-2010) and Medicare claims for dual-enrollees, study authors analyzed the first concomitant use of clopidogrel and 1 of 10 selected NSAIDs (ibuprofen, celecoxib, naproxen, rofecoxib, meloxicam, diclofenac, indomethacin, valdecoxib, nabumetone, etodolac) after a 1-year baseline period.

Interim Data Show Andexanet Alfa Rapidly Reverses Anti-Factor Xa Activity

By March 13, 2018

ANNEXA-4 is a global, single-arm, open-label clinical trial designed to evaluate andexanet alfa in patients who present with an acute major bleed while receiving apixaban, rivaroxaban, edoxaban or enoxaparin.

Hematological Reference Values

March 06, 2018

Reference values for commonly ordered blood tests.

Pharmacokinetic Dosing Software for Hemophilia A Treatment Now Available

By March 05, 2018

The myPKFiT software can be used to generate Advate dosage amount and frequency recommendations for routine prophylaxis for an individual patient 16yrs of age and older and body weight of 45kg or greater.

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.

Lusutrombopag Under Priority Review for Thrombocytopenia in Liver Disease

By February 26, 2018

The primary endpoint was the number of patients who required no platelet transfusion priory to primary invasive procedure and no rescue therapy for bleeding for 7 days post-procedure.

BLA for Novel Hereditary Angioedema Treatment Accepted by FDA

By February 26, 2018

Results found that those who received a subcutaneous administration of 300mg lanadelumab once every 2 weeks had an 87% reduction in mean frequency of HAE attacks.

Pharmacologic Tx for Severe Neonatal Jaundice to Be Reviewed by FDA

By February 23, 2018

The FDA has set an action date of August 22, 2018 to respond to the NDA

Treating Endothelial Function May Target Root Disease in Marfan Syndrome

February 13, 2018

The researchers found that the MFS/ATR1a-null mice showed unabated aortic root enlargement and remained responsive to losartan, despite being hypotensive and resistant to AngII vasopressor effects.

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.

Antibiotic Prophylaxis Rates Low for Pediatric Sickle Cell Patients

February 06, 2018

Data were included for 2,821 children with 5,014 person-years. The researchers found that 18 percent of children received ≥300 days of antibiotics.

FDA Approves Expanded Use for Feraheme in Iron Deficiency Anemia

By February 05, 2018

The Food and Drug Administration broadened the existing labeling for Feraheme (ferumoxytol injection) beyond the current chronic kidney disease indication.

Results Released in Multi-Year IV Bevacizumab Study for HHT-Related Bleeding

February 05, 2018

After completion of the initial bevacizumab treatment cycle, there was a significant reduction in epistaxis severity scores and RBC transfusion requirements.

Should all Syncope Patients Undergo Evaluation for Pulmonary Embolism?

January 30, 2018

At 90 days, the prevalence of venous thromboembolism varied from 0.3 to 1.37% for all patients and from 0.75 to 3.86% for hospitalized patients.