Hematology

FDA expands Vonvendi approval for all von Willebrand disease types, enhancing treatment options for adults and children with this rare condition.

Agios announces a 3-month extension for mitapivat's FDA review.

An audio recap of the top 5 stories in healthcare news from the week of 08/24-08/30.

Stay updated with the latest healthcare breakthroughs, including FDA news, vaccine guidelines, and orforglipron data, in this week's essential news roundup.

If approved, the investigative therapy could provide a frontline treatment for patients with SAA who do not have matched sibling donors for cell transplantation.

Stay updated with the latest healthcare breakthroughs, including FDA news and new guidelines, in this week's essential news roundup.

This new monoclonal antibody has achieved a statistically significant improvement in time to treatment failure, indicating a potential to revolutionize ITP.

The FDA approved generic versions of Venofer (iron sucrose) injection from Viatris Inc and Amphastar Pharmaceuticals.

This FDA News Month in Review provides a round-up of regulatory decisions from July 2025.

The therapy was originally approved under the name Alhemo for people with hemophilia ages 12 and up without inhibitors.

Our recap of the first half of 2025 highlights 5 regulatory updates, 5 trial announcements, and 3 top perspectives in hematology.

A review of the 16 novel drugs approved by the US Food and Drug Administration during the first half of 2025, with links to coverage from MJH Life Sciences publications.

A relatively quiet quarter for hematology, punctuated by both successes and failures in clinical trials and a handful of Orphan Drug and Fast Track designations.

The FDA distributed several designations and many trials successfully met their endpoints during an eventful June for hematology.

Parent company Novo Nordisk has announced intentions to submit the treatment for approval later in 2025.

Data from the Phase 1/2 BEACON trial indicate the efficacy and safety of this investigative 1-time therapy, particularly in decreasing blood cell sickling.

Hereditary hemorrhagic telangiectasia has no approved treatments; this designation, in addition to the European Medicines Agency granting a positive opinion, positions DIAG723 to be the first.

Investigators also noted the risk of Hb overshoot in patients switching from ESA to roxadustat, emphasizing the importance of monitoring Hb levels before and after treatment.

An audio recap of the top 5 stories in healthcare news from the week of 06/02-06/08.

Stay updated with the latest healthcare breakthroughs, including FDA approvals and new phase 3 clinical trial data, in this week's essential news roundup.

An ongoing phase 1 study aims to prove the safety and tolerability of VAS-101, as well as its effect on blood flow dynamics, red blood cell sickling, and oxygen dissociation.

Novartis has announced the safety and efficacy of iptacopan, a twice-daily oral monotherapy for adult patients who switched from eculizumab or ravulizumab.

If approved, mavorixafor will be the second FDA-approved treatment and the first oral medication for CN.

Investigators compared the medications across various patient cohorts with different causes of anemia, finding evidence almost uniformly in favor of FCM.

A recent analysis reveals no significant difference between restrictive and liberal transfusion strategies on quality of life in patients with myocardial infarction and anemia.