Hematology

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.

A relatively quiet month for hematology, marked by brief ups and downs for trials and new research on anemia and sickle cell disease.

Investigators examined emergency department utilization as a product of age, patient-reported severity of vaso-occlusive episodes, and Social Vulnerability Index score.

Iron deficient diets can lead to anemia and various other health complications in adulthood.

Iron deficiency affects millions globally, especially women, yet remains underdiagnosed. Regular screening can prevent severe health issues and improve lives.

Prothena halts birtamimab development after missing key Phase 3 trial endpoints for AL amyloidosis, prompting workforce reductions and cost-cutting measures.

Stay updated with the latest healthcare breakthroughs, including FDA approvals and innovative treatments, in this week's essential news roundup for professionals.

Interim results from the ongoing trial indicated achievement of all efficacy endpoints, with no safety concerns reported.

A study reveals a significant link between iron deficiency anemia and increased ischemic stroke risk in young adults, highlighting urgent screening needs.