Hematological Disorders

The PDUFA action date is May 10, 2024.

Aphexda is expected to be available in September 2023.

Crovalimab is an investigational anti-complement 5 recycling monoclonal antibody.

During extended treatment, bleeding risk was similar for apixaban and rivaroxaban.

Overall median annualized bleeding rate was 0 with concizumab.

Lower treatment retention rates seen with 30-day, out-of-pocket costs $150 or greater.

The approval was based on data from the pivotal phase 3 COMMANDS trial that compared luspatercept to epoetin alfa in 356 patients with anemia and lower-risk MDS.

Imetelsat is an investigational first-in-class telomerase inhibitor.

Balfaxar is a non-activated 4 factor prothrombin complex concentrate containing vitamin K-dependent factors.

Study supports the benefit of potential new Alzheimer drug; RSV treatment approved for neonates and infants; Veklury approval expanded; a blood test used to diagnose life-threatening heart muscle injuries has been recalled; Brenzavvy available for adults.

The safety, tolerability, and immunogenicity of STAR-0215 is being evaluated in the ALPHA-STAR clinical trial.

Study findings suggest clinicians may safely prescribe alpha blockers to help manage hypertension in patients receiving hemodialysis.

Endo’s product is the only ready-to-use formulation of bivalirudin currently on the market.

Ryplazim works by temporarily increasing plasminogen levels in blood.

The table below is a review of notable updates that occurred in June 2023 for investigational products in development (not an inclusive list). Click on the status to view our full coverage.

Roctavian is the first gene therapy approved for severe hemophilia A.

A regulatory decision is expected in the second quarter of 2024.

Risk for anemia incidence and decline in ferritin greater for seniors receiving low-dose aspirin.

No improvement seen in favorable functional outcome at 6 months, but reduced risk of death at 28 days after injury.