FDA

Legend Biotech has a target action date of February 28 for the company’s BLA for ciltacabtagene autoleucel (cilta-cel) for adults with relapsed and/or refractory multiple myeloma. The BLA was submitted by Legend’s collaboration partner, Janssen Biotech Inc., a Johnson & Johnson company.

BioMarin

BioMarin updated several of the company’s gene therapy programs on February 17 following a September 2021 update, when the drug manufacturer reported the U.S. Food and Drug Administration had placed a clinical hold on the BMN 307 Phearless Phase I/II trial.

The U.S. Food and Drug Administration approved Agios Pharmaceuticals Inc.’s oral PK activator Pyrukynd (mitapivat) as the first approved disease-modifying therapy for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency. 

The U.S. Food and Drug Administration approved Sanofi’s proposed drug to address red blood cell destruction in patients living with a rare form of anemia called cold agglutinin disease (CAD). 

Sierra Oncology shared positive topline results from the company’s study on the effectiveness of momelotinib to treat patients with myelofibrosis.

The next “giant leap” for humans may be a trip to Mars, but having enough oxygen-carrying red blood cells for the journey might present a challenge, new research suggests.

BioMarin Pharmaceutical shared positive results from the company’s ongoing Phase III GENEr8-1 trial on the viability of valoctocogene roxaparvovec as a treatment for patients diagnosed with severe hemophilia A.

Brisbane, California-based Sangamo Therapeutics announced that Sanofi US was returning rights to SAR445136 as the Paris-based company shifts its approach from personalized cell therapies to allogeneic off-the-shelf genomic approaches.

Shares of Global Blood Therapeutics were up more than 10 percent in trading on December 17 following regulatory approval of a supplemental New Drug Application for oral Oxbryta (voxelotor) tablets for the treatment of pediatric sickle cell disease (SCD).

GlaxoSmithKline

GlaxoSmithKline is lining up a potential new treatment for patients with anemia due to chronic kidney disease following positive results in a Phase III study that showed the investigational candidate daprodustat improved or maintained hemoglobin levels without an increase in cardiovascular risk.