Voxelotor Gets Priority Review for Pediatric Sickle Cell Disease
The sNDA and NDA are supported by data from the open-label phase 2a HOPE-KIDS1 study.
Sickle Cell Disease
Treatment at Infusion Center Beneficial in Sickle Cell Disease
Outcomes significantly better for treatment of vaso-occlusive crises at infusion center versus emergency department
Ferriprox Approved for Transfusional Iron Overload Due to Sickle Cell Disease
The Food and Drug Administration (FDA) has expanded the approval of Ferriprox® (deferiprone) tablets to include the treatment of transfusional iron overload due to sickle cell disease (SCD) or other anemias in patients 8 years of age and older. Additionally, the oral solution formulation of Ferriprox has been approved for use in patients 3 years…
Gene Therapy for Sickle Cell Did Not Cause Patient’s Leukemia
Bluebird Bio says it has not found evidence that its gene therapy caused the sickle cell patient’s leukemia
Trial of Gene Therapy for Sickle Cell Disease Halted
Two patients participating in the study developed cancer years after treatment
MPR Weekly Dose Podcast Episode 57
This week we cover a new treatment submission to the FDA for the prevention of diabetes; The launch of a bupivacaine implant to treat post op pain; The DEA schedules a newly approved opioid; The FDA classifies over 200 drugs as essential and wants manufacturers to ramp up production; And more news on rifamixin and other drugs that are in the pipeline.
Endari Label Updated With Benefit Statement, Instructions for Use
Endari (L-glutamine; Emmaus Medical) is an amino acid indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients aged 5 years and older.
Rifaximin Gets Orphan Drug Status for Sickle Cell Disease
Rifaximin is believed to reduce VOCs in patients with sickle cell disease through microbial modulation.
Siklos Now Available in 100mg Scored Tablets
Siklos is indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in children with sickle cell anemia.
Medical Marijuana Tied to Fewer Admissions in Sickle Cell Disease
But no change in opioid use observed for those receiving medical marijuana certification
