Zydelig US Indications Withdrawn for Follicular Lymphoma, Small Lymphocytic Leukemia
Continued approval required further verification and description of clinical benefit in confirmatory trials.
Hematologic Disorders
FDA Extends Review Period for Gene Therapies for Beta-Thalassemia, Cerebral Adrenoleukodystrophy
The FDA extended the target dates for beti-cel and eli-cel to allow time to review additional clinical information for both applications.
Red Cross Says US Blood Supply at Dangerously Low Level
People who have received COVID-19 vaccines can donate blood and platelets as long as they are symptom-free
Durable Response Observed With Gene Therapy for Hemophilia A
The phase 3 GENEr8-1 study evaluated valoctocogene roxaparvovec in adults with severe hemophilia and residual FVIII levels less than or equal to 1 IU/dL.
New Drugs Approved in 2021
Visit our New Drug Products page to stay up-to-date on the latest drug approvals.
Eltrombopag Augments Immunosuppression in Severe Aplastic Anemia
Improvement seen in the rate, rapidity, strength of hematologic response in previously untreated patients with severe aplastic anemia
Oxbryta Approval Expanded to Younger Patients With Sickle Cell Disease
The approval for both applications was based on data from the open-label phase 2a HOPE-KIDS1 study, which evaluated the efficacy and safety of Oxbryta 300mg dispersible tablet in 45 patients aged 4 to 11 years with sickle cell disease.
Injectafer Approval Expanded to Pediatric Patients With Iron Deficiency Anemia
Injectafer is an iron replacement product.
Orencia Approved for Prevention of Acute Graft Versus Host Disease
The approval was based on data from the placebo-controlled phase 2 GVHD-1 trial and real world evidence from a registry trial.
Siklos Approval Expanded to Include Adults With Sickle Cell Anemia
The approval was based on data from the European Sickle Cell Disease Cohort study, which evaluated the efficacy and safety of Siklos in 1077 adults with sickle cell disease.
