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.
Miscellaneous Neurodegenerative Disorders
Ravulizumab Gets Priority Review for Generalized Myasthenia Gravis
The application is supported by data from the phase 3 study that evaluated the efficacy and safety of ravulizumab-cwvz in 175 adults with generalized myasthenia gravis who were not previously treated with a complement inhibitor.
Gene Therapy Gets Priority Review for Cerebral Adrenoleukodystrophy
Elivaldogene autotemcel (eli-cel) is a one-time gene therapy designed to add functional copies of the ABCD1 gene into a patient’s own hematopoietic stem cells, resulting in the production of ALDP.
Rozanolixizumab Meets All Endpoints in Generalized Myasthenia Gravis Trial
Rozanolixizumab is a humanized monoclonal antibody that specifically binds to human neonatal Fc receptor (FcRn) with high affinity.
Trofinetide Promising for Patients With Rett Syndrome
Trofinetide is a novel synthetic analogue of the amino-terminal tripeptide of IGF-1 designed to treat the core symptoms of Rett syndrome.
Trehalose Fast Tracked for Spinocerebellar Ataxia
SLS-005 contains the active ingredient trehalose, a low molecular weight disaccharide (0.342 kDa) that has been shown to penetrate muscle and cross the blood brain barrier.
FDA Clears Language Independent Computerized Test to Detect Cognitive Impairment
By using animal images, the test avoids cultural or educational bias and can be used repeatedly with no learning effect.
Novel CRISPR Therapy Gets Orphan Drug Status for Transthyretin Amyloidosis
The Company is currently evaluating the efficacy and safety of NTLA-2001 in adults with hereditary ATTR amyloidosis with polyneuropathy in a phase 1 clinical study.
FDA Assigns PDUFA Date for Donepezil Transdermal System
Adlarity is a once-weekly transdermal formulation of donepezil.
MIND Diet Tied to Better Cognitive Function in Older Adults
MIND diet is a hybrid of the Mediterranean diet and the Dietary Approaches to Stop Hypertension diet
