September 2022 Recap: Drug Pipeline Updates
The table provided is a review of notable updates that occurred in September 2022 for investigational products in development.
Neurology
Trofinetide Gets Priority Review for 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.
Systemic, Inhaled Glucocorticoid Use Linked to Changes in the Brain
To investigate the association between glucocorticoids and and brain structure changes, researchers used data from the UK Biobank.
FDA Rejects Pimavanserin sNDA for Alzheimer Disease Psychosis
The sNDA for Alzheimer disease psychosis included data from the phase 3 HARMONY and phase 2 Study-019 trials.
![HYQVIA (immune globulin [human] 10% with recombinant human hyaluronidase) by Baxter and Halozyme](https://www.empr.com/wp-content/uploads/sites/7/2018/12/hyqviaarticle_656631.png)
Hyqvia Reduces Relapse in Chronic Inflammatory Demyelinating Polyradiculoneuropathy Trial
The ADVANCE-1 trial included adults with a confirmed diagnosis of CIDP who had remained on a stable dosing regimen of IVIG therapy for at least 3 months prior to screening.
Acadia Seeks Approval for Rett Syndrome Treatment Trofinetide
Trofinetide is a novel synthetic analogue of the amino-terminal tripeptide of IGF-1 designed to reduce neuroinflammation and supporting synaptic function.
Effects of Intranasal Insulin on Cognition, Mobility Studied in Elderly
Evidence from phase 2 trial suggests positive effects on cognition and gait in elderly patients with type 2 diabetes
Labeling for Migraine Treatment Rimegepant Updated With Lactation Data
Rimegepant is a calcitonin gene-related peptide (CGRP) receptor antagonist.
Elamipretide Gets Orphan Drug Status for Friedreich Ataxia
Elamipretide has been shown to improve frataxin levels in Friedreich ataxia patient-derived cells, and improve motor and cardiac function in an animal model of Friedreich ataxia.
Gene Therapy for Canavan Disease Receives Multiple FDA Designations
The gene therapy candidate is designed to deliver the ASPA gene directly to the oligodendrocyte cells in the fluid surrounding the brain and spinal cord.
