January 2023 Recap: Drug Pipeline Updates
The table provided is a review of notable updates that occurred in January 2023 for investigational products in development.
Diagnosis & Disease Information
Olezarsen Fast Tracked for Familial Chylomicronemia Syndrome
Olezarsen is an investigational LICA antisense medication designed to inhibit the production of apoC-III for patients at risk of disease due to elevated triglyceride levels.
Antibody Oligonucleotide Conjugate Fast Tracked for Facioscapulohumeral Muscular Dystrophy
AOC 1020 is designed to reduce the expression of DUX4 mRNA and DUX4 protein in muscles.
December 2022 Recap: Drug Pipeline Updates
The table provided is a review of notable updates that occurred in December 2022 for investigational products in development.
Vamorolone Under Review for Duchenne Muscular Dystrophy
Vamorolone is a first-in-class dissociative steroidal drug that retains the anti-inflammatory activity of corticosteroids but with a novel mechanism of action.
Olpruva Approved for Patients With Urea Cycle Disorders
The application was approved through the FDA’s 505(b)(2) regulatory pathway.
Bitopertin Granted Orphan Drug Status for Erythropoietic Protoporphyria
Bitopertin is an investigational oral, selective inhibitor of glycine transporter 1 designed to modulate heme biosynthesis by limiting glycine uptake.
FDA Accepts Resubmitted BLA for Pegunigalsidase Alfa for Fabry Disease
Pegunigalsidase alfa is a long-acting recombinant, PEGylated, cross-linked α-galactosidase-A investigational product candidate.
Gene Therapy Gets Priority Review for Duchenne Muscular Dystrophy
SRP-9001 is an investigational gene transfer therapy designed to deliver a shortened, functional component of dystrophin to muscle tissue.
October 2022 Recap: Drug Pipeline Updates
The table provided is a review of notable updates that occurred in October 2022 for investigational products in development.
