Cipaglucosidase Alfa, Miglustat Under Review for Pompe Disease
AT-GAA consists of cipaglucosidase alfa administered in conjunction with miglustat.
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.
Wegovy Gains Approval for Treating Obesity in Adolescents
Wegovy contains semaglutide, a glucagon-like peptide-1 (GLP-1) receptor agonist.
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.
Recall of Sodium Bicarbonate Injection Due to Vial Breakage Expanded
The recalled product was distributed between October 26, 2021 and August 10, 2022.
Oxlumo Indication Expanded to Include Patients With Advanced PH1
The approval was based on data from the phase 3 ILLUMINATE-C study that included 21 dialysis-independent and -dependent patients with PH1.
Leniolisib Gets Priority Review for Activated Phosphoinositide 3-Kinase Delta Syndrome
Leniolisib is a small molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K with immunomodulating and potentially antineoplastic activities.
FDA to Review Velmanase Alfa for Alpha-Mannosidosis
Velmanase alfa is a recombinant form of human alpha-mannosidase intended to provide or supplement natural alpha-mannosidase.
Orkambi Indication Expanded to Include More Children With Cystic Fibrosis
The safety profile of Orkambi was found to be similar in patients aged 1 to less than 2 years compared with those 2 years of age and older.
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