Kalydeco Approval Expanded to Include Younger Cystic Fibrosis Patients
The approval was based on data from a phase 3 open-label safety study that included a cohort of 6 children 4 months to <6 months of age.
Inborn Errors Of Metabolism
Transdermal Cannabidiol Gets Orphan Drug Status for 22q11.2 Deletion Syndrome
The efficacy and safety of Zygel is being evaluated in the INSPIRE study.
FDA Approves Once Daily Dosing Regimen for Nityr
The total daily dose may now be administered once daily in patients aged 5 years and older who have undetectable serum and urine succinylacetone concentrations after a minimum of 4 weeks on a stable dosage.
Casimersen Gets Priority Review for Duchenne Muscular Dystrophy
Casimersen is a phosphorodiamidate morpholino oligomer that works by binding to exon 45 of dystrophin pre-mRNA.
Vosoritide NDA for Achondroplasia Submitted for Review
Vosoritide is an investigational analogue of C-type natriuretic peptide.
Viltepso Now Available for Duchenne Muscular Dystrophy
Viltepso is available through specialty distributors and specialty pharmacy providers.
FDA Approves Viltepso for Duchenne Muscular Dystrophy
Viltolarsen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomers subclass.
Gene Transfer Therapy Fast-Tracked for Duchenne Muscular Dystrophy
The FDA has granted Fast Track designation to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin; Sarepta Therapeutics) for the treatment of Duchenne muscular dystrophy (DMD).
FDA Approves First Treatment for Long-Chain Fatty Acid Oxidation Disorders
The FDA has approved Dojolvi™ (triheptanoin; Ultragenyx) as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders.
Lumasiran Granted Priority Review for Primary Hyperoxaluria Type 1
The FDA has accepted for Priority Review the New Drug Application for lumasiran for the treatment of primary hyperoxaluria type 1.
