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.
Metabolic Disorders
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.
FDA Cites Deficiencies in Veverimer NDA for Metabolic Acidosis in CKD
Veverimer is a non-absorbed, orally-administered polymer designed to treat metabolic acidosis in patients with CKD.
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.
CDC Updates List of Who Is at Highest Risk for COVID-19
Agency now says risk among adults increases steadily with age, and is not limited to those over 65.
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.
Antiobesity Meds May Curb Weight Regain After RYGB
Statistical models suggest phentermine, topiramate, combination can slow, prevent weight regain
Lonafarnib Gets Priority Review for Progeria, Progeroid Laminopathies
The FDA has accepted for Priority Review the New Drug Application (NDA) for lonafarnib (Zokinvy™; Eiger BioPharmaceuticals) for the treatment of progeria and progeroid laminopathies, rare, genetic conditions of accelerated aging in children.
Factors ID’d for Positive SARS-CoV-2 Test Result in Primary Care
Sociodemographic risk factors include male sex, deprivation, population density, ethnicity, obesity.
Setmelanotide Granted Priority Review for Rare Genetic Obesity Disorders
The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for setmelanotide for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity.
