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.
The FDA has accepted for Priority Review the New Drug Application for lumasiran for the treatment of primary hyperoxaluria type 1.
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.
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.
Horizon Therapeutics announced the availability of Procysbi (cysteamine bitartrate) delayed-release oral granules in tear-open packets for the treatment of nephropathic cystinosis in patients aged 1 year and older.
The FDA has granted Fast Track designation to vutrisiran (Alnylam) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.
The FDA has approved Koselugo (selumetinib; AstraZeneca) for the treatment of patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas.
The FDA has granted Fast Track designation to MRT5005 (Translate Bio), an inhaled messenger RNA (mRNA) therapeutic being investigated for the treatment of cystic fibrosis.
This week on the pod we cover a first-in-class cholesterol lowering therapy; a new indication for Trulicity; Macrolide prescriptions in the first trimester and their relation to fetal malformations; a new fragile X diagnostic test; and a migraine prevention IV treatment.
The Food and Drug Administration (FDA) has granted Fast Track designation to LYS-SAF302 (Lysogene), an investigational gene therapy for the treatment of mucopolysaccharidosis Type IIIA (MPS IIIA).
