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FDA OKs Nitisinone for Ultra-Rare Black Urine Disease

The FDA approved nitisinone (Harliku) for the reduction of urine homogentisic acid (HGA) in adults with alkaptonuria, Cycle Pharmaceuticals announced on Thursday. Alkaptonuria is an ultra-rare hereditary disorder of tyrosine and phenylalanine catabolism, and is commonly known as black urine disease due to the noticeable buildup of HGA. A slow-progressing disease, alkaptonuria can cause tissue […]

FDA Approves Nipocalimab for Adult and Pediatric Myasthenia Gravis

The FDA approved nipocalimab (Imaavy) injection to treat adults and children ages 12 and older with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) or anti-muscle-specific kinase (MuSK) antibody positive, Johnson & Johnson announced Wednesday. Nipocalimab is designed to block the immunoglobulin G (IgG) binding site on the neonatal Fc receptor (FcRn) to reduce […]

FDA OKs Injectable Antibiotic for Three Different Uses

The FDA approved the injectable drug ceftobiprole medocaril sodium (Zevtera) to treat adults with Staphylococcus aureus bacteremia (SAB), the agency announced Wednesday. The antibiotic prodrug is indicated for people with right-sided infective endocarditis, those with acute bacterial skin and skin structure infections (ABSSSI), and adult and pediatric patients 3 months to less than 18 years […]

Novel Drug Approved for All Duchenne Muscular Dystrophy Variants

The FDA approved oral givinostat (Duvyzat) to treat Duchenne muscular dystrophy (DMD) in patients ages 6 years and older, the agency announced Thursday. Givinostat is a histone deacetylase (HDAC) inhibitor that targets pathogenic processes to reduce inflammation and loss of muscle. It’s the first nonsteroidal drug approved for patients with all genetic variants of DMD, […]

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