Posts in category: News

FDA Approves Xphozah (tenapanor) to Reduce Serum Phosphorus in Adults with Chronic Kidney Disease

Ardelyx, Inc., a biopharmaceutical company, has announced that the U.S. Food and

Drug Administration (FDA) has approved Xphozah (tenapanor), the first and only phosphate absorption inhibitor, indicated to reduce serum phosphorus in adults with chronic kidney disease (CKD) on dialysis as add-on therapy in patients who have an inadequate response to phosphate binders or who are intolerant of any dose of phosphate binder therapy.

Xphozah, discovered and developed by Ardelyx, is a first-in-class, phosphate absorption inhibitor with a differentiated mechanism of action that acts locally in the gut to inhibit the sodium hydrogen exchanger 3 (NHE3), thereby reducing phosphate absorption through the paracellular pathway, the primary pathway of phosphate absorption. Xphozah is a single tablet, taken twice daily. Diarrhea was the most common side effect experienced by patients taking Xphozah in clinical trials.

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FDA Approves Zilbrysq (zilucoplan) for the Treatment of Adults with Generalized Myasthenia Gravis

UCB, a global biopharmaceutical company, today announced that Zilbrysq® (zilucoplan) has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody-positive.

Zilbrysq is the first once-daily subcutaneous, targeted peptide inhibitor of complement component 5 (C5 inhibitor). It is the only once-daily gMG target therapy for self-administration by adult patients with antiAChR antibody positive gMG.

gMG is a rare, chronic, heterogeneous, unpredictable autoimmune disease characterized by dysfunction and damage at the neuromuscular junction (NMJ).

Several factors are understood to be drivers of gMG disease pathology, including the complement cascade, immune cells, and pathogenic Immunoglobulin G (IgG) autoantibodies.

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FDA Approves Daybue (trofinetide) for the Treatment of Rett Syndrome

Acadia Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has approved Daybue (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. Daybue is the first and only drug approved for the treatment of Rett syndrome.

Rett syndrome is a complex, rare, neurodevelopmental disorder typically caused by a genetic mutation on the MECP2 gene. It is characterized by a period of normal development until six to 18 months of age, followed by significant developmental regression with loss of acquired communication skills and purposeful hand use. Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities. Rett syndrome is believed to affect 6,000 to 9,000 patients in the U.S.,with a diagnosed population of approximately 4,500 U.S. patients. Daybue is not recommended for patients with moderate or severe renal impairment.

Daybue™ is expected to be available in the U.S. by the end of April, 2023.

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