Endocrinology and Metabolic Disorders

FDA approves transthyretin amyloidosis therapy of Akcea, Ionis Patients with an orphan, hereditary disease that is transthyretin amyloidosis with a second new treatment option are available after the Food and Drug Administration approved Akcea Therapeutics and Ionis Pharmaceuticals' Tegsedi.  As Tegsedi is approved, Akcea squarely faces competition with Alnylam Pharmaceuticals, which got an approval from the FDA formerly for its drug Onpattro. Both therapies target RNA, degrading the genetic instructions that are responsible for the production of a misfolded protein, which builds up in the organs of patients with the disease. Prenatal inflammation is a probable answer ...