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BioWorld - Wednesday, March 3, 2021

Endocrine/Metabolic

Home » Topics » Disease categories and therapies » Endocrine/Metabolic
  • Obesity weight
    March 2, 2021
    By Anette Breindl

    Gene variants can increase fat, decrease cardiometabolic risk

    In the public mind, fat and unhealthy are more or less synonymous. But reality is more complicated, as reality often is. Even among individuals with a BMI of 30 or higher, somewhere between 15% and 45% have metabolism typical of much lower-weight individuals.
  • March 1, 2021
    By Michael Fitzhugh

    Bridgebio's Origin secures FDA approval for MoCD type A therapy

    Molybdenum cofactor deficiency (MoCD) type A, an ultra-rare metabolic disorder causing intractable seizures, brain injury and death, now has a world-first treatment in Nulibry (fosdenopterin), a new I.V. therapy developed by Bridgebio Pharma Inc. subsidiary Origin Biosciences Inc. The agency's priority review, supported by its orphan, breakthrough and rare pediatric disease programs, also yielded a priority review voucher (PRV) for Origin.
  • Feb. 26, 2021
    By Michael Fitzhugh

    Tricida's veverimer troubles continue with appeal denied

    A failure of Tricida Inc.'s most recent efforts to overcome FDA objections to an NDA for the company's sole candidate, veverimer, for treating metabolic acidosis, sent company shares (NASDAQ:TCDA) 30.6% lower to $5.11 on Feb. 26. The complex story appears focused now on the agency's desire for additional and more reliable data to support a potential approval. Tricida President and CEO Gerrit Klaerner on Thursday suggested the ongoing renal outcomes study, Valor-CKD, might provide it.
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Authors

  • Randy Osborne
  • Cormac Sheridan
  • Michael Fitzhugh
  • Brian Orelli
  • Joan Tur
  • Mari Serebrov
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