Review of 2021 FDA-Approved Drugs for Genetic Conditions

The FDA approved five new drugs in 2021 for use for genetic conditions, including molybdenum cofactor deficiency, Von Hippel-Lindau (VHL) disease, achondroplasia, and progressive familial intrahepatic cholestasis (PFIC). This article and slide deck reviews each in detail, including their mechanisms of action, originators, key clinical data, disease context, target rationale, and more…

 this content is exclusive to
Premium members

Read it now by upgrading your membership.

RECENT ARTICLES

DRUG DISCOVERY RESOURCES

JOIN SUBSCRIBERS from

  • Johnson & Johnson
  • Roche
  • Pfizer
  • Novartis
  • Bayer
  • Merck
  • Sanofi
  • GlaxoSmithKline
  • AbbVie
  • Eli Lilly
  • Boehringer Ingelheim
  • Amgen
  • Bristol-Myers Squibb
  • Gilead
  • AstraZeneca
  • Teva
  • Novo Nordisk
  • Merck KGaA
  • Takeda
  • Celgene (BMS)
  • Biogen
  • Astellas
  • Daiichi Sankyo
  • Regeneron
  • UCB
  • Eisai
  • Servier
  • Kyowa Kirin
  • Shionogi
  • Vertex
  • Lundbeck
  • Taisho
  • Ono
  • Seagen
  • Incyte
  • LEO Pharma
  • Janssen
  • Galapagos
  • Idorsia
  • Astex
  • Arvinas
  • Kymera
  • Relay
  • Schrodinger
  • Mirati
  • Revolution
  • Nimbus
  • Plexxikon
  • Forma
  • Arcus
  • RA Capital
  • ARCH Venture Partners
  • MPM Capital
  • Orbimed
  • The Column Group
  • 5AM Ventures
  • Versant Ventures
  • Deerfield
  • Goldman Sachs
  • Citi
  • Piper Sandler
  • Softbank
  • …and hundreds of others