NULIBRY is the first and only treatment in Great Britain for patients with molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening genetic disorder that often progresses rapidly in infants with a median overall survival age of about four years
The Medicines and Healthcare products Regulatory Agency’s (MHRA) decision is based on efficacy and safety data collected to date.
MoCD Type A is known to impact fewer than 150 patients globally with a median survival age of four years.
NULIBRY is a first-in-class synthetic cPMP substrate replacement therapy that was approved by the U.S. Food and Drug Administration (FDA) in 2021 to reduce the risk of mortality in patients with MoCD Type A. Following this decision by the MHRA, NULIBRY is the first and only approved therapy in GB for MoCD Type A.
https://sentynl.com/news/sentynl-therapeutics-receives-mhra-authorization-of-nulibry-fosdenopterin-for-treatment-of-mocd-type-a-in-great-britain/
