BRIEF—MRHA approves rare disease drug Nulibry

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has today approved the medicine Nulibry (fosdenopterin) to treat the genetic disease Molybdenum Cofactor Deficiency (MoCD) Type A.

The drug was developed by US biotech BridgeBio Pharma and Sentynl Therapeutics, a USA-based subsidiary of India’s Zydus Lifesciences.

MoCD is a rare genetic disorder in which the body is not able to produce cyclic pyranopterin monophosphate (cPMP).

In coming to its decision, the MHRA has relied on a European Commission (EC) decision (EMEA/H/C/005378), in accordance with the advice from the Committee for Medicinal Products for Human Use (CHMP). This is known as the EC Decision Reliance Procedure.