BridgeBio spins gold out of an Alexion castoff, scoring an FDA approval in rare metabolism disorder
Now part of the fold at AstraZeneca, rare disease specialist Alexion doesn’t miss often when it comes to taking meds across the finish line. But in one special case, BridgeBio took an Alexion castoff on board — and now it’s turned the drug into a winner.
The FDA approved BridgeBio’s Nulibry (fosdenopterin), once known as ALXN1101, on Friday to treat molybdenum cofactor deficiency type A, an autosomal recessive, inborn error of metabolism caused by mutations in the molybdenum cofactor synthesis 1 gene (MOCS1).
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