In a press release, the independent specialty pharmacy Biologics by McKesson recently announced that it has been selected to the be the sole provider of fosdenopterin (marketed as NULIBRY) by the biotechnology company Origin Biosciences, Inc., which is affiliated with BridgeBio Pharma, Inc. This is the first-ever US Food and Drug administration (FDA) approved treatment for the rare disease molybdenum cofactory deficiency type A (MoCD).
About Molybdenum Cofactor Deficiency (MoCD)
Molybdenum cofactor deficiency (MoCD) is a very rare disease which is characterized by the absence of molybdopterin. This consequently means that its molybdenum complex, or molybdenum factor, is also absent. These conditions lead to neurological damage as a result of a buildup of excess sulfite. In many cases, affected people die in only a few months after being born. There are different types that are linked to different genetic mutations; in the type A form, the MOCS1 gene is affected. Less than 1,000 cases have been recorded. Symptoms include seizures very early in life, and most patients present with very low uric acid in the blood along with high levels of uric acid, sulfite, and xanthine in the urine. There are also characteristic MRI images associated with MoCD. Fosdenopterin (NULIBRY) is the only approved treatment for the disease, which can reduce the risk of death. To learn more about MoCD, click here.
About Fosdenopterin (NULIBRY)
NULIBRY, delivered by injection, is a type of substrate replacement therapy that replaces cyclic pyranopterin monophosphate (cPMP), which is then converted into molybdopterin. This represents the first disease-altering therapy for MoCD, as previous treatment was only supportive and typically involved anti-seizure medications. Biologics by McKesson is prepared for a rapid response in transporting this therapy, as the timing of treatment can be critical when MoCD type A has been diagnosed.
NULIBRY also marks the first drug approval for BridgeBio, which focuses on developing therapies for cancers with defined genetic drivers and other genetic diseases.
Biologics by McKesson will play a critical role in getting people living with this very rare illness the vital treatment that they need to increase their chances of survival.
