FDA Accepts Treatment of Molybdenum Cofactor Deficiency Type A

BridgeBio Pharma, Inc. and its affiliate Origin Biosciences announced the US Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for fosdenopterin, a cyclic pyranopterin monophosphate (cPMP) substrate replacement therapy, for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A. There is currently no approved treatment to treat type A MoCD, which can cause severe and irreversible nerve damage in infants and children.

The mission of BridgeBio Pharma is to find, develop, and deliver breakthrough medicines for genetic diseases to patients as quickly and safely as possible. This is BridgeBio's first NDA to be accepted, and the FDA has granted priority review qualification for this NDA. Previously, fosdenopterin has been granted Breakthrough Therapy Designation and Rare Pediatric Disease Designation in the US and may be eligible for a priority review voucher if approved. It received Orphan Drug Designation in the US and Europe. This is BridgeBio's first NDA acceptance.

MoCD Type A is an ultra-rare, autosomal recessive, inborn error of metabolism caused by disruption in molybdenum cofactor (MoCo) synthesis which is vital to prevent buildup of s-sulfocysteine, a neurotoxic metabolite of sulfite. Patients are often infants with severe encephalopathy and intractable seizures. The disease progresses rapidly and the infant mortality rate is high.

Those infants who survive the first few months are developmentally delayed and suffer from irreversible neurological damage, including brain atrophy with white matter necrosis, facial deformities, and spastic paraplegia. The clinical presentation that can be similar to hypoxic-ischemic encephalopathy (HIE) or other neonatal seizure disorders may lead to misdiagnosis and underdiagnosis. Immediate testing for elevated sulfite levels and S-sulfocysteine in the urine and very low serum uric acid may help with suspicion of MoCD.

Fosdenopterin is a pioneering cPMP hydrobromide dihydrate designed to treat type A molybdenum cofactor deficiency by replacing cPMP and allowing the two remaining MoCo synthesis steps (MoCo-dependent enzyme activation and sulfite removal) to continue.

Fosdenopterin is being developed for the treatment of patients with MoCD Type A. Currently, there are no approved therapies for the treatment of MoCD Type A, which results in severe and irreversible neurological injury with a median survival between 3 to 4 years.

Neil Kumar, Ph.D. CEO and founder of BridgeBio, said: "We want to thank the patients, families, scientists, physicians and all others involved who helped us reach this critical milestone. molybdenum cofactor deficiency Type A is a devastating disease with a median survival of fewer than four years and we are eager for our investigational therapy to be available to patients, who currently have no approved treatment options. BridgeBio exists to help as many patients as possible afflicted with genetic diseases, no matter how rare. We are grateful that the FDA has accepted our first NDA for priority review and we look forward to submitting our second NDA later this year for infigratinib for second-line treatment of cholangiocarcinoma."

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