The US Food and Drug Administration (FDA) approved Nulibry (fosdenopterin) for injection to reduce the risk of death due to molybdenum cofactor deficiency Type A, a rare, genetic, metabolic disorder that typically presents in the first few days of life, causing intractable seizures, brain injury and death.

The Administration approved Origin Biosciences' new drug Nulibry to reduce the risk of death due to type A molybdenum cofactor deficiency (MoCD). Hylton V. Joffe, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine (ORPURM), said: "This approval is the first time the FDA has approved a treatment for this devastating disease."

Patients with molybdenum cofactor deficiency Type A experience severe and rapidly progressive neurologic damage including intractable seizures, feeding difficulties and muscle weakness from the accumulation of toxic sulfite metabolites in the central nervous system. Most patients die in early childhood from infections. Before today's approval, the only treatment options included supportive care and therapies directed towards the complications arising from the disease.

Nulibry is a "first-in-class" cPMP hydrobromide dihydrate, which has been granted breakthrough therapy designation and rare pediatric disease designation. The FDA press release stated that out of 13 patients treated with Nulibry, Nulibry was proven to be effective in treating type A molybdenum cofactor deficiency, while 18 matched untreated patients.

The data showed that the 3-year survival rate of Nulibry-treated patients was 84%, while that of untreated patients was 55%. The most common side effects include complications related to intravenous fluids, fever, respiratory infections, vomiting, gastroenteritis zhuzhou cemented carbide and diarrhea.

The FDA noted that phototoxicity was observed in animals, so patients treated with fosdenopterin should avoid exposure to sunlight and wear sunscreen, protective clothing, and sunglasses when exposed to the sun.

It is reported that Origin Biosciences is a subsidiary of BridgeBio Pharma. BridgeBio was established in 2015 to identify and advance translational drugs to treat Mendelian diseases and cancers with clear genetic drivers. In 2019, BridgeBio has been listed on the Nasdaq exchange in the United States and won the crown of the year's largest biotechnology IPO.

Origin Biosciences is the biotechnology company responsible for the development and commercialization of molybdenum cofactor deficiency Type A drugs. The approved Tungsten Carbide Round Rod Nulibry is the first new drug that BridgeBio submitted.

The FDA granted this application priority review and breakthrough therapy designations. Fosdenopterin for molybdenum cofactor deficiency Type A also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. Origin Biosciences is also receiving a rare pediatric disease priority review voucher under a program intended to encourage the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. A voucher can be redeemed by a sponsor at a later date to receive a priority review of a subsequent marketing application for a different product.

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