RARE Daily

FDA Grants BridgeBio and Its Affiliate Origin Biosciences Approval for MoCD Therapy

March 1, 2021

Rare Daily Staff

The U.S. Food and Drug Administration granted approval to BridgeBio Pharma and its affiliate Origin Biosciences’ Nulibry, the first therapy to reduce the risk of mortality in patients with the ultra-rare metabolic condition molybdenum cofactor deficiency type A.

Molybdenum cofactor deficiency (MoCD) type A is a progressive condition that affects less than 150 patients globally. The median survival is four years. The condition presents shortly after birth, often with severe encephalopathy and intractable seizures.

Nulibry is a first-in-class cPMP substrate replacement therapy that provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase, an enzyme that reduces levels of neurotoxic sulfites.

Clinical trials have demonstrated that patients treated with Nulibry or recombinant cPMP had an improvement in overall survival compared to the untreated, genotype-matched, historical control group.

“The FDA’s approval of Nulibry means that patients with MoCD Type A and their families have an approved therapy for the first time,” said Neil Kumar, founder and CEO of BridgeBio. “It also reflects our belief that every life matters and that no disease is too rare to address.”

Animal studies have identified that Nulibry has phototoxic potential. In the clinical trials, the most common adverse reactions reported in two or more Nulibry-treated patients with MoCD type A were catheter-related complications (89 percent), fever (78 percent), viral infection (56 percent), pneumonia (44 percent), otitis media (ear infection; 44 percent), vomiting (44 percent) and cough/sneezing (44 percent). Adverse reactions for the rcPMP-treated patients were similar to the Nulibry-treated patients.

“Today’s approval represents new hope for patients and families affected with MoCD Type A,” said Donald Basel, section chief and associate professor of pediatrics at Children’s Wisconsin. “I am encouraged by the clinical data showing that Nulibry not only lowers the levels of toxic SSC, but importantly extends the lives of patients who previously had only a three- to four-year median survival.”

Nulibry was reviewed under Priority Review and received Orphan Drug, Breakthrough Therapy, and Rare Pediatric Disease designations from the FDA. With this approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher to Origin.

Photo: Neil Kumar, founder and president of BridgeBio

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