BridgeBio Pharma and Affiliate Origin Biosciences Announce FDA Approval of NULIBRY (fosdenopterin), the First and Only Approved Therapy to Reduce the Risk of Mortality in Patients with MoCD Type A

Molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening genetic disorder that progresses rapidly, results in severe and largely irreversible neurological injury, and has a high infant mortality rate; median overall survival age is about four years

Clinical trials with NULIBRY or recombinant cPMP showed a meaningful increase in overall survival compared to a natural history study

NULIBRY is BridgeBio’s first FDA-approved therapeutic

PALO ALTO, Calif., Feb. 28, 2021 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio) and affiliate Origin Biosciences, Inc. (Origin) today announced the U.S. Food and Drug Administration (FDA) has approved NULIBRY (fosdenopterin) for Injection as the first therapy to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A. This is the first therapy of its kind. The novel therapy was developed based on BridgeBio’s commitment to developing a treatment for MoCD Type A in collaboration with the experts and families in the MoCD Type A community. The announcement was made on Rare Disease Day, which aims to raise awareness about the impact of rare diseases on patients.

MoCD Type A is an ultra-rare and progressive condition, known to impact less than 150 patients globally with a median survival of four years. MoCD Type A presents shortly after birth, often with severe encephalopathy and intractable seizures. NULIBRY is a first-in-class approved cPMP substrate replacement therapy.

“The FDA’s approval of NULIBRY means that patients with MoCD Type A and their families have an approved therapy for the first time. It also reflects our belief that every life matters and that no disease is too rare to address. As is often true in rare disease drug development, this was a community effort in which we were able to play a part – we’d like to thank the patients, caregivers, physicians, scientists, and advocates who played an essential role in achieving this important milestone,” said BridgeBio founder and CEO Neil Kumar, Ph.D.

The efficacy of NULIBRY for the treatment of patients with MoCD Type A was established based on data from three clinical trials compared to data from a natural history study. In these studies, NULIBRY or recombinant cPMP (rcPMP; same active moiety and biologic activity as NULIBRY) reduced the risk of death by 82% compared to the untreated, genotype-matched, historical control group in the natural history study (HR=0.18, 95% CI 0.04, 0.72). At three years on study, the probability of survival in NULIBRY or rcPMP-treated patients (n=13) was 84% (95% CI 49%,96%) compared to 55% (95% CI 30%,74%) for untreated genotype-matched patients in the historical control group (n=18) at three years (Figure 1). In addition to the survival analysis, treatment with NULIBRY led to a reduction of urine concentrations of S-sulfocysteine (SSC), a toxic substance that leads to neurological damage, in patients with MoCD Type A, and the reduction was sustained with long-term treatment over 48 months.