BridgeBio Pharma Inc. (BBIO) and Sentynl Therapeutics Inc., on Tuesday, announced that the European Commission has granted marketing authorization for NULIBRY for the treatment of patients with Molybdenum Cofactor Deficiency Type A.
Molybdenum Cofactor Deficiency Type A (MoCD Type A) is an ultra-rare disease caused by mutations in the molybdenum cofactor synthesis 1 gene. This disease is characterized by severe and largely irreversible neurological injury, and has a high infant mortality rate, with the median overall survival age being about four years.
The marketing authorization follows the positive opinion granted by the European Committee for Medicinal Products for Human Use in July 2022, which was supported by data from three clinical trials that demonstrated the efficacy of NULIBRY for the treatment of patients with MoCD Type A compared to data from a natural history study.
These studies showed that in the genotype-matched analysis, patients treated with NULIBRY had a 7.1 times lower risk of death than their control counterparts, with 86% of NULIBRY-treated patients surviving at three years compared to 52% of the genotype-matched control group.
NULIBRY was approved in the U.S. by the FDA for the above indication last February and by the Israeli Ministry of Health in July of this year.
Sentynl acquired the global rights to NULIBRY from BridgeBio in March of this year and is responsible for the ongoing development and commercialization of NULIBRY in the United States and developing, manufacturing, and commercializing the drug globally.
The two companies share development responsibilities through the approval of the marketing authorization application under accelerated assessment with the European Medicines Agency (EMA) and through approval of NULIBRY’s regulatory submission with the Israeli Ministry of Health.
BBIO has traded in a range of $4.98 to $53.57 in the last 52 weeks. The stock is currently trading at $11.32, up 0.35%.
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