BridgeBio and Sentynl Therapeutics’ Nulibry Gets Greenlighted In Europe

BridgeBio and Sentynl Therapeutics (a subsidiary of Zydus Cadila Pharmaceuticals) have obtained approval from the European Commission (EC) to market Nulibry (fosdenopterin) for Injection, a treatment for molybdenum cofactor deficiency (MoCD) Type A. 

Nulibry was first approved by the US FDA in 2021, and recently obtained approval in Israel. 

In March 2022, Sentynl bought the global rights to Nulibry, additionally taking over its development and commercialization in the US. Under the terms of the deal, BridgeBio is eligible for milestone payments from Sentynl and tiered royalties according to sales of Nulibry. 

Related Article: AVROBIO’s First-of-its-Kind Gene Therapy Receives Rare Pediatric Disease Designation

Substrate Replacement For An Ultra-Rare Disease

Affecting less than 150 patients globally, MoCD Type A is an ultra-rare pediatric disease. Mutations in the molybdenum cofactor synthesis 1 gene leads to deficiency in molybdenum cofactor production and lack of related molybdenum-dependent enzyme activity.  

MoCD Type A is most commonly presented with seizures, feeding difficulties, and brain damage. Patients who survive beyond infancy suffer from progressive brain damage, leading to severe difficulty in making coordinated movements or communicating with their environment. 

Nulibry is a substrate replacement therapy that provides cPMP, which is converted ultimately to molybdenum cofactor to restore the activity of molybdenum-dependent enzymes. 

The EC approval follows a positive opinion granted by CHMP in July, which was based on results from three clinical trials showing the efficacy of Nulibry in the treatments of MoCD Type A. The studies showed that patients treated with Nulibry had a 7.1 times lower risk of death than patients in the untreated control group, with 86% of Nulibry-treated patients surviving at three years compared to 52% of the control group. 

Related posts:

Brukinsa Receives EU Approval for the Treatment of Rare Lymphoma AstraZeneca’s Rare Disease Treatment Wins Third Approval in European Union Albireo Signals To Apply For FDA Approval After Trial Win Agios Pharmaceuticals’ Lead Candidate Mitapivat Achieves Primary Endpoint in ACTIVATE Trial

Author

Joy Lin

Without knowledge, there is no imagination. Without imagination, knowledge is meaningless