US arm of Zydus Lifesciences, Sentynl Therapeutics and BridgeBio Pharma has recently announced the European Commission (EC) has granted marketing authorisation for fosdenopterin injection marketed under the name Nulibry as the first therapy for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A.

In a joint statement, the companies said that the EC  decision to grant marketing authorisation is based on the efficacy and safety data collected compared to data from a natural history study.

Fosdenopterin was the first cPMP substrate replacement therapy to get approval from the United States Food and Drug Administration in 2021 to reduce the risk of death in patients with MoCD Type A, and the EC decision will make it the first and only approved treatment in the European Union for the disease, the joint statement added.

"The European Commission's approval of Nulibry is an exciting step in delivering this therapy to all children suffering with MoCD Type A worldwide, and it bolsters our belief at BridgeBio that no disease is too rare to address,”  BridgeBio founder and CEO, Dr Neil Kumar said.

“We are grateful that the European Commission sees the value of this treatment, and to the patients, caregivers, physicians, scientists, and advocates whose efforts made this possible," he added.

Commenting  on the development, the CEO of Sentynl, Matt Heck said, “This is a major milestone for those patients living with MoCD Type A in Europe.”

“Marketing authorisation is an important step in providing access to Nulibry and creating awareness of MoCD Type A as many patients are often missed,” Mr Heck added.

MoCD Type A is an extremely rare and progressive condition, which impacts less than 150 patients globally with a median survival of four years.

This devastating metabolic disease causes severe developmental delay and death is attributed to a gene abnormality, which causes a toxic sulfite to collect in the brain.