BioMarin announces positive data from early stage trials of haemophilia A gene therapy

pharmafile | April 21, 2016 | News story | Research and Development BioMarin Pharmaceutical, Phase II, drug trial, haemophilia A gene therapy 

BioMarin Pharmaceutical (Nasdaq: BMRN) said early-stage trials for its haemophilia A gene therapy showed encouraging data.

Chief Medical Officer, Hank Fuchs, said, the therapy, called BMN 270, could potentially reduce or eliminate need for infusions of Factor VII, a protein that helps with clotting.

In the study, eight patients with severe haemophilia A received a single dose of BMN 270, with six patients treated at the highest dose. Five of the six high-dose patients experienced increases in factor VIII of more than 5%, with two achieving activity levels of over 50%, the company said.

BioMarin noted that all six high-dose patients saw the severity of their disorder improve from severe to moderate, mild or normal range in terms of factor levels based on World Federation of Hemophilia criteria.

“We’re encouraged by the consistency of the data from six to 12 months in both safety and efficacy, and plan to initiate a Phase 3 study by the end of the year,” Fuchs said, adding, “By addressing the root cause of achondroplasia with vosoritide treatment and normalizing annualized growth velocity in children with achondroplasia, we ultimately hope to improve the medical complications of disproportionate bone growth.”

BioMarin is planning to initiate Phase III trials by the end of 2016 in children with achondroplasia ages 5-14.  The company is also planning a separate Phase II study evaluating the effect of vosoritide in infants and toddlers. 

Vosoritide has orphan designation in both the US and Europe.  

Anjali Shukla

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