logo
Share SHARE
FONT-SIZE Plus   Neg

BioMarin Reports Encouraging Data In Hemophilia A Gene Therapy Program

BioMarin Pharmaceutical Inc. (BMRN) announced encouraging preliminary data from an ongoing Phase 1/2 clinical trial with BMN 270, an investigational gene therapy treatment for hemophilia A.

The company said that a total of eight patients with severe hemophilia A received a single dose of BMN 270, six of whom have been treated at the highest dose of 6 x 1013 vg/kg, and to date, post-treatment follow-up ranges from five to 16 weeks.

Patients with hemophilia A are not able to produce enough functional Factor VIII to prevent bleeding, and are currently treated with prophylactic or on-demand infusions of plasma-derived or recombinant Factor VIII.

At last observation, patients at the highest dose experienced increasing Factor VIII activity levels ranging between 4 and 60 percent, with five of six patients treated at the high dose showing factor VIII levels above 5 percent and two of six showing increasing levels of factor VIII at over 50 percent.

All the high dose patients improved from severe to either moderate, mild or normal range in terms of factor levels based on World Federation of Hemophilia criteria.

"We are encouraged by this early data on BMN 270 and the trend we are seeing in increasing Factor VIII levels over time. BMN 270 could have the potential to reduce and possibly eliminate the need for infusions of Factor VIII," said Hank Fuchs, Chief Medical Officer at BioMarin.

BioMarin plans to discuss these findings with UK regulatory authorities prior to dosing the remaining patients.

by RTT Staff Writer

For comments and feedback: editorial@rttnews.com

Business News

Quick Facts

Editors Pick
10 Worst Places To Raise A Family Whether you are a young couple building a nest or seasoned parents looking for a change of scenery, there are number of places around the country that should be avoided, according to personal finance website WalletHub. They looked at mainly five categories for... A passenger has filed a lawsuit against Canadian budget airline Sunwing Airlines for serving him cheap sparkling wine instead of champagne on board a flight, according to media reports. The Canadian passenger, Daniel MacDuff, reportedly boarded his Sunwing flight to Cuba in February, on a flight package that advertised a complimentary on-board champagne toast. Senate Health Committee Chairman Lamar Alexander, R-Tenn., and ranking member Patty Murray, D-Wash., announced Thursday that twenty-two other Senators are cosponsoring their bipartisan plan to provide short-term stability to Obamacare. The bipartisan bill includes a two-year extension of the cost-sharing...
comments powered by Disqus
Follow RTT