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BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the New England Journal of Medicine (NEJM) published an independently peer-reviewed article on up to three years of data from an ongoing Phase 1/2 study to evaluate safety and efficacy of investigational AAV gene therapy, valoctocogene roxaparvovec, for severe hemophilia A. This is the second article published by the NEJM on valoctocogene roxaparvovec.
The NEJM article, "Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A", demonstrated that a single infusion of valoctocogene roxaparvovec "resulted in sustained, clinically relevant benefit, as measured by a substantial reduction in annualized rates of bleeding events and complete cessation of prophylactic factor VIII use in all 13 participants who had received 4e13vg/kg or 6e13 vg/kg." Twelve of these participants also experienced a full resolution of target joints. Recurrent bleeding into the same joint results in a "target joint" and is defined as three bleeds into the same joint within a 6-month period. Bleeding into joints causes pain and ultimately results in impairment of the joint and permanent damage (hemophilic arthropathy).[i] Resolution of a target joint is when there have been less than two bleeds into the joint within a consecutive 12‐month period.
In addition to reporting safety and efficacy, the article contributes to a greater understanding of the variability observed in gene therapy studies and provides insights into mechanisms of DNA persistence and durability. The article noted that the most common adverse event was a transient elevation in alanine aminotransferase (ALT) levels with no indications of ongoing liver damage, and that no participants withdrew from the study. For additional safety data, see Safety section in press release.
"With each passing year, we come to appreciate further the transformation in patient lives that may be possible with gene therapy. As pioneers in the field, we are proud to sponsor research that is published in leading scientific journals so that we and the scientific community can learn together about how to characterize long-term benefits and ultimately optimize patient outcomes," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "We look forward to providing a four year update at a scientific congress in the middle of this year, during which we'll also provide a third year of follow up after lower levels of expression were achieved using a lower dose." ...
Published in the New England Journal of Medicine (January 2, 2020):
https://doi.org/10.1056/NEJMoa1908490
