The U.S. Food and Drug Administration (FDA) is on a mission to recuperate from the review lag seen through the early phases of COVID-19 pandemic and has provided special designations for two treatments that could improve the lives of millions with recurrent glioblastoma or hemophilia diagnoses.
The Phase III Xtend-1 study met the primary and secondary endpoints, revealing the efficacy of BIV001. The primary endpoint focused on the prevention of bleeds over the course of 52 weeks, while the secondary endpoint examined the treatment’s ability to prevent bleeds in comparison to prophylactic factor VIII replacement therapy. Minor treatment-emergent adverse events were recorded including back pain, headaches and arthralgia. Adverse incidents did not occur too often to the point where there were serious concerns. With Breakthrough Designation, the FDA acknowledges the potential for BIVV001 to improve the lives of those with life-threatening hemophilia A, in comparison to treatments that are already available. The designation also manifests the administration’s intent to accelerate the review of data related to BIVV001. In 2017, BIVV001 was granted Orphan Drug Designation by the FDA. Following suit, the European Union (EU) also awarded Orphan Drug Designation.
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