Vertex’s Inaxaplin (VX-147) Receives the US FDA’s Breakthrough Therapy Designation & EMA’s PRIME Designation for APOL1-Mediated FSGS & AMKD
- Inaxaplin has received BTD from the US FDA for APOL1-mediated FSGS & PRIME designation from EMA for APOL1-mediated CKD
- The BTD was based on the P-II study of Inaxaplin in patients with APOL1-mediated FSGS which showed a clinical mean reduction in protein in the urine, a sign of kidney damage in 47.6% @13wks., was well-tolerated with no serious AEs
- The PRIME designation was based on clinical PoC data from the P-II study of Inaxaplin in APOL1-mediated FSGS. Additionally, the P-II/III adaptive trial is still ongoing, and it will first analyze two doses of Inaxaplin for 12wks. & then examine the efficacy and safety of the single dose in the P-III section of the study
Ref: Vertex | Image:Vertex
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