The FDA grants Rare Pediatric Disease designation to Axovant Gene Therapies’ (NASDAQ:AXGT) AXO-AAV-GM1, an AAV9-based gene remedy for the GM1 gangliosidosis, a uncommon inherited dysfunction characterised by the progressive destruction of nerve cells (neurons) within the mind and spinal wire.
Uncommon Pediatric Illness designation supplies for the issuance of a precedence evaluation voucher following FDA approval. The voucher can be utilized for accelerated approval of a future software or it may be offered to a 3rd celebration.
A Phase 1/2 clinical trial is in course of.
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