Ultragenyx Receives Breakthrough Therapy Designation For GTX-102

(RTTNews) - Ultragenyx Pharmaceutical (RARE) has received Breakthrough Therapy Designation from the FDA for GTX-102 as a treatment for Angelman syndrome. The decision is based on preliminary clinical evidence including positive data from the Phase 1/2 study in 74 patients with a full maternal UBE3A gene deletion.

Eric Crombez, chief medical officer at Ultragenyx, said: "Based on the strength of the Phase 2 data and with strong support and interest from the Angelman syndrome community, our Phase 3 Aspire study is rapidly enrolling across our global sites."

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