05:54 PM EDT, 07/17/2024 (MT Newswires) -- Ultragenyx Pharmaceutical ( RARE ) reported late Wednesday the "successful" completion of an end-of-phase 2 meeting with the US Food and Drug Administration, supporting its late-stage study plans for GTX-102 for Angelman syndrome.

"FDA alignment on our Phase 3 study design for GTX-102 allows for rapid initiation of a global double-blind sham-controlled pivotal study by the end of this year," said chief medical officer Eric Crombez.

"In addition to this pivotal study in patients with a full UBE3A gene deletion, we are working to initiate a study to evaluate GTX-102 in patients with other mutations," Crombez said. "This will allow for the potential treatment of more children and adults impacted by this devastating disease."

The stock was up about 1.4% in extended trading.

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