Tara Ahmad

Tara Ahmad asked for clarification on the timeline for a final decision after resubmitting the UX111 BLA following an IRL. She also inquired about updated thoughts on DTX-401 (GSDIa) pricing and its potential launch trajectory.

Answer

Emil Kakkis, Chief Executive Officer and President, explained that after resubmitting the BLA with additional information, the FDA would take a couple of weeks to determine completeness, and then a PDUFA date would be set approximately six months after the original submission. For DTX-401, he expects strong, steady demand due to the urgent nature of GSDIa, but not an immediate steep launch like MPS IIIA. He mentioned a pricing range of $1 million-$2 million for GSDIa, compared to $2 million-$4 million for MPS IIIA.

Tara Ahmad asked for clarification on the timeline for UX111's review to final decision after resubmitting responses to the IRL. She also inquired about updated thoughts on DTX-401 (GSDIa) pricing and expectations for its launch trajectory (slow and steady versus steep).

Answer

CEO Emil Kakkis explained that after resubmitting the BLA with additional information, the FDA would take a couple of weeks to determine completeness, then set a PDUFA date approximately six months after the original submission. He noted that the time to compile and resubmit the documents is currently being diligently worked on. For DTX-401 (GSDIa), Kakkis expects strong, steady demand due to the urgent need (patients drinking starch every two hours), but anticipates a slower, steadier launch trajectory compared to MPS IIIA, which has an immediate need to prevent brain loss. He mentioned a pricing range of $1 million-$2 million for GSDIa, compared to $2 million-$4 million for MPS IIIA.