Type A Mtg To Clarify Resubmission, With CMC Resolvable & No Clinical Concerns

研报英文原文证据摘录

Type A Mtg To Clarify Resubmission, With CMC Resolvable & No Clinical Concerns

a wide range of estimates,

there appears to be 42.5-68K aSAH patients each year who reach the hospital for intervention.

All patients receive nimodipine while in the hospital; however, as an on-target vasodilator aSAH

patients are at risk for clinically significant decreases in blood pressure/hypotension - which

can lead to inadequate nimodipine dosing, or stopped treatment. The peaks/troughs of oral

dosing drive hypotension. Clinicians can use more frequent lower dosing to lower the risk, or

use vasopressors for continued nimodipine dosing. This process is onerous, and can lead to

noncompliance and underdosing, given a dynamic ICU and surgical intervention for aSAH

patients.

Our consultants indicate that there is a clear value proposition for all stakeholders, with (1)

crucial benefits to patient outcomes, (2) improving the physician/nursing burden, and (3)

potential to reduce financial burden to the healthcare system for hospitals/payors. While

the Phase III safety study was not designed to capture clinically significant efficacy, KOLs

believe that results show compelling evidence that GTx-104 IV allows for increased dosing of

nimodipine, which should maximize neuroprotection and optimize functional outcomes. This

is supported by consultants’ positive views on the secondary subanalyses, with positive trends

for multiple functional outcomes. In addition, the primary endpoint of clinically significant

hypotension suggests that GTx-104 has slightly better, or at worst similar tolerability – with very

similar AE profile to oral nimodipine. Given this profile, KOLs are excited to add GTx-104 to their

treatment armamentarium.