Alstrom: August FDA Meeting for Regulatory Pathwayhttps://www.docdroid.net/WjrVWcg/prometic-190718.pdf
The addressable patient population for Alstrom is very small. Only c. 1200 affected individuals have been identified worldwide. However, Alstrom may prove to be of vital strategic importance to Prometic on several levels. As a case study for PBI-4050’s ability to treat fibrosis, we can not imagine a more challenging model with multiple organs being susceptible to the formation of scar tissue. The phase 2 open label trial has already shown some excellent and sustained results, and Prometic is due to meet with the FDA in August in order to delineate a regulatory pathway.
Given the ultra-orphan nature of the condition it is possible that Alstrom will prove to be an easier first route to market than IPF. We would not expect the pivotal phase 3 trial in IPF to complete before 2021, although it the the six- month interim data is likely to be published earlier. Given the patient population and complete absence of current treatments for Alstrom, we believe that the time and cost to approve Alstrom in a clinician’s setting is likely to be considerably less than for IPF, particularly if the FDA grants Fast Track designation.
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