Hopefully that re-designation occurs after a positive P3 trial and approval by the FDA. I get the impression, based on numbers that HIE will be right on the cusp of non-orphan but its hard to be sure.
According to Neuren's numbers in the Annual Report, PMS impacts between 1/8,000 - 1/15,000 people (resulting in approx 24,000 patients in the USA). Acknowledge those numbers might have increased after the recent publication discussed on the forum.
The HIE announcement suggests that 2-3 out of every 1,000 people in rich countries are impacted, up to 10-30 people per 1,000 people in poor countries. Hoping someone can check my math - but with ~3.6m people born in the US last year, the patient range would be between 7,200 - 10,800.
That is within the Orphan band/range as I understand it (<200k) but the interesting point is that the patient range will replenish every year. PMS numbers are a total as I understand it and growth each year will not be 1:1 with existing patient numbers, whereas with HIE, I gather it will be 1:1 growth every year. Google was not clear when I asked it but seemed to indicate that HIE impacts children and not really adults (can happen) so perhaps they out grow that diagnosis - hope that makes sense.
Either way, the growth trajectory of 2591 in HIE would arguably be parabolic!
Side note - The Cleveland Clinic does not directly link Autism to HIE, but does make a point of saying that Autism is likely caused by a combination of genetics and certain things related to pregnancy, labor and delivery (what you might see referred to as “environmental factors” or “prenatal events”). One of the listed "Environmental Factors" is 'Reduced oxygen to the fetus during pregnancy or delivery' - Autism Spectrum Disorder (ASD) Symptoms & Causes. Whilst not authoritative and to be taken with a grain of salt given their financial interests, this law firm is making the link (or at least attempting to do so) between HIE and Autism - Autism Caused by Hypoxic Ischemic Encephalopathy (HIE) | BILA
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