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
(20min delay)