Replies to post #247325 on Biotech Values

[Mufaso]

Thanks for the additional info. Makes sense that the 0214 readout would likely be circa September.

Question for you (or anyone else on this board with expertise in PRV's)- do you think VK0214 can qualify for a rare disease pediatric voucher given the prognosis in the Cerebral form of X-ALD?
From: https://www.childrenshospital.org/conditions/adrenoleukodystrophy-ald

The defective gene in ALD, commonly referred to as a genetic mutation, can cause several different but related conditions: adrenomyelopathy (AMN), Addison’s disease, and — the most common and most devastating form — cerebral ALD. Cerebral ALD strikes boys between ages 4 and 10, leading to permanent disability and death usually within four to eight years.

Here is some info I found that leads me to think VK0214 would qualify for a pediatric PRV:

The revised draft guidance incorporates public comments received on the initial draft and provides FDA’s thinking regarding the provisions of the Advancing Hope Act of 2016, which updated the definition of a rare pediatric disease as one that is a rare disease and one where the disease is serious or life-threatening with the serious or life-threatening manifestations primarily affecting individuals from age zero to 18.

https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs