Empowered Patient Podcast Oral Treatment for Rare Disease Pediatric Growth Hormone Deficiency with Rick Hawkins Lumos Pharma
Rick Hawkins, CEO and Chairman of Lumos Pharma, is developing an oral compound to treat idiopathic pediatric growth hormone deficiency. Currently, the only treatment for this condition is an injection of recombinant growth hormone. The small molecule drug LUM-201 increases the amplitude of the 23-25 peak releases of growth hormone during the day and night and stays within the normal physiology of the endocrine feedback loop.
Rick explains, "Although we are a drug that we're starting with pediatric growth hormone deficiency, there are actually 11 different diseases of growth hormone deficiency. PGHD happens to be one of the largest ones, which is why we are starting there. In addition to the fact that the burden of care, kids taking lots of injections for that period of time, having an oral alternative is a huge step up in not just compliance and ease of use, but the overall burden of care improves dramatically."
"First of all, this is another one of those examples in the pharmaceutical industry where Big Pharma puts a drug on the shelf, and, once again, it takes maybe one individual or a small group of people to understand that this is the drug that really needs to be taken outside the traditional walls of the pharmaceutical industry. In this case, it was Merck. Merck developed this small molecule as an indication in diseases or the symptoms of aging."
"The drug essentially did what it was supposed to do. However, the FDA essentially said you have no regulatory pathway. These are symptoms of aging and really not a disease. So, they put the drug on the shelf."
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