Repurposing Leptin (Part 2): It Really Is A One-In-A-Million Drug
This article was originally published in RPM Report
Two decades after the “obesity gene” was the hottest story in biotech, a product from that research is finally going to reach the market. But it is for a disease that occurs in about .0001% of the US population.
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For larger organizations with interests in rare diseases, Bionest Partners believes it is necessary to maintain a separation from the rest of the company in order to keep the culture needed for successful product commercialization. Easier said than done.
Company sees “opportunity for a one-time price increase” when it takes over the lipodystrophy drug’s launch from AstraZeneca. At a current cost of about $325,000 per year, metreleptin already is on the ultra-high spectrum on par with other rare disease drugs.
FDA’s use of REMS continues to fall to almost undetectable levels, with the agency using the safety tools just a handful of times in the sixth full year of the program. REMS are now very much the exception and not the rule for new products – but still a very important exception.