Abstract: 554 AN OPEN-LABEL, DOSE ESCALATION STUDY TO ASSESS THE SAFETY AND EFFICACY OF AAV1-LPL S447X GENE THERAPY WITH ALIPOGENE TIPARVOVEC (AMT-011) FOR PATIENTS WITH SEVERE HYPERTRIGLYCERIDEMIA DUE TO LIPOPROTEIN LIPASE DEFICIENCY (LPLD)

Abstract

Lipoprotein Lipase Deficiency is a classic target for gene replacement therapy. Glybera (alipogene tiparvovec) is a genetically engineered adeno-associated virus (AAV) that introduces a functional copy of the lipoprotein lipase gene into patients’ muscle cells. It is also the most expensive drug ever made. Doctors, health insurers, and pharmacy benefits companies, among others, are joining the chorus of patient voices complaining about high drug prices. Some moderation of drug prices is likely, but severe price controls could have a devastating effect on the biotech Valley of Death (VoD). Angel investors are attracted to the lucrative drug development pathway that enables stratospheric prices for specialty drugs. If no exits can be achieved at enormous multiples because the system will not pay for these multibillion-dollar drug franchises, then early-stage investors will have much less incentive to participate in very high risk bets. Incentives need to be adjusted to encourage rationalization of the flow of money in the VoD, which will in turn improve outcomes.