Last week’s Advisory Committee vote implies the Praluent and Repatha labels might be somewhat restricted until their outcomes trials are completed. Presumably positive outcomes data would lead to de-restriction of the labels
The Praluent trial (ODYSSEY) has a scheduled completion date of January 2018, however the trial includes two interim assessment points which occur after approximately 50% and 75% of the expected number (1,613) of cardio- and cerebrovascular events has occurred
The trial can end at the 50% assessment only for futility; it cannot end at this point with a positive finding. We estimate that a finding of futility at the 50% point, if it occurred, should occur no later than October of this year
The trial can end at the 75% assessment point either for futility, or because sufficient evidence of efficacy exists. A finding for futility, if it occurred, should occur no later than September of 2016. A finding that sufficient evidence of efficacy exists at the 75% assessment should occur no earlier than November of 2016
Assuming a July approval, we’ll have to wait at least 15 months for positive outcomes data, and the wait could be as long as 29 months
We remain bullish on PCSK9’s generally, and on Praluent specifically. Even with restricted labels, for practical reasons we expect large numbers of persons to take the PCSK9’s even ahead of outcomes evidence. These include patients with high CV risks scores who still have high LDL-C’s despite aggressive statin therapy, and patients with high LDL-C’s who are intolerant to statins
The feasibility of limiting the PCSK9’s to persons with familial hypercholesterolemia is limited by the fact that very few heterozygous patients are formally diagnosed. And, despite the Advisory Committee’s legitimate suspicion that many patients claiming to be statin intolerant could tolerate statins if carefully re-challenged, such careful re-challenges rarely occur in clinical practice – and become even less likely to occur once PCSK9’s exist as an alternative
For our full research notes, please visit our published research site
