FDA committee recommends Kynamro
pharmafile | October 23, 2012 | News story | Research and Development, Sales and Marketing | FDA, Genzyme, Kynamro, Sanofi
Sanofi’s anti-cholestrol drug Kynamro has been recommended by an FDA committee, paving the way for US approval early next year.
Developed by Sanofi subsidiary Genzyme, the drug is the lead product of Isis Pharmaceuticals and is designed as a novel treatment for homozygous familial hypercholesterolemia (HoFH).
This is a genetic condition, inherited from both parents, which causes high LDL cholesterol (LDL-C) levels and leads to increased risk of premature heart disease.
Kynamro (mipomersen sodium) is an apo-B synthesis inhibitor aimed at reducing LDL-C by preventing atherogenic lipoproteins – the particles that carry cholesterol through the bloodstream – from forming in the first place.
The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted in favour of the efficacy and safety data provided by Genzyme – in particular looking at one pivotal Phase III double-blind, placebo-controlled study in HoFH patients.
Though far from unanimous – the vote on the drug’s risk/benefit was 9 to 6 – the positive opinion will be taken into account by the FDA, which expects to reach a decision by the end of January 2013.
The investigational product is also awaiting European approval.
Other data reviewed by the US committee included three Phase III studies in other high-risk hypercholesterolemia patients, and an ongoing long-term extension study.
Genzyme chief executive David Meeker declared himself ‘very encouraged’ by the verdict, saying it was “a significant and positive step in our efforts to bring this important new therapy to patients and families affected by this often unrecognised genetic disorder”.
The most severely-affected patients can have LDL-C which is two to four times higher than recommended levels, even though they may already be taking other cholesterol-lowering medicines.
“Kynamro could represent a significant advance for patients with HoFH, who are unable to adequately control their LDL-C and remain at significant risk of a cardiac event,” said B. Lynne Parshall, chief operating officer of Isis.
Kynamro blocks the production of apolipoprotein B (apo B), the protein that provides the structural core for LDL and lipoprotein-a (Lp(a)).
Other drugs are currently in production to combat HoFH, including Sanofi and Regeneron’s SAR236553, an injectable fully-human antibody targeting the PCSK9 protein, which is currently in a global Phase III trial.
Amgen’s AMG 145, which was in Phase Ib trials, has a similar action, and analysts expect other drugs blocking the PCSK9 pathway, including those in earlier stages of development at Merck and Bristol-Myers Squibb, could generate annual future sales of up to $20 billion.
Adam Hill
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