CHMP turns down Kynamro
pharmafile | December 17, 2012 | News story | Sales and Marketing | CHMP, Genzyme, Kynamro, Sanofi
Genzyme has expressed frustration at the CHMP’s negative opinion of its novel drug Kynamro, and plans to ask it to reconsider.
The EMA committee’s stance flies in the face of a positive decision given in October by an influential FDA advisory panel: the FDA is currently reviewing it and expects to reach a final judgment in January.
Sanofi subsidiary Genzyme has developed Kynamro (mipomersen) as a novel treatment for homozygous familial hypercholesterolaemia (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.
“We are disappointed by the [CHMP]’s recommendation,” said Genzyme president David Meeker. “Patients with HoFH carry extreme, ongoing cardiovascular risk with significantly elevated LDL-C levels despite use of currently available therapies.”
Kynamro 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 company would ‘work closely’ with the CHMP during the re-examination process to address its concerns, Meeker pledged.
The drug is the lead product of Isis Pharmaceuticals, whose chief operating officer B. Lynne Parshall insisted: “We believe that we have generated significant evidence in support of Kynamro.”
In some ways the CHMP decision cannot come as a complete surprise: while the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee did vote in favour, it was far from unanimous.
Reviewing all the efficacy and safety data provided by Genzyme, the US committee voted just 9 to 6 on the drug’s risk/benefit.
The company has put forward as evidence a pivotal Phase III double-blind, placebo-controlled study in HoFH patients as well as three Phase III studies in other high-risk hypercholesterolemia patients, and an ongoing long-term extension study.
Other drugs are also 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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