In June, Alexion Pharmaceuticals acquired Massachusetts-based biopharma firm Synageva for $8.4bn (€7.5bn), adding the company’s lead product Kanuma (sebelipase alfa) - a recombinant form of the human LAL enzyme for the treatment of lysosomal acid lipase deficiency – to its orphan drug pipeline.
Kanuma was accepted for evaluation in February under the US Food and Drug Administration’s (FDA) priority review programme. A target action date of September 8 was established, but the Agency has now extended this by three months.
“In response to a recent request from the FDA, Alexion submitted additional Chemistry, Manufacturing and Controls (CMC) information” the company said in a statement.
“Due to the timing of this submission, the FDA extended the PDUFA date to allow additional time for review of the new information. The FDA has not asked for additional clinical data.”
The US delay comes a week after the biologic was approved for commercialisation in Europe for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase deficiency.
Last year, NICE - the UK’s governing body for the use of drugs in the National Health Service - delayed its decision to approve Alexion’s lead product Soliris (Eculizumab) after demanding further R&D and manufacturing information to justify the high price tag. The product was eventually recommended despite the annual £340,000 ($513,000) cost per patient.
John Byrne from PR firm Drury Porter Novelli told us Alexion would not provide further information on this matter.
Kanuma Manufacturing
Kanuma is made using a proprietary expression platform - acquired as part of the Synageva deal – which is based on a system of vectors and methods that allow the generation of genetically modified hens to produce high levels of therapeutic protein in egg white (EW).
“The EW matrix facilitates bulk storage of unpurified EW prior to purification for prolonged periods and is one of a number of manufacturing advantages of our platform over traditional protein manufacturing technologies,” Synageva’s 2014 annual report says.
Some clinical production of Kanuma occurred at in-house manufacturing facilities in Athens, Georgia, and Holden, Massachusetts - both of which are now operated by Alexion - but the firm had also entered “into commercial service and supply agreements with third party providers to produce commercial product in preparation for the potential approval of Kanuma,” the report states.