In February 2009, the US Food and Drug Administration (FDA) approved GTC Biotherapeutics’ orphan drug ATryn, an anticoagulant used for the prevention of blood clots in patients with hereditary antithrombin (AT) deficiency, made in the milk of genetically engineered (GE) goats.
Almost seven years later and the FDA has approved a second biopharmaceutical product made using a transgenic animal: Alexion Pharmaceuticals’ Kanuma (sebelipase alfa) for the treatment of patients with the rare disease lysosomal acid lipase (LAL) deficiency.
Kanuma is made using GE chickens containing an rDNA construct responsible for producing rhLAL – a protein that functions in place of the missing or inactive LAL protein in the patient - in their egg whites. The whites are then refined and the rhLAL protein extracted and purified.
Alexion, therefore, received both the approval of the FDA’s Center for Drug Evaluation and Research (CDER) and its Center for Veterinary Medicine (CVM) which approved the application for a recombinant DNA (rDNA) construct in GE chickens.
“We reviewed all of the data to ensure that the hens do produce rhLAL in their egg whites, without suffering any adverse health effects from the introduced rDNA construct,” said CVM Director Bernadette Dunham.
“The company has taken rigorous steps to ensure that neither the chickens nor the eggs will enter the food supply, and we have confirmed their containment systems by inspecting the manufacturing facilities.”
Alexion gained Kanuma in June through its $8.4bn (€7.5bn) acquisition of Massachusetts-based biopharma firm Synageva.
The news this week comes as a relief for Alexion which saw the approval of Kanuma pushed back by the FDA which demanded more chemistry, manufacturing and controls (CMC) data in September.
CEO David Hallal said in a statement he was “pleased with the FDA approval” which helps “strengthens Alexion’s global leadership in rare diseases.”
Alexion’s lead product is Soliris, a monoclonal antibody for the rare disease atypical Haemolytic Uraemic Syndrme (uHUS) which is priced – in the UK at least – at over $550,000 per patient per year.
Kanuma is expected to be launched in the first week of January.