The United States Food and Drug Administration’s Center for Veterinary Medicine has just approved a new pharmaceutical application from Alexion which will use genetically modified DNA from a chicken to develop a new treatment of an extremely rare enzyme disorder.
At the same time, the US FDA Center for Drug Evaluation and Research has approved the treatment—under the brand name Kanuma—which is actually purified from the egg whites of those genetically modified chickens.
“Using this technology, these patients for the first time ever have access to a treatment that may improve their lives and chances of survival,” comments FDA drug evaluation director Janet Woodcock.
Babies born with this enzyme deficiency often do not survive past a few months. This enzyme—LAL—is a key component in fat metabolism; this means that those born without this enzyme will have fat building up in the organs.
“LAL deficiency is a rare inherited genetic disorder that can lead to serious and life-threatening organ damage, especially when onset begins in infancy,” Woodcock continues.
In the clinical trial, six of nine babies who had been administered Kanuma actually survived beyond 12 months. No child with this condition has ever lived past 8 months.
Of course, this is great news, even with the limited success; still, Alexion Chief Executive David Hallal comments, “given the extremely low awareness of this disease, the high mortality and low incidence in prevalence, we would expect the dynamics of this launch to be slow and steady. It takes many years in ultra-rare diseases to reach a $1 billion threshold.”
Hallal goes on to say, “We expect that over time we’ll have success in identifying patients with the disease and help them get the treatment.”