Alexion Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) for Kanuma (sebelipase alfa), a new enzyme replacement therapy (ERT), to treat patients suffering from lysosomal acid lipase deficiency (LAL-D).
Kanuma is the first therapy approved in the US to treat patients with LAL-D, a genetic and progressive ultra-rare metabolic disease where patients suffer multi-organ damage and premature death.
LAL-D is an ultra-rare disease affecting fewer than 20 patients in one million.
These patients can experience a rapid onset of life-threatening disease manifestations, and without treatment, the youngest patients with LAL-D face rapid disease progression that is typically fatal within a matter of months.
Approval was based on data from two clinical trials and a supporting open-label extension study comprising infant, paediatric, and adult patients with LAL-D.
The results showed significant benefits in terms of survival (67%) in patients with the infant form of LAL-D beyond 12 months, compared with none out of 21 patients in an untreated historical cohort.
The company noted that in paediatric and adult patients with LAL-D (ages four to 58 years), treatment with Kanuma resulted in larger reductions from baseline in ALT values and liver fat content, as measured by MRI, compared to treatment with placebo.
Alexion chief executive officer David Hallal said: "We are pleased with the FDA approval of Kanuma, a transformative treatment for patients with LAL-D, a devastating, ultra-rare disease that causes premature death in infants and multi-organ damage in those who survive.
"Importantly, the label includes a survival benefit in infants and reductions in important markers of liver disease, including ALT and liver fat content, as well as significant improvements in lipid parameters, in children and adults.
"This approval also strengthens Alexion’s global leadership in rare diseases as we broaden our product portfolio to transform the lives of more patients with severe and life-threatening disorders. We look forward to bringing Kanuma to patients with LAL-D and their physicians in the US."
LAL-D occurs due to genetic mutations that result in a marked decrease or loss in LAL enzyme activity in the lysosomes across multiple body tissues, which leads to the chronic build-up of cholesteryl esters and triglycerides in the liver, blood vessel walls, and other organs.
LAL Solace board of directors Brett Billmeyer said: "Patients with LAL-D often suffer for years from a delayed diagnosis, only to be further devastated once properly diagnosed because there have been no approved treatments for this disease.
"Today, we are thrilled to welcome the FDA approval of Kanuma, providing an approved and effective treatment to patients with LAL-D and their families for the first time and, with it, bringing much-needed awareness to this often overlooked and devastating disease."
Alexion will provide support to LAL-D patients through its OneSource programme, which offers personalised support from a dedicated nurse case manager, who can help patients understand their insurance benefits and receive reimbursement assistance.
The company expects to make Kanuma commercially available for patients during the first week of January 2016.
Image: Alexion’s Kanuma will be launched in the US in January 2016. Photo: courtesy of Business Wire/Alexion Pharmaceuticals.