“It takes many years in ultra-rare diseases to reach a US$1 billion threshold”. Alexion said at the time that it expected annual Kanuma sales to surpass $1 billion, though the company also said that education is required because LAL-D is little-known, suggesting that the launch might be slow.
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The Center for Drug Evaluation and Research (CDER) director Janet Woodcock commented: “LAL deficiency is a rare inherited genetic disorder that can lead to serious and life-threatening organ damage, especially when onset begins in infancy”. It is an ultra-rare disease, which is defined as a disease that affects fewer than 20 patients per one million of the general population.
Kanuma is administered once per week via intravenous infusion in patients with rapidly progressing LAL deficiency presenting in the first six months of life. The company charges about $440,000 a year for the treatment, which is used in a handful of patients who have a rare disease called PNH that causes the body to destroy its own red blood cells.
The press release notified that the Kanuma update involved approvals from two FDA centers.
Alexion will offer support to patients with LAL-D through its OneSource™ program. Clinicians could only provide nutrition and supportive care to infants, says Burton, who worked with Alexion to conduct the clinical trials. Without treatment, LAL-D is typically fatal within a matter of months in the youngest patients.
Alexion said it is preparing to serve patients in the USA with Kanuma and expects that Kanuma will become available commercially during the first week of January 2016.
The FDA approved Kanuma under Priority Review, and had previously granted Breakthrough Therapy Designation for Kanuma for LAL-D presenting in infants. The manufacturer of Kanuma was granted a rare pediatric disease priority review voucher — a provision meant to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
Both drugs were cleared in Europe back in September, but United Kingdom cost regulators have rejected use of Strensiq on the National Health Service in England and Wales because the firm failed to adequately justify the drug’s high cost (£336,000 per patient per year). The agency has now approved 41 new therapies in 2015, matching the chart-topping record for all of a year ago.
As per medical experts, people start experiencing onset of LAL-D when they are around the age of five. Reduced ALT values were generally seen within 2 weeks. Cholesteryl ester storage disease (CESD) is a milder version of LAL deficiency that presents later in childhood and beyond.
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The most common human side effects of the drug included diarrhea, vomiting, fever, nasal inflammation, anemia, cough, headache, constipation and nausea.
