FDA approves Alexion drug for ultra-rare disease

By Bill Berkrot and Samantha Kareen Nair (Reuters) - The U.S. Food and Drug Administration on Tuesday approved Alexion Pharmaceutical Inc's treatment for an ultra-rare and potentially fatal genetic disorder. Alexion's enzyme-replacement therapy, Kanuma, which received European approval in September, was approved to treat patients, including infants, with lysosomal acid lipase deficiency (LAL-D), that can lead to liver failure, heart disease and early death. Alexion shares were up $8.07, or 4.5 percent, at $187.22. There are only a few thousand patients with LAL-D in the United States, Alexion said. It estimates four to eight babies per year will be born with the infantile form of LAL-D. But life-saving treatments for ultra-rare diseases can command extremely high prices. The company believes Kanuma will eventually generate annual sales in excess of $1 billion. "But 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," Alexion Chief Executive David Hallal said in a telephone interview. "It takes many years in ultra-rare diseases to reach a $1 billion threshold." Onset of LAL-D tends to happen around the age of 5. Infants born with the disease usually die before their first birthday. In a clinical trial, six of nine babies who received Kanuma survived beyond 12 months. "We expect that over time we'll have success in identifying patients with the disease and help them get the treatment," Hallal said. Kanuma, which the company acquired with its $8.4 billion purchase of Synageva BioPharma in June, could be priced at $300,000 to $400,000 annually, Morningstar analyst Stefan Quenneville forecast. Alexion's flagship drug Soliris, which treats two rare diseases and is in late-stage testing for three more, costs around $500,000 a year. The company plans to discuss pricing for Kanuma later on Tuesday. Unlike most drugmakers that charge much higher prices for medicines sold in the United States, Alexion plans similar pricing globally. "That's a strong belief that we have that our drug launches outside of the U.S. should not be subsidized by the U.S.," Hallal said. Kanuma is developed from the egg whites of genetically engineered chickens and required approval from both the FDA's veterinary division and its normal drug evaluation division. "Using this technology, these patients for the first time ever have access to a treatment that may improve their lives and chances of survival," FDA drug evaluation director Janet Woodcock said in a statement. (Additional reporting by Rosmi Shaji in Bengaluru; Editing by Don Sebastian and James Dalgleish)