Alexion's Kanuma FDA Action Date Delayed by Three Months

Alexion Pharmaceuticals, Inc. ALXN announced that the FDA has extended the review period by three months for the biologics license application (BLA) for its investigational enzyme replacement therapy, Kanuma, for the treatment of lysosomal acid lipase deficiency (LAL-D).

The FDA is now expected to render a decision on Kanuma’s BLA by Dec 8, 2015. We note that the company had submitted additional Chemistry, Manufacturing and Controls information, which led to the extension of the review period.

The FDA has granted Breakthrough Therapy designation to Kanuma for LAL-D in infants. Alexion has also submitted a new drug application for Kanuma to Japan’s Ministry of Health, Labour and Welfare.

We note that earlier this month, Kanuma was approved in the EU as a long-term enzyme replacement therapy in patients suffering from LAL-D. The company plans to launch the product in Germany in October and is working on reimbursement processes with health care authorities in all of the major European countries.

Alexion has reiterated its 2015 guidance despite the delay in the FDA action date for Kanuma. The company expects adjusted earnings per share in the range of $4.70 to $4.80 for 2015. The company expects revenues in the range of $2.6–$2.62 billion.

There are currently no therapies approved for the treatment of LAL-D, indicating an untapped market for Kanuma. Approval of Kanuma would reduce the company’s dependence on Soliris.