FDA Delays Decision on Alexion's Lead Drug Kanuma by Three Months

FDA Delays Decision on Alexion's Lead Drug Kanuma by Three Months
September 4, 2015
By Alex Keown, BioSpace.com Breaking News Staff

CHESHIRE, Conn. – Alexion Pharmaceuticals Inc. will have to wait an additional three months for the U.S. Food and Drug Administration (FDA)’s approval of Kanuma, an investigational enzyme replacement therapy for the treatment of lysosomal acid lipase deficiency (LAL-D), the company announced this morning.

Kanuma had been given a breakthrough therapy designation by the FDA and was expected for regulatory review Sept. 8. However, this morning Alexion said the company submitted additional Chemistry, Manufacturing and Controls (CMC) information following a request by the regulatory agency. The FDA did not ask for additional clinical data, Alexion said, but the data submitted will cause the three-month delay.

Alexion acquired Kanuma in May after the company inked an $8.4 billion cash and stock deal to acquire Synageva BioPharma corp. . The acquisition was expected to create the most robust rare disease pipeline in the biotech industry, David Hallal, Alexion’s chief executive officer said at the time of the acquisition. Kanuma was one of the new therapies seen as a jewel of the acquisition.

Geoffrey C. Borges, an analyst with Bernstein said although he anticipated Alexion’s stock to take a brief hit, he expected the weakness to be a “buying opportunity” for investors since it was unlikely the FDA would reject Kanuma altogether

“We continue to be optimistic about the likelihood of Kanuma's approval and its likely label. Given the strong clinical data in trials, the high level of unmet need, and the recent EC approval, we continue to expect that the drug will ultimately be approved and include a 100 percent probability of success in our models,” Borges said in an email.

was slightly down this morning, trading at $174.67 at the time of this writing.

LAL-D is a fatal disorder that leads to the buildup of fatty material in the liver, blood vessel walls and other tissues. Kanuma, if approved in all markets, which includes the U.S., could bring in approximately $1 billion in annual revenue, Hallal told Reuters. Hallal’s prediction is backed up by Borges. He predicted annual sales of $1.1 billion by 2021. Borges said he expects sales to grow exponentially between final approval by the FDA and 2021.

The European Commission granted regulatory approval for Kanuma for the treatment of patients of all ages with LAL-D on Sept. 1. In addition, a New Drug Application for Kanuma has been submitted to Japan’s Ministry of Health, Labor and Welfare.

Also on Sept. 1, the EU approved Alexion’s Strensiq to treat pediatric-onset hypophosphatasia (HPP). Strensiq enzyme-replacement therapy was developed to heal the skeletal manifestations of HPP, a sometimes fatal metabolic bone disease.

Before Kanuma and Strensiq received regulatory approval, Alexion’s only approved drug was Soliris, which was developed to treat paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Soliris has generated more than $2 billion in revenue since its launch in 2007.

Alexion is reiterating its 2015 financial guidance announced on July 30, 2015.

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