Baxalta wins FDA approval with a treatment for a rare bleeding disease

Baxalta CEO Ludwig Hantson

Baxalta ($BXLT) picked up FDA approval for a drug that treats von Willebrand disease, a rare inherited bleeding disorder that results from a missing protein.

The treatment, developed as BAX 111, is a synthetic version of von Willebrand factor, a protein key to the coagulation process. Von Willebrand disease, which affects about 1% of the population, stems from a lack of that protein, and Baxalta's injectable therapy is designed to halt resulting bleeds when used on demand.

Baxalta plans to launch the drug under the brand name Vonvendi, expanding its multibillion-dollar franchise of treatments for bleeding disorders, led by the hemophilia treatments Advate and Feiba. Last month, Baxalta won FDA approval for Adynovate, a long-acting version of Advate poised to contend with a new therapy from Biogen ($BIIB) and in-development products from Bayer and Novo Nordisk ($NVO).

The company, which spun out of Baxter ($BAX) over the summer, got off the ground with the ambitious goal of launching 20 new products by 2020, planning to grow its annual revenue to $8.5 billion in the process. To get there, Baxalta has embraced external innovation, widening its aperture for potential partnerships to expand its pipeline in oncology and inflammatory disease.

Meanwhile, the company is facing some unwelcome M&A attention from Shire ($SHPG), rebuffing a $30 billion buyout offer earlier this year. Shire has publicly stated that it's unwilling to give up on the idea of a merger, and reports have surfaced over the past month suggesting the company may come forward with a sweetened pitch in the coming weeks.

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