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CSL Behring gets FDA approval for rare genetic disease drug
  • By Marian Chu
  • Published 2017.06.23 11:44
  • Updated 2017.06.23 11:44
  • comments 0

The U.S. Food and Drug Administration Thursday approved Haegarda, CSL Behring’s drug for hereditary angioedema (HAE), a rare genetic condition that affects around 8,000 people in the United States.

Haegarda is an injection created from large pools of human plasma from U.S. donors administered under the skin to counter the rapid swelling of hands, feet, limbs, face, intestinal tract or airway in patients with HAE.

Patients with HAE have insufficient amounts of a blood protein called C1-esterase inhibitor (C1-INH). Haegarda is the first “C1 Esterase Inhibitor (Human)” administered under the skin to prevent attacks triggered by stress, surgery or infection in teenage and adult patients.

"The approval of Haegarda provides a new treatment option for adolescents and adults with Hereditary Angioedema," said Peter Marks, director of FDA’s Center for Biologics Evaluation and Research. "The subcutaneous formulation allows patients to administer the product at home to help prevent attacks."

Haegarda also received an Orphan Drug designation, which gives incentives to pharmaceutical companies to develop drugs to treat rare diseases. An orphan drug refers to a treatment that pharmaceutical companies often avoid due to high cost and limited potential for profitability. Drugs for rare diseases often fall under this category since developing medicinal products for these illnesses consume extensive research and development for a small target population.

Experts said that CSL Behring’s win would set back Shire’s position as a leader in the HAE market. The two companies have been in a battle to gain first mover advantage with Shire suing CSL for patent infringement of its HAE antibody, lanadelumab, showing the former’s efforts to block the rival’s drug launch. Both drugs have scored higher regarding efficacy and convenience over Shire’s enzyme replacement therapy, Cinryze.

yjc@docdocdoc.co.kr

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