The U.S. Food and Drug Administration on Thursday approved Ionis Pharmaceuticals’ drug to prevent instances of severe swelling in various parts of the body of patients with a rare genetic disorder.<\/p>\n
Shares of the company were up 1.1% in afternoon trading.
\nThe drug, Dawnzera, is approved as a so-called “prophylaxis” to prevent symptoms of a rare genetic disease called hereditary angioedema (HAE) in adults and pediatric patients 12 years of age and older.<\/p>\n
The condition causes frequent attacks of severe swelling in various parts of the body, including the hands, feet, genitals and face.<\/p>\n
Dawnzera will be available in the United States in the coming days with a list price of $57,462 per dose, the company said.<\/p>\n
HAE is estimated to affect about 7,000 patients in the U.S., according to Ionis.
\nThe drug, chemically known as donidalorsen, prevents HAE attacks by lowering levels of a protein called prekallikrein (PKK) that drives swelling and inflammation.<\/p>\n
Dawnzera can be self-administered through an under-the-skin injection once every four or eight weeks.<\/p>\n
The approval was supported by late-stage study results that showed the drug, dosed once every four weeks, reduced monthly attacks by 81% compared to a placebo over 24 weeks.<\/p>\n
Current FDA-approved therapies for HAE fall into two groups: prophylaxis to prevent attacks and on-demand medicines to treat acute attacks.<\/p>\n
Other approved prophylactic drugs include Takhzyro, developed by Japan’s Takeda Pharmaceutical and Haegarda from CSL Behring.<\/p>\n
There is a lot of excitement for donidalorsen as patients who are not doing well on existing therapies have an option to switch to Dawnzera, which has a higher efficacy, TD Cowen analyst Yaron Werber said ahead of the approval.<\/p>\n
Takhzyro, given every two weeks, showed an average of 87% fewer attacks compared to a placebo over 6.5 months in a separate study involving adult and adolescent patients.<\/p>\n
Werber estimates peak annual sales of Ionis’ drug to be $509 million by 2032.<\/p>\n","protected":false},"excerpt":{"rendered":"
The U.S. Food and Drug Administration on Thursday approved Ionis Pharmaceuticals’ drug to prevent instances of severe swelling in various parts of the body of patients with a rare genetic disorder. Shares of the company were up 1.1% in afternoon trading. The drug, Dawnzera, is approved as a so-called “prophylaxis” to prevent symptoms of a […]<\/p>\n","protected":false},"author":2,"featured_media":1509,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[40],"tags":[372],"class_list":{"0":"post-1508","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-sitefansalaran-food-and-drug","8":"tag-us-fda-approves-ionis-drug-for-rare-genetic-disorder"},"yoast_head":"\n