Biopharmaceutical firm BioCryst Pharmaceuticals Inc. (BCRX:NASDAQ), which is focused on the development of novel, oral, small-molecule medicines to address rare unmet diseases, announced yesterday that "the U.S. Food and Drug Administration (FDA) has approved oral, once-daily ORLADEYO™ (berotralstat) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older."
Anthony J. Castaldo, president and CEO of the U.S. Hereditary Angioedema Association (HAEA), commented, "ORLADEYO offers people with HAE and their physicians the first orally administered non-steroidal option for preventing HAE attacks and represents an important and welcome step in making more treatment options available to physicians and patients."
The company reported that ORLADEYO significantly reduced attacks at 24 weeks, and this reduction was sustained through 48 weeks during its pivotal Phase 3 APeX-2 trial. The firm noted that "HAE patients who completed 48 weeks of treatment (150 mg) saw reductions in their HAE attack rates, from a mean of 2.9 attacks per month at baseline to a mean of 1.0 attacks per month after 48 weeks of therapy."
APeX-2 trial investigator Marc Riedl, M.D., professor of medicine and clinical director of the U.S. Hereditary Angioedema Association Center at U.C.S.D., remarked, "Patients and physicians acknowledge that HAE treatments can add a burden to patients' lives. As an oral, once-daily option, ORLADEYO can provide significant attack reduction and lessen the burden associated with injections and infusions."
BioCryst Pharmaceuticals' President and CEO Jon Stonehouse stated, "The FDA approval of ORLADEYO fulfills a promise BioCryst made to HAE patients that we were committed to helping them achieve the dream of an oral, once-daily medicine to prevent and reduce the burden of their attacks."
The firm said that it is rolling out a new program designed to streamline access to therapy for HAE patients taking ORLADEYO. The company stated that through EMPOWER Patient Services, which is administered by Optime Care Inc., healthcare providers and HAE patients will have a single point of contact for easy access to ORLADEYO along with a special dedicated care coordinator to aid patients with financial support tools and insurance reimbursement support.
The company explained that "ORLADEYO™ (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older and that one capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein."
HAE is a disorder characterized by recurrent episodes of severe swelling often in face, limbs, intestinal tract and airway which frequently occur without a known trigger. Intestinal tract episodes can cause severe abdominal nausea, pain and vomiting while swelling in the air passage way can restrict breathing resulting in a potentially life-threatening airway obstruction.
BioCryst is a biopharmaceutical company based in Research Triangle Park, N.C. The firm concentrates its efforts on discovering and developing novel, oral, small-molecule medicines to treat rare diseases with unmet medical needs where enzymes play a key role in the disease's biological pathway. The company noted that ORLADEYO™ has been submitted for regulatory review and approval in the EU and Japan.
BioCryst Pharmaceuticals began the day with a market capitalization of around $906.7 million with approximately 176.6 million shares outstanding and a short interest of about 21.6%. BCRX shares opened 14% higher today at $5.85 (+$0.715, +13.92%) over yesterday's $5.135 closing price. The stock has traded today between $5.745 and $6.25 per share and is currently trading at $6.03 (+$0.90, +17.27%).[NLINSERT]
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