BioCryst Gains Drug Approval in Canada, Switzerland

A drug produced by BioCryst Pharmaceuticals of Durham has won regulatory approval in Canada and Switzerland for preventing hereditary angioedema (HAE), a rare disease of swollen tissues caused by excessive fluid buildup.

Health Canada, the government agency responsible for health policy, authorized the use of Orladeyo (berotralstat) for preventing recurrent HAE attacks in patients 12 years and older.

“We appreciate Health Canada’s thorough and timely review of Orladeyo, and with this authorization we are one step closer to bringing this important treatment option to Canadians living with HAE,” said Jared Rhines, general manager of BioCryst Canada. “We look forward to working with stakeholders across the country to ensure these Canadians can receive timely and appropriate access to Orladeyo.”

Swissmedic, the government authority for authorizing therapeutic products in Switzerland, granted similar marketing authorization in that country.

“Orladeyo offers people with HAE in Switzerland and their physicians the first orally administered non-steroidal therapy for preventing HAE attacks and provides the community with another vitally important treatment option,” said Henrik Balle Boysen, executive vice president and chief operating officer of HAE International, a global non-profit network of patient associations dedicated to improving the lives of people with HAE.

The Canadian and Swiss authorizations follow approvals in the United States, Europe, and other markets.

HAE is estimated to affect about one in 10,000 to one in 50,000 people worldwide.

The genetic disorder involves recurrent attacks of severe swelling, or edema, in various parts of the body, including the hands, feet, genitals, stomach, face and/or throat. Swelling in the airway can restrict breathing and be fatal.

Orladeyo, a synthetic small molecule, is the first and only oral therapy designed specifically to prevent HAE attacks. One capsule per day works by decreasing the activity of plasma kallikrein, an enzyme involved in fluid buildup outside the blood vessels.

Company focused on rare diseases

BioCryst discovers novel, oral small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease.

In addition to Orladeyo, the company has one other commercially approved medicine, Rapivab, a single-dose intravenous drug for treating influenza.

Other potential medicines in development include BCX9930, an oral Factor D inhibitor for treating complement-mediated diseases; BCX9250, an ALK-2 inhibitor for treating fibrodysplasia ossificans progressive; and galidesivir, a broad-spectrum antiviral for treating Marburg virus disease and yellow fever.

BioCryst was founded in 1986 and became a publicly traded company in 1994. Its shares are traded on the Nasdaq stock exchange under the ticker symbol BCRX.

In addition to its headquarters in Durham, BioCryst has European headquarters in Dublin and a research and development center in Birmingham, Ala.