RARE Daily

Alexion Sets Stage for Sale of Ultomris in Canada for PNH, aHUS

October 24, 2023

Rare Daily Staff

Alexion Pharma Canada, AstraZeneca Rare Disease, said it concluded a letter of intent with the pan-Canadian Pharmaceutical Alliance for Ultomiris for the treatment of patients with paroxysmal nocturnal hemoglobinuria or atypical hemolytic uremic syndrome, a critical step to creating access for patients in Canada.

The filing of the letter of intent now allows individual provinces and territories to initiate the process to list Ultomiris on their formularies. The timing of that, Alexion said, will vary by province and territory.

PNH is a rare, chronic, progressive and potentially life-threatening blood disorder. It is characterized by red blood cell destruction within blood vessels (also known as intravascular hemolysis) and white blood cell and platelet activation, which can result in blood clots.

aHUS is a progressive, chronic condition with relapses. It is a type of thrombotic microangiopathy, which is a group of severe and potentially life-threatening rare disorders that cause blood clots and damage to the walls of the smallest blood vessels. The blood clots can cause injury to organs that may lead to organ failure and death.

Ultomiris, the first and only long-acting C5 complement inhibitor, provides immediate, complete and sustained complement inhibition. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Ultomiris is administered intravenously every eight weeks in adult patients, following a loading dose.

Ultomiris is approved in the United States, European Union, and Japan for multiple rare, autoimmune conditions.

“Our hope now is that provinces and territories quickly follow suit and provide reimbursement for this treatment,” said Barry Katsof, founder and president of the Canadian Association of PNH Patients.

Photo: Barry Katsof, founder and president of the Canadian Association of PNH Patients

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