Alexion inks intent letter to make Ultomiris available in Canada for aHUS

Aim is to get approved therapy onto public drug plans in all provinces

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Alexion, AstraZeneca Rare Disease signed off on a letter of intent in Canada that brings Ultomiris (ravulizumab) — approved in the country for certain people with atypical hemolytic uremic syndrome (aHUS) — a step closer to becoming available under public drug plans.

The letter, to the pan-Canadian Pharmaceutical Alliance (pCPA), will allow patients with aHUS and the acquired disorder paroxysmal nocturnal hemoglobinuria (PNH) to have publicly funded access to the rare disease medication.

With the letter of intent in place, individual provinces and territories across Canada may now begin the process of listing Ultomiris on their formularies, though the timeline may vary by province and territory.  

“It is critical that progress is made on the implementation of Canada’s rare disease strategy and that patients have access to the therapies they need to live healthier lives,” Gaby Bourbara, general manager of Alexion Canada, said in a company press release.

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The company now is working with the pCPA — a collaborative effort between provinces and territories to negotiate public drug plans in Canada — to determine under what conditions Ultomiris can be reimbursed.

“We look forward to our ongoing collaboration with provincial, territorial and federal jurisdictions to finalize listing agreements to better serve the rare disease community,” Bourbara said.

Both aHUS and PNH occur when the complement system, a domino-like chain of proteins that work with the immune system to protect the body against infection, becomes activated in an uncontrolled manner.

In aHUS, this causes thrombotic microangiopathy — when clots form in small blood vessels throughout the body, blocking blood flow to tissues and potentially damaging internal organs, particularly the kidneys.

Ultomiris is an antibody that works by targeting and inhibiting C5, a complement protein. By doing this, the medication prevents the complement system from becoming overly active, thereby helping to control symptoms of aHUS or PNH.

It is approved in Canada and elsewhere to treat complement-mediated thrombotic microangiopathy in adults and children, ages 1 month and older, who have a diagnosis of aHUS. The therapy also is approved for certain adults with PNH.

In the U.S., Ultomiris is available only through a restricted program called Ultomiris REMS due to the risk of meningococcal bacterial infection, which can be serious and life-threatening.

Having a choice in treatment is very valuable to the aHUS community. It’s important that ravulizumab [Ultomiris] be listed soon on all provincial and territorial formularies so patients can live their lives to the fullest.

“aHUS Canada is pleased that the pCPA has come to an agreement to make ravulizumab [Ultomiris] publicly available to aHUS patients in Canada,” said Michael Eygenraam, chair at aHUS Canada, a patient advocacy organization.

“Having a choice in treatment is very valuable to the aHUS community. It’s important that ravulizumab [Ultomiris] be listed soon on all provincial and territorial formularies so patients can live their lives to the fullest,” Eygenraam said.

For people with PNH, the decision is equally important.

“In many countries around the world, ravulizumab [Ultomiris] is first-line therapy for most patients with PNH. We’re pleased to see that the path is now clear for patients in Canada to soon have publicly funded access as well,” said Christopher Patriquin, MD, chair of the Canadian PNH Network.

Alexion voiced its appreciation for all of the provinces and territories that have worked with the company to help make Ultomiris available to patients.

“We would like to thank participating jurisdictions within the pCPA for their partnership in recognizing the need for this important innovation for Canadians living with PNH, as well as those with aHUS,” Bourbara said.