Alexion, AstraZeneca Rare Disease, concludes Letter of Intent (LOI) with the pan-Canadian Pharmaceutical Alliance (pCPA) for Ultomiris for the treatment of patients with paroxysmal nocturnal hemoglobinuria or atypical hemolytic uremic syndrome

The LOI is a critical step in ensuring patients living with PNH or aHUS have public access to Ultomiris, the first and only long-acting C5 complement inhibitor

 MISSISSAUGA, ON, Oct. 24, 2023 /CNW/ - Alexion Pharma Canada Corp., AstraZeneca's Rare Disease group, has entered into a Letter of Intent (LOI) with the pan-Canadian Pharmaceutical Alliance (pCPA) for Ultomiris (ravulizumab) for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) and the treatment of adult and paediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy. With the LOI in place with the pCPA, individual provinces and territories may now initiate the process to list Ultomiris on their formularies, the timing for which will vary by province and territory.  

PNH is a rare, chronic, progressive and potentially life-threatening blood disorder. It is characterised by red blood cell destruction within blood vessels (also known as intravascular haemolysis) and white blood cell and platelet activation, which can result in thrombosis (blood clots). ^1-3

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.^6-9

"In many countries around the world, ravulizumab 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 Dr. Christopher Patriquin, MD, MSc, FRCPC, Chair of the Canadian PNH Network. 

"The Canadian Association of PNH Patients welcomes the recent decision by the pCPA to enable Canadian patients to join PNH patients from many other countries around the world who are already experiencing the benefits of ravulizumab. Our hope now is that provinces and territories quickly follow suit and provide reimbursement for this treatment," said Barry Katsof, Founder & President of the Canadian Association of PNH Patients.

"aHUS Canada is pleased that the pCPA has come to an agreement to make ravulizumab publicly available to aHUS patients in Canada. Having a choice in treatment is very valuable to the aHUS community. It's important that ravulizumab be listed soon on all provincial and territorial formularies so patients can live their lives to the fullest," said Michael Eygenraam, Chair, aHUS Canada.

Gaby Bourbara, General Manager of Alexion Canada, said: "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. 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. We look forward to our ongoing collaboration with provincial, territorial and federal jurisdictions to finalize listing agreements to better serve the rare disease community."

Notes

PNH

PNH is a rare, chronic, progressive and potentially life-threatening blood disorder. It is characterised by red blood cell destruction within blood vessels (also known as intravascular haemolysis) and white blood cell and platelet activation, which can result in thrombosis (blood clots). ^1-3

PNH is caused by an acquired genetic mutation that may happen any time after birth and results in the production of abnormal blood cells that are missing important protective blood cell surface proteins. These missing proteins enable the complement system, which is part of the immune system and is essential to the body's defence against infection, to 'attack' and destroy or activate these abnormal blood cells.¹ Living with PNH can be debilitating, and signs and symptoms may include blood clots, abdominal pain, difficulty swallowing, erectile dysfunction, shortness of breath, excessive fatigue, anaemia and dark-coloured urine.^1,4,5

aHUS

aHUS 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.^6-9 Signs, symptoms and complications of TMA include organ damage, such as to the kidneys, heart, brain, and/or other organs; shortness of breath; high blood pressure; thrombosis (blood clots); low platelet count; anaemia; fatigue, confusion and bruising.^6,8-14

aHUS is a progressive, chronic condition with relapses. It is caused by a combination of genetic and/or environmental factors resulting in dysregulation of the complement system, which is part of the body's immune system and essential to the body's defence against infection. It may appear in the presence or absence of a "trigger" or co-existing condition.^15-19

Ultomiris

Ultomiris (ravulizumab), 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 US, EU and Japan for the treatment of certain adults with generalised myasthenia gravis.

Ultomiris is also approved in the US, EU and Japan for the treatment of certain adults with paroxysmal nocturnal haemoglobinuria (PNH) and for certain children with PNH in the US and EU.

Additionally, Ultomiris is approved in the US, EU and Japan for certain adults and children with atypical haemolytic uraemic syndrome to inhibit complement-mediated thrombotic microangiopathy.

Further, Ultomiris is approved in the EU and Japan for the treatment of certain adults with neuromyelitis optica spectrum disorder (NMOSD).

As part of a broad development programme, Ultomiris is being assessed for the treatment of additional haematology and neurology indications.

Alexion

Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for more than 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialisation of life-changing medicines. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on haematology, nephrology, neurology, metabolic disorders, cardiology, and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. Please visit https://alexion.com/worldwide/canada.

AstraZeneca

AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries, and its innovative medicines are used by millions of patients worldwide. Please visit www.astrazeneca.ca and follow the Company on X @AstraZenecaCA.

References

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SOURCE Alexion AstraZeneca Rare Disease

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