Evkeeza is a first-in-class medicine approved by Health Canada, the U.S. Food and Drug Administration (FDA) and European Commission (EC) to treat an ultrarare, inherited form of high cholesterol

TORONTO, Sept. 25, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare genetic diseases, today announced the approval of Evkeeza^® (evinacumab) in Canada. Evkeeza^® was approved by Health Canada as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients aged 5 years and older with homozygous familial hypercholesterolemia (HoFH) and will be commercially available in Canada effective 25-November-2023. Evkeeza is a first-of-its-kind treatment for HoFH and is the first monoclonal antibody inhibiting the angiopoietin-like 3 protein (ANGPTL3). Evkeeza is delivered via 60-minute intravenous infusion every 4 weeks. The treatment is now available to prescribe for patients with HoFH in Canada.

“The approval and launch of Evkeeza in Canada for the treatment of homozygous familial hypercholesterolemia exemplifies our commitment to bring innovative therapies for people living with rare and ultrarare genetic diseases,” said Monty Keast, Vice President and General Manager at Ultragenyx Canada. “We look forward to working collaboratively with health care providers, the HoFH patient community and payers from across the country to make this potentially life-changing therapy accessible to people living with this serious disease.”

“HoFH is a severe atherosclerotic disease associated with premature cardiovascular morbidity, which most often does not adequately respond to cholesterol-lowering treatment currently available,” said Prof. Daniel Gaudet, Lipidologist and Director of the Severe Lipid Disorders Unit at the Community Genetic Medicine Center, Department of Medicine, Université de Montréal. “Our experience in recent years in phase 2 and 3 clinical studies, clearly demonstrates that in combination with diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies, evinacumab represents an effective option to substantially lower the levels of LDL-cholesterol and other atherogenic lipoproteins in HoFH patients.”

About Homozygous Familial Hypercholesterolemia (HoFH)

HoFH, also known as homozygous FH, is the most severe form of inherited hypercholesterolemia. This ultrarare disease affects 1 in 300,000 people worldwide. There is a founder effect in the French-Canadian population with a prevalence estimated to be 1 in 250,000 people. Homozygous familial hypercholesterolemia runs in families and is usually passed down by both father and mother. People with this condition have extremely high levels of LDL-cholesterol (‘bad cholesterol’) from birth. Such high levels can lead to heart attacks, heart valve disease, or other problems at an early age. For more information about HoFH please go to www.hofhdisease.ca.

About Evkeeza^® (evinacumab)

Evinacumab, the active substance in Evkeeza, attaches to a protein in the body called ANGPTL3 and blocks its effects. ANGPTL3 is involved in controlling cholesterol levels and blocking its effect reduces the level of cholesterol in the blood. Evkeeza is delivered via an infusion every 4 weeks.

Evkeeza is approved by Health Canada as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients aged 5 years and older with homozygous familial hypercholesterolemia (HoFH). The effects of Evkeeza on cardiovascular morbidity and mortality have not been determined. Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) discovered and developed Evkeeza, and solely commercializes the product in the U.S. Ultragenyx is responsible for commercialization efforts for Evkeeza in countries outside of the U.S.

Indication

In Canada, EVKEEZA^® (evinacumab for injection) is indicated as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients aged 5 years and older with homozygous familial hypercholesterolemia (HoFH).

The effects of Evkeeza on cardiovascular morbidity and mortality have not been determined.

IMPORTANT SAFETY INFORMATION FOR EVKEEZA^® (evinacumab) INFUSION

To help avoid side effects and ensure proper use, talk to your healthcare professional before you take Evkeeza. Hypersensitivity reactions, including anaphylaxis and infusion reactions (e.g., infusion site pruritus), have been reported with Evkeeza. If signs or symptoms of serious hypersensitivity reactions occur, discontinue treatment with Evkeeza, treat according to the standard of care, and monitor until signs and symptoms resolve.

The most common adverse reactions to Evkeeza were nasopharyngitis (13.7% vs. 13.0% in placebo), influenzalike illness (7.7% vs. 5.6% in placebo), dizziness (6.0% vs. 0% in placebo), back pain (5.1% vs. 3.7% in placebo), and nausea (5.1% vs. 1.9% in placebo). Fatigue (15%) was identified as an adverse reaction to Evkeeza for pediatric patients aged ≥ 5 to 11 years only.

Who should not use Evkeeza?

Evkeeza may not be used if you are allergic to evinacumab or to any of the ingredients in Evkeeza.

If you are pregnant, think you might be pregnant, or plan to become pregnant, ask your healthcare professional for advice before taking Evkeeza. Evkeeza may harm your unborn baby. Tell your healthcare professional if you become pregnant while using Evkeeza. For people who are able to become pregnant:

• Your healthcare professional may do a pregnancy test before you start treatment with Evkeeza.
• You should use an effective method of birth control during treatment and for at least 5 months after the last dose of Evkeeza. Talk to your healthcare professional about birth control methods that you can use during this time.
• If you are breastfeeding or plan to breastfeed, ask your healthcare professional for advice before you are given Evkeeza. It is not known if Evkeeza passes into your breast milk. You and your healthcare professional should decide if you will receive Evkeeza or breastfeed.
  

You may report side effects via email to ultragenyx@primevigilance.com.

Please see PRODUCT MONOGRAPH INCLUDING PATIENT MEDICATION INFORMATION for more information.

About Ultragenyx Pharmaceutical Inc.

Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit ultragenyx.ca.

Contacts

Ultragenyx Pharmaceutical, Inc.
Media
Jeff Blake
+1-415-612-7784
media@ultragenyx.com

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