Health Canada Extends the Approval of Evkeeza® (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH)

TORONTO, July 14, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultra-rare genetic diseases, today announced that Health Canada has extended the approval of Evkeeza^® (evinacumab) as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies to treat children aged 6-months and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza, an angiopoietin-like 3 (ANGPTL3) inhibitor initially received Health Canada approval in September 2023 as an adjunct to diet and other LDL-C lowering therapies for the treatment of adult and pediatric patients aged 5 years and older with HoFH, a disease associated with dangerously high levels of LDL-C.

"Early detection and prompt initiation of effective treatment are essential to prevent the devastating consequences of very high cholesterol that begins at a very young age for patients with HoFH. However, conventional treatment options are challenging for infants and children,” stated Dr. Brian McCrindle, a pediatric lipid specialist at The Hospital for Sick Children, University of Toronto. “This extended approval will enable the very early and effective lowering of lipid levels essential for these vulnerable patients."

The pharmacokinetics and efficacy of the drug in pediatric patients aged 6 months to less than 5 years with HoFH have been predicted from a model-based extrapolation analysis. Results of these analyses show that pediatric patients aged 6 months to less than 5 years are predicted to experience a similar or higher magnitude of percent change in LDL-C at week 24 compared to older pediatric patients and adults, when receiving a 15 mg/kg dose every 4 weeks. In addition, supportive data for five patients who initiated treatment between 1 and 4 years of age via compassionate use showed a clinically meaningful reduction of LDL-C consistent with that observed in patients aged 5 years or older in clinical studies. Based on the currently available data, the safety profile in pediatric patients aged 6-months to 5 years old is expected to be similar to the safety profile in older pediatric patients. No new safety concerns have been identified in the compassionate use program.

"HoFH is a severe, life-threatening condition that, if left untreated, can lead to heart disease and death as early as childhood. This makes diagnosing it and reducing the resulting high LDL-C levels an urgent need,” said Monty Keast, Vice President and General Manager at Ultragenyx Canada. “We are proud to be able to provide children as young as 6-months old living with HoFH a medicine that could reduce LDL-C levels and will work collaboratively with healthcare providers and payers across the country to ensure it is accessible for families.”

Evkeeza is reimbursed and commercially available to prescribe for appropriate patients with HoFH in Canada via private drug plans and through the public drug program in Quebec, the UK, U.S., Italy, Japan, the Netherlands, Spain and Luxembourg. It is also available via early access programs in 13 additional countries including Austria and France.

About Homozygous Familial Hypercholesterolemia (HoFH)

HoFH is a devastating form of inherited hypercholesterolemia, affecting 1 in 300,000 people globally and 1 in 275,000 in the French Canadian population. HoFH occurs when two copies of the familial hypercholesterolemia (FH)-causing genes are inherited, one from each parent, resulting in dangerously high levels (>10 mmol/L) of LDL-C, or bad cholesterol. Patients with HoFH are at risk for premature atherosclerotic disease and cardiac events at an early age. For more information about HoFH please go to .

About Evkeeza (evinacumab)

Evinacumab, the active substance in the medicine, 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. The medicine is delivered via an infusion every month (4 weeks).

Regeneron Pharmaceuticals, Inc. discovered and developed Evkeeza, and commercializes the product in HoFH in the U.S. under the generic name evinacumab-dgnb, with dgnb as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the FDA. Ultragenyx is responsible for development and commercialization efforts in countries outside of the U.S.

What is Evkeeza used for?

Evkeeza is used to treat adults and children aged 6 months and older with very high cholesterol caused by a condition called homozygous familial hypercholesterolemia (HoFH). Evkeeza is used with a low-fat diet and other medicines to bring down cholesterol levels.

Do not use Evkeeza if:

• Allergic to evinacumab or to any of the ingredients in this medicine.

To help avoid side effects and ensure proper use, a healthcare professional should be consulted before taking Evkeeza. Talk about any health conditions or problems, including:

• Allergic reactions (hypersensitivity), including a severe reaction known as anaphylaxis. Symptoms may include swelling of the lips, tongue, or throat that make it difficult to swallow or breathe, and may also include wheezing, feeling dizzy, or fainting. If any of these symptoms are noticed, stop taking Evkeeza and get immediate medical help.

• If pregnant, might be pregnant, or plan to become pregnant, ask a healthcare professional for advice before taking Evkeeza. Evkeeza may harm an unborn baby. Tell a healthcare professional if become pregnant while using Evkeeza.

• If breastfeeding or plan to breastfeed, ask a healthcare professional for advice before being given Evkeeza. It is not known if Evkeeza passes into breast milk.

Tell a healthcare professional about all the medicines taken, including any drugs, vitamins, minerals, natural supplements, or alternative medicines.

What are the possible side effects from using Evkeeza?

These are not all the possible side effects when taking Evkeeza. If any side effects not listed here are experienced, tell a healthcare professional. • Abdominal pain • Back pain • Constipation • Decreased energy • Dizziness • Flu symptoms • Itchiness at the site of the injection • Nausea • Pain in legs or arms • Runny nose • Fatigue (for patients aged 5-11 years old) • Sore throat or sinus infection

Reporting Side Effects:

Any suspected side effects associated with the use of health products can be reported to Health Canada by:

• Visiting the Web page on Adverse Reaction Reporting () for information on how to report online, by mail or by fax; or

• Calling toll-free at 1-866-234-2345.

NOTE: Contact a health professional if information is needed about how to manage side effects. The Canada Vigilance Program does not provide medical advice.

Please see 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 ultra-rare 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 the company's website at: .

Ultragenyx Forward-Looking Statements and Use of Digital Media

Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause the Company’s clinical development programs, commercial success of its products and product candidates, continued collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, risks related to reliance on third party partners to conduct certain activities on the Company’s behalf; the potential for any license or collaboration agreement, including Ultragenyx’s collaboration agreement with Regeneron to commercialize Evkeeza outside of the United States, to be terminated; uncertainty and potential delays related to clinical drug development; uncertainties and unpredictability of obtaining regulatory approval for the Company’s product candidates and the scope of such potential regulatory approval; smaller than anticipated market opportunities for the Company’s products and product candidates; fluctuations in buying or distribution patterns by distributors and specialty pharmacies; competition to the Company’s products and product candidates; potential undesirable or serious side effects from the Company’s products or product candidates; the Company’s ability to effectively manage the expansion of its commercial organization; market acceptance of the Company’s current or future products; uncertainties related to insurance coverage and reimbursement status of newly approved products; manufacturing risks and supply chain disruptions; competition from other therapies or products; and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the Company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and product candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements.

For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 7, 2025, and its subsequent periodic reports filed with the SEC.

In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website () and LinkedIn website ().

Contacts

Ultragenyx Pharmaceutical Inc.

Investors

Joshua Higa

Media

Joey Fleury