Ultragenyx Pharmaceutical RARE announced that the European Commission (EC) has expanded the eligible patient population for one of its marketed products, Evkeeza (evinacumab), an ANGPTL3 inhibitor. Per the latest approval, Evkeeza is now indicated as an adjunct to diet and other lipid-lowering therapies to treat children aged six months and older with homozygous familial hypercholesterolemia (HoFH). The EC’s approval makes Evkeeza the first HoFH medicine indicated for this pediatric population in the EU.

HOFH is an inherited disease that causes dangerously high levels of low-density lipoprotein cholesterol (LDL-C), or bad cholesterol. It affects approximately 1,600 people in the EU and 1 in 300,000 globally. Please note that Evkeeza was initially approved in the EU in 2021 as an adjunct to diet and other LDL-C-lowering therapies to treat adult and adolescent patients aged 12 years and older with HoFH. The drug’s label was later expanded in 2023 to include the treatment of children aged 5-11 years old with HoFH. The drug is also approved in multiple other geographies for the HoFH indication like the United States, United Kingdom and Canada, among others.

In the past three months, shares of Ultragenyx have plunged 20% compared with the industry’s 9.4% decline.

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More on Evkeeza’s Label Update for Younger HoFH Patients

Please note that the EC’s approval forEvkeeza to treat HoFH patients aged six months and older follows the positive recommendation received from its advisory committee in November 2024.

Evkeeza's efficacy in pediatric patients aged six months to less than five years with HoFH has been predicted through model-based extrapolation. These analyses suggest that this age group is expected to experience a similar or greater reduction in LDL-C at week 24 compared to adults when given a 15 mg/kg dose of the drug, every 4 weeks. Additionally, supportive data for five HoFH patients aged 1 to 4 years who receive Evkeeza via compassionate use also showed a clinically meaningful LDL-C reduction consistent with 5 years or older pediatric patients in clinical studies. The safety profile for this younger age group is expected to align with that of older pediatric patients, with no new safety concerns identified.

Please note that Evkeeza was originally discovered and developed by Regeneron Pharmaceuticals REGN. In 2022, Ultragenyx signed a licensing agreement with Regeneron for Evkeeza to treat HoFH. Per the deal, RARE obtained the rights to develop, commercialize and distribute Evkeeza outside the United States. The regions include the European Economic Area. The collaboration with Regeneron for Evkeeza gives Ultragenyx a fourth-approved product that adds to the top line. However, REGN solely commercializes Evkeeza in the United States.

RARE’s Other Marketed Products

Ultragenyx’s commercial portfolio comprises three other medicines — Mepsevii (vestronidase alfa), Crysvita (burosumab) and Dojolvi (UX007).

Mepsevii is approved for the treatment of children and adults with Mucopolysaccharidosis VII, also known as Sly syndrome.

Crysvita, an antibody targeting fibroblast growth factor 23, is approved in the United States for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients. Crysvita received European conditional marketing authorization in February 2018 for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older, and adolescents with growing skeletons. Crysvita has also been approved for another indication, tumor-induced osteomalacia, in adults and pediatric patients aged two years and older by the FDA in June 2020.

Dojolvi was approved by the FDA in 2020 for the treatment of pediatric and adult patients for all forms of long-chain fatty acid oxidation disorders with a molecularly confirmed diagnosis. Dojolvi is the first FDA-approved therapy for these lifelong and life-threatening genetic disorders and is now available to patients in the United States.

Ultragenyx Pharmaceutical Inc. Price and Consensus

Ultragenyx Pharmaceutical Inc. price-consensus-chart | Ultragenyx Pharmaceutical Inc. Quote

RARE’s Zacks Rank & Stocks to Consider

Ultragenyx currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks from the sector are Castle Biosciences CSTL and Vanda Pharmaceuticals VNDA, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 30 days, 2024 estimates for Castle Biosciences’ earnings per share have remained constant at 34 cents. During the same timeframe, loss per share estimates for 2025 have remained constant at $1.84. In three months, shares of Castle Biosciences have lost 2.2%.

CSTL’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 172.72%.

In the past 30 days, estimates for Vanda Pharmaceuticals’ 2024 loss per share have remained constant at 32 cents. Estimates for 2025 loss per share have remained constant at 35 cents during the same timeframe. In three months, Vanda’s shares have gained 6.7%.

VNDA has delivered a negative earnings surprise of 45.79% in the past four quarters.

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