Microsoft shares jump after fourth-quarter earnings beat on AI-fueled cloud growth
TORONTO - Health Canada has approved Ultragenyx Pharmaceutical Inc.’s (NASDAQ:RARE) Evkeeza (evinacumab) for children as young as 6 months old with homozygous familial hypercholesterolemia (HoFH), the company announced Monday. The announcement comes as Ultragenyx, currently valued at $2.8 billion, faces market headwinds with its stock down over 25% in the past week. According to InvestingPro analysis, the company maintains strong liquidity with current assets exceeding short-term obligations.
The approval extends the medication’s use as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies for younger patients with HoFH, a rare genetic disease characterized by dangerously high cholesterol levels. Evkeeza was initially approved in Canada in September 2023 for patients aged 5 years and older. Despite recent market challenges, InvestingPro data shows Ultragenyx’s revenue grew by 33% in the last twelve months, reflecting the company’s expanding commercial portfolio.
"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," said Dr. Brian McCrindle, a pediatric lipid specialist at The Hospital for Sick Children, University of Toronto.
The expanded approval was based on model-based extrapolation analysis predicting similar or higher magnitude of LDL-C reduction in younger patients compared to older children and adults when receiving the recommended 15 mg/kg dose every four weeks. Supporting data from five patients who began treatment between ages 1 and 4 via compassionate use showed clinically meaningful LDL-C reduction consistent with results in older patients.
HoFH affects approximately 1 in 300,000 people globally and 1 in 275,000 in the French Canadian population. The condition can lead to premature heart disease and early death if untreated.
Evkeeza, an angiopoietin-like 3 (ANGPTL3) inhibitor, is administered via monthly infusion. The medication is currently reimbursed and commercially available through private drug plans and the public drug program in Quebec, as well as in several other countries including the U.S., UK, Italy, and Japan.
The information in this article is based on a press release from Ultragenyx Pharmaceutical. While the company is currently trading near its 52-week low, seven analysts have revised their earnings estimates upward for the upcoming period. For deeper insights into Ultragenyx’s financial health and growth prospects, including exclusive analysis and additional ProTips, visit InvestingPro, where you’ll find comprehensive research reports covering over 1,400 US stocks.
In other recent news, Ultragenyx Pharmaceutical Inc. announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter for its gene therapy UX111, intended for treating Sanfilippo syndrome type A. The FDA requested additional information concerning chemistry, manufacturing, and controls, which Ultragenyx believes are related to facilities and processes rather than product quality. The company plans to address these observations and resubmit its Biologics License Application, anticipating a review period of up to six months. The FDA acknowledged the robustness of the neurodevelopmental outcome data and supportive biomarker evidence, with no issues cited regarding the clinical data package. Meanwhile, TD Cowen reiterated its Buy rating on Ultragenyx despite disappointing interim results for its setrusumab treatment, maintaining confidence in its efficacy. Morgan Stanley also maintained an Overweight rating, highlighting the progress in the Phase 3 Orbit study for setrusumab. The investment firm noted a 67% fracture reduction in a previous trial, emphasizing the unmet need in osteogenesis imperfecta. These developments reflect ongoing efforts and challenges in Ultragenyx’s therapeutic pipeline.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.