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NOVATO, Calif. - Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company with a market capitalization of $3.5 billion and impressive revenue growth of 33% over the last twelve months, has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration for GTX-102 (apazunersen) as a treatment for Angelman syndrome, the company announced Friday. According to InvestingPro analysis, the company is currently trading below its Fair Value, suggesting potential upside for investors.
The FDA’s decision was based on preliminary clinical evidence from a Phase 1/2 study involving 74 patients aged 4-17 years with full maternal UBE3A gene deletion. According to the company, participants showed developmental gains with improvements across multiple symptom domains when treated for up to three years.
Breakthrough Therapy Designation aims to expedite development and review of drugs for serious conditions that demonstrate substantial improvement over existing therapies.
"FDA Breakthrough Therapy Designation underscores both the urgent need for an effective treatment for patients and families affected by Angelman syndrome and the clinically meaningful results demonstrated to date with GTX-102," said Eric Crombez, chief medical officer at Ultragenyx.
Enrollment in the global Phase 3 Aspire study began in December 2024 and is expected to include approximately 120 children with Angelman syndrome caused by full maternal UBE3A gene deletion. The company also plans to initiate the Aurora study in the second half of 2025 to evaluate GTX-102 across other Angelman syndrome genotypes and ages.
GTX-102 is an antisense oligonucleotide therapy delivered via intrathecal administration designed to reactivate expression of the deficient UBE3A protein. The drug has previously received Orphan Drug Designation, Rare Pediatric Disease Designation, and Fast Track Designation from the FDA.
Angelman syndrome affects approximately 60,000 people in commercially accessible regions and causes cognitive impairment, motor issues, balance problems, and seizures. There are currently no approved therapies for the condition.
The information in this article is based on a company press release.
In other recent news, Ultragenyx Pharmaceutical Inc. reported a 28% increase in total revenue for Q1 2025, reaching $139 million, although the company posted a net loss of $151 million, slightly missing the earnings per share (EPS) forecast. The revenue growth was largely driven by the performance of its flagship product, Crysvita, which alone generated $103 million. William Blair has rated Ultragenyx’s stock as Outperform, with a target price of $65, citing confidence in the company’s ability to sustain its revenue growth. Ultragenyx has projected its 2025 revenue to be between $640 million and $670 million, anticipating a 14-20% increase. The firm is also preparing for significant product launches and expects a transformational year with potential Biologics License Application (BLA) submissions and approvals. Despite high operating expenses impacting profitability, the company’s strong pipeline and strategic commercialization efforts are seen as positive indicators of future performance. The company’s ongoing commitment to addressing rare genetic diseases continues to be a focal point for its financial success and investor interest.
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