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Can RARE's Approved Product Portfolio Drive Its Next Growth Phase?
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Key Takeaways
Ultragenyx generated $350 million in first-half 2026 revenues, up 14%, led by Crysvita's $249 million.
Evkeeza revenues surged 56% to $39 million, supported by new-country launches and early access.
Genglycos and Fayuvi offer Ultragenyx opportunities to expand its revenue base and drive future growth.
Ultragenyx Pharmaceutical (RARE - Free Report) is broadening its commercial portfolio of treatments for rare and ultra-rare diseases. The company generated $350 million in total revenues in the first half of 2026, up 14% year over year, supported by higher revenues across its marketed products. With two additional gene therapies now approved in the United States, Ultragenyx has more opportunities to expand its revenue base and reduce its dependence on established products.
Crysvita (burosumab) remains the company’s primary revenue driver. Approved for X-linked hypophosphatemia and tumor-induced osteomalacia, the drug generated $249 million in first-half 2026 revenues, up 11% year over year, accounting for roughly 71% of total revenue. Dojolvi (triheptanoin), approved for long-chain fatty acid oxidation disorders, generated $45 million, up 13%. Meanwhile, Mepsevii (vestronidase alfa), approved for mucopolysaccharidosis VII, generated $17 million, broadly unchanged from the prior-year period. These established therapies provide a commercial foundation, although Crysvita’s large contribution leaves overall growth exposed to its performance.
Evkeeza (evinacumab) provides another growth avenue. Ultragenyx commercializes the Regeneron-partnered drug outside the United States for homozygous familial hypercholesterolemia (HoFH). Evkeeza generated $39 million in first-half revenues, up 56% year over year, supported by new-country launches and early access. Its international expansion complements the company’s established rare-disease therapies and provides RARE with an incremental revenue stream.
Ultragenyx has also expanded its portfolio through two recent FDA approvals. Genglycos (pariglasgene brecaparvovec) received accelerated approval in August 2026 for adults and children aged eight years and older with glycogen storage disease type Ia. In September, Fayuvi (rebisufligene etisparvovec) received full approval as the first treatment for Sanfilippo syndrome type A in eligible pediatric patients. Ultragenyx also agreed to sell the priority review voucher awarded with Genglycos’ approval for $210 million. The transaction is expected to provide additional non-dilutive capital upon closing, although 20% of gross proceeds is payable to the NIH under an existing license agreement.
Overall, the expanding portfolio could drive RARE’s next growth phase, but the commercial impact of the new gene therapies will depend on treatment-center access, reimbursement and patient uptake. Ultragenyx’s 2026 revenue guidance of $730-$760 million excludes potential revenues from new product launches, making their contribution an additional opportunity rather than a guaranteed source of growth.
RARE Faces Competition Across Rare-Disease Markets
Ultragenyx’s competitive landscape includes established biopharmaceutical company Amgen (AMGN - Free Report) and rare-disease-focused biotechnology company Denali Therapeutics (DNLI - Free Report) .
Amgen markets Repatha (evolocumab), an LDL-cholesterol-lowering treatment approved for adults and pediatric patients aged 10 years and older with HoFH. Repatha’s use in this patient population places it in the same treatment market as Evkeeza. Repatha generated $1.83 billion in first-half 2026 sales, up 35% year over year across all indications. Although Repatha and Evkeeza work through different mechanisms and can serve distinct roles in treatment, Amgen’s strong commercial presence creates a competitive consideration for Evkeeza.
Denali is advancing zafinofusp alfa (DNL126), an ETV-enabled investigational enzyme replacement therapy for Sanfilippo syndrome type A. Early-stage data showed substantial reductions in disease-related biomarkers upon treatment with the candidate. Based on such results, DNLI is preparing to initiate a global phase III confirmatory study of zafinofusp alfa for Sanfilippo syndrome type A. Denali expects a biologics license application submission and potential accelerated approval for zafinofusp alfa for Sanfilippo syndrome type A in 2027. Successful clinical progress could eventually introduce a competing treatment option for Fayuvi.
Year to date, RARE shares have lost 35.1% against the industry’s 1.8% growth. Ultragenyx has also underperformed the sector and the S&P 500 during the same time frame, as seen in the chart below.
RARE Stock Underperforms the Industry, Sector & the S&P 500
Image Source: Zacks Investment Research
Ultragenyx is trading at a discount to the industry, as seen in the chart below. Going by the price/sales ratio, the company’s shares currently trade at 2.12, which is less than 2.39 for the industry. The stock is trading significantly below its five-year mean of 7.74.
RARE Stock Valuation
Image Source: Zacks Investment Research
Loss estimates for 2026 have narrowed from $4.08 to $3.95 per share over the past 60 days. During the same time frame, RARE’s 2027 loss per share estimates have narrowed from 75 cents to 13 cents.
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Image: Bigstock
Can RARE's Approved Product Portfolio Drive Its Next Growth Phase?
Key Takeaways
Ultragenyx Pharmaceutical (RARE - Free Report) is broadening its commercial portfolio of treatments for rare and ultra-rare diseases. The company generated $350 million in total revenues in the first half of 2026, up 14% year over year, supported by higher revenues across its marketed products. With two additional gene therapies now approved in the United States, Ultragenyx has more opportunities to expand its revenue base and reduce its dependence on established products.
Crysvita (burosumab) remains the company’s primary revenue driver. Approved for X-linked hypophosphatemia and tumor-induced osteomalacia, the drug generated $249 million in first-half 2026 revenues, up 11% year over year, accounting for roughly 71% of total revenue. Dojolvi (triheptanoin), approved for long-chain fatty acid oxidation disorders, generated $45 million, up 13%. Meanwhile, Mepsevii (vestronidase alfa), approved for mucopolysaccharidosis VII, generated $17 million, broadly unchanged from the prior-year period. These established therapies provide a commercial foundation, although Crysvita’s large contribution leaves overall growth exposed to its performance.
Evkeeza (evinacumab) provides another growth avenue. Ultragenyx commercializes the Regeneron-partnered drug outside the United States for homozygous familial hypercholesterolemia (HoFH). Evkeeza generated $39 million in first-half revenues, up 56% year over year, supported by new-country launches and early access. Its international expansion complements the company’s established rare-disease therapies and provides RARE with an incremental revenue stream.
Ultragenyx has also expanded its portfolio through two recent FDA approvals. Genglycos (pariglasgene brecaparvovec) received accelerated approval in August 2026 for adults and children aged eight years and older with glycogen storage disease type Ia. In September, Fayuvi (rebisufligene etisparvovec) received full approval as the first treatment for Sanfilippo syndrome type A in eligible pediatric patients. Ultragenyx also agreed to sell the priority review voucher awarded with Genglycos’ approval for $210 million. The transaction is expected to provide additional non-dilutive capital upon closing, although 20% of gross proceeds is payable to the NIH under an existing license agreement.
Overall, the expanding portfolio could drive RARE’s next growth phase, but the commercial impact of the new gene therapies will depend on treatment-center access, reimbursement and patient uptake. Ultragenyx’s 2026 revenue guidance of $730-$760 million excludes potential revenues from new product launches, making their contribution an additional opportunity rather than a guaranteed source of growth.
RARE Faces Competition Across Rare-Disease Markets
Ultragenyx’s competitive landscape includes established biopharmaceutical company Amgen (AMGN - Free Report) and rare-disease-focused biotechnology company Denali Therapeutics (DNLI - Free Report) .
Amgen markets Repatha (evolocumab), an LDL-cholesterol-lowering treatment approved for adults and pediatric patients aged 10 years and older with HoFH. Repatha’s use in this patient population places it in the same treatment market as Evkeeza. Repatha generated $1.83 billion in first-half 2026 sales, up 35% year over year across all indications. Although Repatha and Evkeeza work through different mechanisms and can serve distinct roles in treatment, Amgen’s strong commercial presence creates a competitive consideration for Evkeeza.
Denali is advancing zafinofusp alfa (DNL126), an ETV-enabled investigational enzyme replacement therapy for Sanfilippo syndrome type A. Early-stage data showed substantial reductions in disease-related biomarkers upon treatment with the candidate. Based on such results, DNLI is preparing to initiate a global phase III confirmatory study of zafinofusp alfa for Sanfilippo syndrome type A. Denali expects a biologics license application submission and potential accelerated approval for zafinofusp alfa for Sanfilippo syndrome type A in 2027. Successful clinical progress could eventually introduce a competing treatment option for Fayuvi.
RARE’s Stock Price, Valuation & Estimate Movements
Year to date, RARE shares have lost 35.1% against the industry’s 1.8% growth. Ultragenyx has also underperformed the sector and the S&P 500 during the same time frame, as seen in the chart below.
RARE Stock Underperforms the Industry, Sector & the S&P 500
Ultragenyx is trading at a discount to the industry, as seen in the chart below. Going by the price/sales ratio, the company’s shares currently trade at 2.12, which is less than 2.39 for the industry. The stock is trading significantly below its five-year mean of 7.74.
RARE Stock Valuation
Loss estimates for 2026 have narrowed from $4.08 to $3.95 per share over the past 60 days. During the same time frame, RARE’s 2027 loss per share estimates have narrowed from 75 cents to 13 cents.
RARE Estimate Movement
Ultragenyx currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.