Freedom reiterates its Buy rating on BioMarin shares, with 58% upside potential

Freedom analysts reiterated a “Buy” rating for shares of biotechnology company BioMarin Pharmaceutical (BMRN) following the results of the second quarter of 2026. The target price was maintained at $110. With a closing price of $69.55, this implies upside potential of 58.16%.

Amicus integration supported BioMarin’s results

Second-quarter results confirmed an improved financial profile for BioMarin following the integration of Amicus—an American biotechnology company specializing in treatments for rare genetic diseases. The Metabolic Conditions segment continues to show resilient demand: its revenue rose 25% year over year to $695 mln, beating consensus by 6.7%.

PALYNZIQ increased sales by 27% thanks to resilient demand and favorable order timing in the U.S. New products added to the portfolio after the Amicus deal also posted a strong start: GALAFOLD generated $106 mln in revenue, and POMBILITI + OPFOLDA $30 mln. Further growth of GALAFOLD should be supported by improved diagnostics and geographic expansion, while POMBILITI + OPFOLDA should benefit from patient switching and entry into new markets.

At the same time, BioMarin kept its 2026 Metabolic Conditions revenue guidance in the range of $2,725–2,775 bln.

Profitability was significantly above expectations

One of the strongest quarter outcomes was profitability. Non-GAAP gross margin exceeded 80%, and adjusted EBIT margin reached 36.4%. The latter was well above Freedom’s forecast of 26.2%, despite a 30% increase in R&D expenses and a 25% rise in commercial and administrative expenses.

Analysts view the results as confirmation of strong operating leverage after the Amicus integration. Additional support for profitability should come from deal-related cost savings: by 2028, BioMarin expects to achieve $220 mln in annual non-GAAP synergies, with more than 70% of the savings coming from administrative expenses.

Against this backdrop, Freedom raised its BioMarin EPS forecasts for 2026–2028. For 2026, analysts now expect adjusted EPS of $5.05, for 2027—$6.59, and for 2028—$7.04.

VOXZOGO is still coping with intensifying competition

BioMarin’s key drug VOXZOGO also exceeded analysts’ expectations. Its second-quarter sales rose 14% year over year, and the number of patients receiving therapy increased by more than 20%.

The drug’s position among very young children remains especially important: more than half of new U.S. patients were under two years old. In this age category, VOXZOGO remains the only approved treatment for now.

Strong results allowed BioMarin to raise its 2026 VOXZOGO sales guidance from $975 mln–1,025 bln to $1–1.05 bln. However, Freedom analysts do not yet view the guidance raise as grounds to improve their mid-term outlook, since quarterly sales are materially affected by the schedule of international shipments—overseas markets account for about 75% of the drug’s sales.

YUVIWEL remains the main risk for VOXZOGO

Competitive pressure is gradually increasing from Ascendis Pharma’s YUVIWEL. By the end of the second quarter, more than 170 children with achondroplasia in the U.S. had started therapy, and about 60% of them had previously been on VOXZOGO. According to BioMarin, about 10% of the company’s U.S. patients switched to YUVIWEL within five months after the competitor entered the market.

One advantage of YUVIWEL is its weekly dosing regimen versus VOXZOGO’s daily injections. For now, the impact of competition is limited in part because the U.S. provides only about 25% of VOXZOGO sales. However, the expected approval of YUVIWEL in Europe at the end of 2026 could increase pressure. Therefore, Freedom forecasts VOXZOGO sales growth will slow to 5% in 2027.

A new indication could expand the VOXZOGO market

To offset intensifying competition, BioMarin is counting on expanding VOXZOGO’s indications to hypochondroplasia. In a Phase III trial, the drug delivered a statistically significant increase in annualized growth velocity of 2.3 cm versus placebo after 52 weeks of therapy.

If approved, VOXZOGO could become the first treatment for children with hypochondroplasia. BioMarin estimates the potential patient population in the U.S. alone at about 14 thousand people. A potential launch for the new indication is expected in 2027 and could become an additional sales driver.

Why Freedom recommends buying BioMarin shares

Analysts maintain a positive view on BMRN due to patient growth in Metabolic Conditions, improving profitability, and progress in the product portfolio. According to Freedom’s forecast, BioMarin’s revenue will increase from $3.22 bln in 2025 to $3.9 bln in 2026, $4.45 bln in 2027, and $4.7 bln in 2028. Adjusted operating profit over this period could grow from $750 mln to $1.79 bln.

The main risk remains the dependence of further growth on VOXZOGO and intensifying competition. Additional risks cited by analysts include a potential weakening of demand for Metabolic Conditions drugs, as well as failures or delays in pipeline development.

Despite these factors, Freedom maintains its “Buy” rating and $110 target price for BMRN. From $69.55, this corresponds to potential return of 58.16%.

What is happening with other biotechnology companies

Earlier, Freedom presented an investment idea on shares of Upstream Bio (UPB), which develops drugs to treat severe inflammatory respiratory diseases. Analysts set a target price of $11 with the shares trading around $7.30, corresponding to upside potential of 50.7%. The company’s main driver remains Verekitug, a drug for severe asthma and chronic rhinosinusitis: Freedom estimates its potential annual sales at more than $1 bln. Further share performance largely depends on progress of the clinical program and consultations with the U.S. regulator on future registration trials. 

At the same time, not all companies in the biotechnology and healthcare sector show similarly positive prospects. Shares of BioMérieux (BIOX) fell after the French manufacturer of laboratory diagnostics solutions lowered its 2027–2028 outlook. The company expects operating profit in the medium term to be 4–9% below its previous forecasts. According to Freedom analyst Vladimir Chernov, this points to a slowdown in growth in key business areas, primarily molecular diagnostics. Since April, BioMérieux shares had already lost about 20% by the time the news was published. 

Not an individual investment recommendation.