BioMarin

BioMarin develops and sells medicines for rare genetic diseases. Its largest single product is VOXZOGO, a daily injection that increases growth in children with achondroplasia, a form of dwarfism. Its Metabolic Conditions medicines treat enzyme and metabolic disorders: VIMIZIM, NAGLAZYME, PALYNZIQ, BRINEURA, ALDURAZYME and KUVAN.

On April 27, 2026, it bought Amicus Therapeutics and added GALAFOLD for Fabry disease and POMBILITI + OPFOLDA for Pompe disease. BioMarin makes money from product sales around the world, and some products sell at high prices to small numbers of patients. Its pipeline includes BMN 333, a long-acting CNP for achondroplasia, and ALE1, an oral medicine for hypophosphatasia.

The market fears that VOXZOGO, BioMarin's main growth driver, will lose patients to Ascendis's Yuviwel, a weekly CNP injection, while VOXZOGO is a daily injection. It also doubts the value of the Amicus purchase, which BioMarin paid for mostly with $3.65 billion of new debt, and it notes pipeline setbacks: BMN 401 failed one of two main goals in its pivotal study, and BioMarin stopped VOXZOGO in Noonan syndrome. The settlement with Ascendis reduces this fear in part, because Ascendis pays royalties of 20% of U.S. Yuviwel sales until May 2030.

For the gap to close, four things must be true. VOXZOGO must reach at least $1 billion of revenue in 2026 and keep growing in 2027 with Yuviwel on the market. The FDA must approve VOXZOGO for hypochondroplasia, for a launch in 2027, and give full approval in achondroplasia by the target date of February 28, 2027. GALAFOLD and POMBILITI + OPFOLDA must keep growing, with about $220 million of non-GAAP cost savings by 2028.

Gross leverage must fall below 2.5 times by mid-2027, and BioMarin must repay the $600 million of convertible notes due in May 2027. The first evidence comes with the Q3 2026 report, estimated for about October 26, 2026, which will also update the hypochondroplasia application. If these checks pass, the gap can close over the next three to five quarters.

1. Oct 26, 2026 · est.Pending

   Q3 2026 earnings and hypochondroplasia updateVOXZOGO revenue on track for at least $1 billion in 2026 and an FDA review of the hypochondroplasia application on schedule pass; lower VOXZOGO guidance or a delay fails.
2. Feb 28, 2027Pending

   FDA decision on full approval of VOXZOGO in achondroplasiaFull approval by the target date passes; a rejection or a delay fails.
3. Jun 30, 2027 · est.Pending

   Gross leverage below 2.5 timesGross leverage below 2.5 times by mid-2027 and repayment of the May 2027 convertible notes pass; higher leverage fails.

Every passed and failed checkpoint across all picks

BioMarin took on debt in 2026 to buy Amicus. At June 30, 2026, debt was $4.25 billion at principal: $600 million of 1.25% convertible notes due May 2027, $850 million of 5.5% senior notes due 2034, an $800 million term loan A due April 2031 and a $2.0 billion term loan B due April 2033. Read more

• VOXZOGO faces competition from Ascendis's Yuviwel, a weekly CNP injection. VOXZOGO is a daily injection and gave $472 million of revenue in the first half of 2026. The royalties from Yuviwel run only until May 2030.
• Debt rose to $4.25 billion after the Amicus purchase, and interest expense on the new debt is about $200 million a year. The $600 million of convertible notes are due in May 2027.
• BioMarin must integrate Amicus and grow GALAFOLD and POMBILITI + OPFOLDA. It targets peak sales of about $1.4 billion and $1.2 billion and about $220 million of non-GAAP cost savings by 2028. Slower growth would reduce the return on the deal.
• The pipeline had setbacks. BMN 401 failed one of two main goals in its pivotal study, BioMarin stopped VOXZOGO in Noonan syndrome, and it no longer markets ROCTAVIAN. Regulators may also delay or reject the hypochondroplasia application.

• October 6, 2026BioMarin said that the Alesta purchase adds about $283 million of acquired research and development charges in Q3 2026, which lowers Q3 GAAP and non-GAAP EPS by about $1.50. For the full year 2026, the charges are about $320 million. BioMarin will update its 2026 guidance with the Q3 report.
• September 24, 2026BioMarin and Ascendis Pharma signed the final settlement and license agreement. Ascendis pays BioMarin royalties of 20% of U.S. net sales of Yuviwel, from the first sale, and 18% of net sales in the European Union, Brazil and South Korea, until May 2030. The parties dismiss all pending patent cases.
• September 15, 2026BioMarin stopped the development of VOXZOGO for children with Noonan syndrome. It said the decision was based on study feasibility and the treatment landscape, and not on safety or efficacy.