- Company name
- BioMarin
- Sector
- Rare disease biotech
- About
- 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, BMN 440 (ALE1), an oral medicine for hypophosphatasia, and BMN 450, a long-acting enzyme therapy for Fabry disease that patients inject under the skin.
- Thesis
- 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 its 2026 guidance of $975 million to $1,025 million, which depends on a large international order that moved into Q4 2026, and keep growing in 2027 with Yuviwel on the market. The FDA must give full approval in achondroplasia by the target date of February 28, 2027, and approve VOXZOGO for hypochondroplasia by the target date of May 30, 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 next evidence comes with the Q4 2026 report and 2027 guidance in early 2027 and with the two FDA decisions. If these checks pass, the gap can close over the next three to five quarters.
- Risks
- VOXZOGO faces competition from Ascendis's Yuviwel, a weekly CNP injection. VOXZOGO is a daily injection and gave $717 million of revenue in the first nine months of 2026. The royalties from Yuviwel run only until May 2030. More than 75% of VOXZOGO revenue comes from outside the U.S., and large orders from a few international customers make quarterly revenue uneven.
Debt rose after the Amicus purchase and was $4.17 billion at carrying value on September 30, 2026. Interest expense was $56 million in Q3 2026. 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.
- Balance sheet
- Cash: $736M
Debt: $4.2B
Net debt: $3.4B
Interest cover: 1.2×
Undrawn credit line: $600M, to April 2031
Debt due within 12 months: $659M
BioMarin took on debt in 2026 to buy Amicus. At September 30, 2026, debt was $4,174 million at carrying value: $659 million due within 12 months, mainly the $600 million of 1.25% convertible notes due May 2027, and $3,515 million of long-term debt. The long-term debt is $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 at principal, less $15 million of repayments in 2026 and the issuance costs. Cash and cash equivalents were $736 million at September 30, 2026, down from $874 million at June 30, 2026, after BioMarin paid $275 million upfront for Alesta on August 31, 2026. It had no short-term or long-term investments. Its 2026 borrowings of $3,650 million equal the notes and term loans, so the $600 million revolving credit facility, which runs to April 2031, was not used. Interest cover is operating income divided by interest expense for the first nine months of 2026; it is low because of $288 million of acquired research and development charges in Q3 2026. BioMarin targets gross leverage below 2.5 times by mid-2027.
As of Sep 30, 2026.
- Checkpoints
- Nov 2, 2026: Q3 2026 earnings and hypochondroplasia update. Failed. Watch: VOXZOGO 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. Outcome: BioMarin lowered its 2026 VOXZOGO guidance to $975 million to $1,025 million because a large international order moved, although the FDA set a target date of May 30, 2027 for the hypochondroplasia decision.
Feb 2, 2027 (estimate): Q4 2026 results and 2027 guidance. Pending. Watch: VOXZOGO revenue of at least $975 million for 2026 and 2027 guidance with VOXZOGO growth pass; 2026 VOXZOGO revenue below the guidance range or 2027 guidance for lower VOXZOGO revenue fails.
Feb 28, 2027: FDA decision on full approval of VOXZOGO in achondroplasia. Pending. Watch: Full approval by the target date passes; a rejection or a delay fails.
May 30, 2027: FDA decision on VOXZOGO in hypochondroplasia. Pending. Watch: Approval by the target date passes; a rejection or a delay fails.
Jun 30, 2027 (estimate): Gross leverage below 2.5 times. Pending. Watch: Gross leverage below 2.5 times by mid-2027 and repayment of the May 2027 convertible notes pass; higher leverage fails.
- Recent events
- Nov 2, 2026: BioMarin reported Q3 2026 results. Revenue grew 32% to $1,025 million, its first quarter above $1 billion, with $152 million from GALAFOLD and $49 million from POMBILITI + OPFOLDA. VOXZOGO revenue grew 12% to $245 million. GAAP diluted EPS was a loss of $0.63 and non-GAAP diluted EPS was $0.23, after the Alesta research charges. BioMarin lowered its 2026 guidance to total revenue of $3,800 million to $3,900 million, VOXZOGO revenue of $975 million to $1,025 million and non-GAAP EPS of $2.80 to $3.10, because the timing of orders from one international customer is uncertain and because of the Alesta and BMN 450 charges. It licensed BMN 450, a Fabry disease medicine. The FDA set May 30, 2027 as the target date for its decision on VOXZOGO in hypochondroplasia.
Oct 6, 2026: BioMarin 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.
Sep 24, 2026: BioMarin 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.
Sep 15, 2026: BioMarin 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.
Aug 31, 2026: BioMarin completed its purchase of Alesta Therapeutics for $275 million upfront, plus milestone payments. It gained ALE1, an oral medicine in a Phase 1/2a study, which can become the first oral therapy for hypophosphatasia.
Aug 30, 2026: BioMarin agreed binding terms with Ascendis Pharma to settle all patent disputes about Yuviwel, including the case before the U.S. International Trade Commission.
Aug 6, 2026: BioMarin reported Q2 2026 results. Revenue grew 20% to $990 million, and VOXZOGO revenue grew 14% to $253 million. Non-GAAP EPS was $1.20. BioMarin raised its 2026 guidance to total revenue of $3,875 million to $3,925 million and VOXZOGO revenue of $1,000 million to $1,050 million. It submitted VOXZOGO for hypochondroplasia to the FDA and stopped the development of BMN 401.
May 4, 2026: BioMarin reported Q1 2026 results with revenue of $766 million. It raised its 2026 revenue guidance to $3.825 billion to $3.925 billion to include the Amicus products.
Apr 27, 2026: BioMarin completed the purchase of Amicus Therapeutics for total consideration of $5.3 billion. It paid with cash, $850 million of 5.5% notes due 2034 and $2.8 billion of secured term loans.