| Metric | Oct 29, 2024 FY24 Q3 | Feb 19, 2025 FY24 Q4 | May 1, 2025 FY25 Q1 | Aug 4, 2025 FY25 Q2 | Oct 7, 2025 FY25 Q3 | Jan 12, 2026 FY25 Q4 | May 4, 2026 FY26 Q1 | Aug 6, 2026 FY26 Q2 |
|---|---|---|---|---|---|---|---|---|
| For FY2034 | ||||||||
| Total revenues CAGR | New“Mid-teen compound annual growth rate (CAGR)” | — | — | — | — | — | — | — |
| Total revenues compound annual growth rate (CAGR) | New“Mid-teen” | — | — | — | — | — | — | — |
| For FY2030 | ||||||||
| Peak revenue for GALAFOLD | — | — | — | — | — | — | — | New“approximately $1.4 billion” |
| Peak revenue for POMBILITI + OPFOLDA | — | — | — | — | — | — | — | New“approximately $1.2 billion” |
| GALAFOLD and POMBILITI + OPFOLDA, combined, Non-GAAP Operating Margin | — | — | — | — | — | — | — | New“over 60%” |
| BMN 333 achondroplasia approval target | — | — | New“2030” | — | — | — | — | — |
| For FY2028 | ||||||||
| Cost reductions from Amicus acquisition (GAAP basis) | — | — | — | — | — | — | — | New“approximately $280 million” |
| Cost reductions from Amicus acquisition (Non-GAAP basis) | — | — | — | — | — | — | — | New“approximately $220 million” |
| Cost reductions on a GAAP basis | — | — | — | — | — | — | — | New“approximately $280 million” |
| For FY2027 | ||||||||
| Total Revenues | New“Approximately $4 billion” | Reiterated“approximately $4 billion” | — | — | — | — | — | — |
| Gross leverage target | — | — | — | — | — | — | — | New“below 2.5 times” |
| Operating cash flow | New“More than $1.25 billion” | — | — | — | — | — | — | — |
| Operating cash flows | — | New“more than $1.25 billion” | — | — | — | — | — | — |
| VOXZOGO country access expansion | New“more than 20 additional countries” | — | — | — | — | — | — | — |
| VOXZOGO country access target | — | — | — | New“more than 60 countries” | — | — | — | — |
| VOXZOGO country availability | — | New“more than 60 countries” | — | — | — | — | — | — |
| For FY2026 | ||||||||
| Metabolic Conditions Revenue | — | — | — | — | — | — | — | New“Unchanged” |
| Other Revenues | — | — | — | — | — | — | New“$100 to $125” | Reiterated“$100 to $125” |
| PALYNZIQ Revenue | — | — | — | — | — | — | New“expected to increase year-over-year” | — |
| ROCTAVIAN revenue treatment in 2026 | — | — | — | — | — | New“ROCTAVIAN revenue to be excluded from 2026 guidance” | — | — |
| Total Revenues | — | — | — | — | — | — | New“$3,825 to $3,925” | Narrowed“$3,875 to $3,925” |
| Total Revenues (Full-Year) | — | — | — | — | — | — | New“between $3.825 billion and $3.925 billion” | — |
| Total Revenues Expected in Second Half of 2026 | — | — | — | — | — | — | New“more than 55%” | — |
| Total Revenues Growth Rate (Y/Y, at Midpoint) | — | — | — | — | — | — | New“20%” | — |
| Total Revenues Midpoint Growth (Y/Y) | — | — | — | — | — | — | — | New“21%” |
| VOXZOGO full-year 2026 revenue (CEO quote) | — | — | — | — | — | — | — | New“at least $1 billion” |
| VOXZOGO Revenue | — | — | — | — | — | — | — | New“$1,000 to $1,050” |
| Amicus Acquisition Impact on Non-GAAP Diluted EPS | — | — | — | — | — | — | New“slightly dilutive” | — |
| Combined company tax rate assumption for Non-GAAP Diluted EPS | — | — | — | — | — | — | — | New“22%” |
| Non-GAAP Diluted EPS | — | — | — | — | — | — | New“$4.85 to $5.05” | Raised“$4.90 to $5.10” |
| Non-GAAP Diluted EPS - combined company tax rate assumption | — | — | — | — | — | — | New“22%” | — |
| Non-GAAP Diluted EPS - Weighted-Average Diluted Shares Outstanding assumption | — | — | — | — | — | — | New“approximately 200 million” | — |
| Non-GAAP Diluted EPS (Q2 vs Q1) | — | — | — | — | — | — | New“modestly higher than in Q1” | — |
| Non-GAAP Diluted EPS Midpoint Growth (Y/Y) | — | — | — | — | — | — | — | New“59%” |
| Non-GAAP Diluted EPS Recognized in Second Half of 2026 | — | — | — | — | — | — | New“approximately two-thirds” | — |
| Non-GAAP Operating Margin | New“40%” | Reiterated“40%” | — | — | — | — | — | — |
| Weighted-Average Diluted Shares Outstanding assumption for Non-GAAP Diluted EPS | — | — | — | — | — | — | — | New“approximately 200 million” |
| Enzyme Therapies | — | — | — | — | — | — | New“$2,725 to $2,775” | — |
| Metabolic Conditions | — | — | — | — | — | — | — | New“$2,725 to $2,775” |
| Metabolic Conditions Midpoint Growth (Y/Y) | — | — | — | — | — | — | — | New“31%” |
| VOXZOGO | — | — | — | — | — | — | New“$975 to $1,025” | — |
| VOXZOGO Midpoint Growth (Y/Y) | — | — | — | — | — | — | — | New“11%” |
| For FY2025 | ||||||||
| Revenue ( from Q3'25 Earnings) | — | — | — | — | — | New“$3,150M - $3,200M” | — | — |
| Revenue (Today's Update) | — | — | — | — | — | New“~$3.2B” | — | — |
| Revenue and profitability growth | — | New“Double-Digit” | — | — | — | — | — | — |
| Total Enzyme Therapies revenues | — | — | — | New“expected to remain robust” | — | — | — | — |
| Total Revenues | — | New“$3,100 to $3,200” | Reiterated“$3,100 to $3,200” | Narrowed“$3,125 to $3,200” | — | — | — | — |
| VOXZOGO contribution to full-year 2025 Total Revenues | — | New“$900 million to $950 million” | Reiterated“$900 million to $950 million” | Narrowed“$900 million to $935 million” | — | — | — | — |
| VOXZOGO revenue | — | — | — | New“expected to drive higher VOXZOGO revenue in the second half of 2025 compared to the first half of 2025, and weighted to Q4” | — | — | — | — |
| VOXZOGO Revenue ( from Q3'25 Earnings) | — | — | — | — | — | New“$900M - $935M” | — | — |
| VOXZOGO Revenue (Today's Update) | — | — | — | — | — | New“~$920M” | — | — |
| Charge to GAAP Diluted earnings per share (EPS) | — | — | — | — | New“approximately $1.10” | — | — | — |
| Non-GAAP Diluted EPS | — | New“$4.20 to $4.40” | Reiterated“$4.20 to $4.40” | Raised“$4.40 to $4.55” | — | — | — | — |
| Non-GAAP Diluted EPS assumed Weighted-Average Diluted Shares Outstanding | — | — | New“approximately 200 million” | Reiterated“approximately 200 million” | — | — | — | — |
| Non-GAAP Diluted EPS impact from acquired IPR&D charges | — | — | — | — | New“approximately $1.10” | — | — | — |
| Non-GAAP Operating Margin % | — | New“32% to 33%” | Reiterated“32% to 33%” | Raised“33% to 34%” | — | — | — | — |
| Non-GAAP Operating Margin growth | — | New“14% (3.9 percentage points) Y/Y at the mid-point of guidance” | — | — | — | — | — | — |
| ROCTAVIAN Asset Write Down (Non-GAAP Diluted EPS Impact) (Today's Update) | — | — | — | — | — | New“($0.60) – ($0.64)” | — | — |
| Roctavian asset write-down related to inventory expected to reduce Non-GAAP Diluted EPS | — | — | — | — | — | New“approximately $120M – $125M, or ($0.60) – ($0.64) per share” | — | — |
| Weighted-Average Diluted Shares Outstanding (EPS assumption) | — | New“approximately 200 million” | — | — | — | — | — | — |
| Acquired IPR&D charges (pre-tax) | — | — | — | — | New“approximately $221 million” | — | — | — |
| Operating expenses | — | — | — | New“expected to increase in the second half of 2025 vs. the first half of 2025” | — | — | — | — |
| ROCTAVIAN asset write-downs (total, incl. inventory, facilities, intangible assets, severance costs) | — | — | — | — | — | New“$230M – $260M” | — | — |
| For FY2024 | ||||||||
| Total Revenues | New“$2,790 to $2,825” | — | — | — | — | — | — | — |
| Non-GAAP Diluted EPS | New“$3.25 to $3.35” | — | — | — | — | — | — | — |
| Non-GAAP Operating Margin % | New“26.5% to 27.5%” | — | — | — | — | — | — | — |
| Cost transformation program size | — | New“$500 million” | — | — | — | — | — | — |
| Weighted-Average Diluted Shares Outstanding assumption | New“approximately 200 million” | — | — | — | — | — | — | — |
| For FY2023 | ||||||||
| VOXZOGO Revenue CAGR | — | New“exceeding 25%” | — | — | — | — | — | — |
| No period stated | ||||||||
| Treatments for Skeletal Conditions revenue opportunity | New“greater than $5 billion” | — | — | — | — | — | — | — |
| Non-GAAP Operating Margin | New“low- to mid-40% range” | — | — | — | — | — | — | — |
| Enzyme Therapies long-term growth outlook (CAGR) | — | New“high single-digit CAGR” | — | — | — | — | — | — |
| Interest expense associated with the financing | — | — | — | — | — | — | — | New“approximately $200 million on an annualized basis” |
| Interest expense related to Amicus financing (annualized) | — | — | — | — | — | — | — | New“approximately $200 million” |