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| Name | Indication | Approved | Economics | MOA | Admin | IP | | | Price | 109 | |
| Voxzogo (vosoritide) | Achondroplasia | 11/19/2021 | 1 | | | | | | Shares | 185.848915 | Q322 |
| Palynziq (pegvaliase) | PKU | 5/24/2018 | 1 | | | | | | MC | 20257.531735 | |
| Brineura (cerliponase) | Batten | 4/27/2017 | 1 | | | | | | Cash | 1646 | Q322 |
| Vimizim (elosulfase) | MPS IVA | 2/14/2014 | 1 | ERT | | | | | Debt | 1082 | Q322 |
| Kuvan (sapropterin) | PKU | 12/13/2007 | MRK GY | | Oral | | | | EV | 19693.531735 | |
| Naglazyme (galsulfase) | MPS VI | 5/31/2005 | 1 | ERT | | | | | | | |
| Aldurazyme (laronidase) | MPS I | 4/30/2003 | SNY | ERT | | | | | | | |
| | | | | | | | | | CCO: Jeff Ajer | | |
| | | Phase | | | | | | | | | |
| Roctavian (valoctogene roxaparvovec) | Hemophilia A | 3/31/23 PDUFA | 1 | AAV F8 | IV | | | | | | |
| BMN 331 | HAE | I | | | | | | | | | |
| BMN 307 | PKU | PC | | | | | | | | | |
| BMN 255 | Hyperoxaluria | PC | | | | | | | | | |
| BMN 349 | A1AT | PC | | | | | | | | | |
| BMN 351 | DMD | PC | | | | | | | | | |
| BMN 293/DINA-001 | HCM | PC | | | | | | | | | |
| | | | | | | | | | | | |
| THREE KEY QUESTIONS | | | | | | | | | | | |
| 1. How big can Voxzogo get? $400m? $600m? $800m? | | | | | | | | | | | |
| 2. What will valrox do? Zero? $500m? $1B+? | | | | | | | | | | | |
| 2a. Who else is pursuing hemophilia | | | | | | | | | | | |
| | | | | | | | | | | | |
| 3. What is the base business growth, if any? | | | | | | | | | | | |
| 4. Is the rest of the pipeline worth anything? | | | | | | | | | | | |