| RARE PEDIATRIC DISEASE | DRUG NAME | COMPANY | VOUCHER AWARD YEAR AND SALE AMOUNT | BUYER |
|---|---|---|---|---|
| 1. Morquio A syndrome | Vimizim | BioMarin | 2014, $67.5M | Regeneron Pharmaceuticals Inc. and Sanofi SA, redeemed for Praluent |
| 2. High-risk neuroblastoma *Cancer | Unituxin | United Therapeutics | 2015, $350M | AbbVie. Redeemed in 2019 for upadacitinib to treat rheumatoid arthritis. |
| 3. Rare bile acid synthesis disorders | Cholbam | Asklepion | 2015, $245M | Sanofi SA, redeemed for LixiLan |
| 4. Hereditary orotic aciduria | Xuriden | Wellstat | 2015 | AstraZeneca |
| 5. Hypophosphatasia | Strensiq | Alexion | 2015 | Redeemed for ravulizumab |
| 6. Lysosomal acid lipase deficiency | Kanuma (sebelipase alfa) | Alexion | 2015 | |
| 7. Duchenne muscular dystrophy | Exondys 51 | Sarepta Therapeutics | 2016, $125M | Gilead, redeemed for combination of bictegravir, emtricitabine and tenofovir alafenamide for treatment of HIV |
| 8. Spinal muscular atrophy | Spinraza | Biogen (Ionis Pharmaceuticals) | 2016 | |
| 9. Duchenne muscular dystrophy | Emflaza | Marathon | 2017, $130 million | Viiv Healthcare |
| 10. Batten disease | Brineura | BioMarin | 2017, $125M | Novartis Pharmaceuticals |
| 11. B-cell acute lymphoblastic leukemia *Cancer | Kymriah | Novartis | ||
| 12. Mucopolysacchari-dosis (MPS) VII | Mepsevii | Ultragenyx | 2017, $130M | Novartis |
| 13. Biallelic RPE65 mutation-associated retinal dystrophy | Luxturna | Spark Therapeutics | 2018, $110M | Jazz Pharmaceuticals |
| 14. X-linked hypophosphatemia (XLH) | Crysvita (burosumab-twza) | Ultragenyx | 2018, $80.6M | Gilead. Plans to use it for approval of filgotinib, a treatment for rheumatoid arthritis |
| 15. Seizures associated with Dravet Syndrome or Lennox-Gastaut Syndrome | Epidiolex (cannabidiol oral solution) | GW Research | 2018, $105M | Biohaven Pharmaceutical Holding, redeemed for rimegepant (Nurtec ODT) for treatment of migraine in adults |
| 16. Adenosine deaminase-severe combined immunodeficiency (ADA-SCID) | Revcovi (elapegademase-lvlr) injection | Leadiant Bioscience | 2018 | |
| 17. Primary haemophagocytic lymphohistiocytosis (HLH) | Gamifant (emapalumab-lzsg) | Sobi and Novimmune SA | 2018. Sobi sold the voucher for $95 million in August 2019. | AstraZeneca |
| 18. Cystic fibrosis with F508del mutation | Symdeko | Vertex Pharmaceuticals | 2019 | |
| 19. Spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene | Zolgensma (onasemnogene abeparvovec-xioi) | AveXis/Novartis | 2019 | |
| 20. Cystic fibrosis | Trikafta (elexacaftor/ivacaftor/tezacaftor) | Vertex Pharmaceuticals | 2019 | |
| 21. Duchenne muscular dystrophy (patients with error in exon 53) | Vyondys 53 (golodirsen) | Sarepta Therapeutics | 2019. Sold in 2020 for $111m | Vifor, which plans to redeem it for vadadustat, a treatment for anemia due to chronic kidney disease |
| 22. Neurofibromatosis type 1 (NF1) | Koselugo (selumetinib) | AstraZeneca | 2020 | |
| 23. Spinal muscular atrophy in pediatric and adult patients | Evrysdi (risdiplam) | Genentech | 2020 | |
| 24. Duchenne Muscular Dystrophy in patients amenable to Exon 53 Skipping | Viltepso (viltolarsen) | Nippon Shinyaku Co./NS Pharma Inc. | 2020 | |
| 25. Hutchinson-Gilford Progeria syndrome and Processing-Deficient Progeroid Laminopathies | Zokinvy (lonafarnib) | Eiger BioPharmaceuticals | 2020. Sold for $95 million | AbbVie |
| 26. Neuroblastoma, relapsed or refractory * Cancer | Danyelza (naxitamab) | Y-mAb Therapeutics | 2020, sold for $105million | United Therapeutics |
| 27. Obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency | Imcivree (setmelanotide) | Rhythm Pharmaceuticals | 2020, sold for $100 million | Alexion |
| 28. Primary hyperoxaluria type 1 | Oxlumo (lumasiran) | Alnylam Pharmaceuticals | 2020 | |
| 29. Duchenne Muscular Dystrophy in patients with gene amenable to exon 45 skipping | Amondys 45 (casimersen) | Sarepta Therapeutics | 2021 | |
| 30. Molybdenum Cofactor Deficiency Type A | Nulibry (fosdenopterin) | Origin Biosciences, affiliate of BridgeBio Pharma | 2021, sold for $110 million in 2022 | Undisclosed |
| 31. Plasminogen deficiency type 1 (hypoplasminogenemia) | Ryplazim (plasminogen, human-tvmh) | ProMetic Biotherapeutics /Liminal Biosciences | 2021, sold for $105 million | Kedrion Biopharma |
| 32. Pruritus in all types of progressive familial intrahepatic cholestasis (PFIC) | Bylvay (odevixibat) | Albireo Pharma | 2021, sold for $105 million | Undisclosed |
| 33. Cholestatic Pruritus in Patients with Alagille Syndrome | Livmarli (maralixibat) | Mirum Pharmaceuticals | 2021, sold for $110 million | Undisclosed |
| 34. Congenital athymia | Rethymic (allogeneic processed thymus tissue-agdc) | Enzyvant | 2021 | |
| 35. Achondroplasia | Voxzogo (vosoritide) | BioMarin Pharmaceutical | 2021, sold for $110 million in 2022 | Undisclosed |
| 36. Seizures of genetic epilepsy | Ztalmy (ganaxolone) | Marinus Pharmaceuticals | 2022, Sold in 2022 for $110 million. | Undisclosed |
| 37. Beta thalassemia | Zynteglo (betibeglogene autotemcel) | Bluebird Bio | 2022. Sold in 2022 for $102 million. | Argenx |
| 38. Sphingomyelinase deficiency (ASMD) | Xenpozyme (olipudase alfa) | Sanofi | 2022 | |
| 39. Cerebral adrenoleukodystrophy | Skysona (elivaldogene autotemcel) | Bluebird Bio | 2022. Sold in 2023 for $95 million | Bristol Myers Squibb |