| 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 |
| 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 | 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 | |
| 10. Batten disease | Brineura | BioMarin | 2017, $125M | Undisclosed |
| 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 | Undisclosed |
| 15. Seizures associated with Dravet Syndrome or Lennox-Gastaut Syndrome | Epidiolex (cannabidiol oral solution) | GW Research | 2018, $105M | Biohaven Pharmaceutical Holding, will redeem for rimegepant Zydis |
| 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 | |
| 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 | 2019 |
Learn more:
Regulatory Explainer: Everything You Need to Know About FDA’s Priority Review Vouchers