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Priority Review Vouchers for rare pediatric diseases, issued and sold 2012-present

RARE PEDIATRIC DISEASEDRUG NAMECOMPANYVOUCHER AWARD YEAR AND SALE AMOUNTBUYER
1. Morquio A syndromeVimizim BioMarin2014,
$67.5M
Regeneron Pharmaceuticals Inc. and Sanofi SA, redeemed for Praluent
2. High-risk neuroblastoma *CancerUnituxinUnited Therapeutics2015,
$350M
AbbVie. Redeemed in 2019 for upadacitinib to treat rheumatoid arthritis.
3. Rare bile acid synthesis disorders
CholbamAsklepion2015,
$245M
Sanofi SA, redeemed for LixiLan
4. Hereditary orotic aciduria
XuridenWellstat2015AstraZeneca
5. Hypophosphatasia
StrensiqAlexion2015Redeemed for ravulizumab
6. Lysosomal acid lipase deficiency
Kanuma (sebelipase alfa)Alexion2015
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
SpinrazaBiogen (Ionis Pharmaceuticals)
2016
9. Duchenne muscular dystrophy
EmflazaMarathon2017,
$130 million
Viiv Healthcare
10. Batten disease
BrineuraBioMarin2017,
$125M
Novartis Pharmaceuticals
11. B-cell acute lymphoblastic leukemia *Cancer
KymriahNovartis
12. Mucopolysacchari-dosis (MPS) VII
Mepsevii
Ultragenyx2017,
$130M
Novartis
13. Biallelic RPE65 mutation-associated retinal dystrophy
LuxturnaSpark Therapeutics
2018,
$110M
Jazz Pharmaceuticals
14. X-linked hypophosphatemia (XLH)
Crysvita (burosumab-twza)
Ultragenyx2018,
$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 Research2018,
$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) injectionLeadiant Bioscience2018
17. Primary haemophagocytic lymphohistiocytosis (HLH)Gamifant (emapalumab-lzsg)Sobi and Novimmune SA2018. Sobi sold the voucher for $95 million in August 2019.AstraZeneca
18. Cystic fibrosis with F508del mutationSymdekoVertex Pharmaceuticals2019
19. Spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) geneZolgensma (onasemnogene abeparvovec-xioi)AveXis/Novartis2019
20. Cystic fibrosisTrikafta (elexacaftor/ivacaftor/tezacaftor)Vertex Pharmaceuticals2019
21. Duchenne muscular dystrophy (patients with error in exon 53)Vyondys 53 (golodirsen)Sarepta Therapeutics2019. Sold in 2020 for $111mVifor, which plans to redeem it for vadadustat, a treatment for anemia due to chronic kidney disease
22. Neurofibromatosis type 1 (NF1)Koselugo (selumetinib)AstraZeneca2020
23. Spinal muscular atrophy in pediatric and adult patientsEvrysdi (risdiplam)Genentech2020
24. Duchenne Muscular Dystrophy in patients amenable to Exon 53 SkippingViltepso (viltolarsen)Nippon Shinyaku Co./NS Pharma Inc.2020
25. Hutchinson-Gilford Progeria syndrome and Processing-Deficient Progeroid LaminopathiesZokinvy (lonafarnib)Eiger BioPharmaceuticals 2020. Sold for $95 millionAbbVie
26. Neuroblastoma, relapsed or refractory
* Cancer
Danyelza (naxitamab)Y-mAb Therapeutics2020, sold for $105millionUnited Therapeutics
27. Obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency Imcivree (setmelanotide)Rhythm Pharmaceuticals2020, sold for $100 millionAlexion
28. Primary hyperoxaluria type 1Oxlumo (lumasiran)Alnylam Pharmaceuticals2020
29. Duchenne Muscular Dystrophy in patients with gene amenable to exon 45 skippingAmondys 45 (casimersen)Sarepta Therapeutics2021
30. Molybdenum Cofactor Deficiency Type ANulibry (fosdenopterin)Origin Biosciences, affiliate of BridgeBio Pharma2021, sold for $110 million in 2022Undisclosed
31. Plasminogen deficiency type 1 (hypoplasminogenemia)Ryplazim (plasminogen, human-tvmh)ProMetic Biotherapeutics /Liminal Biosciences2021, sold for $105 millionKedrion Biopharma
32. Pruritus in all types of progressive familial intrahepatic cholestasis (PFIC)Bylvay (odevixibat)Albireo Pharma2021, sold for $105 millionUndisclosed
33. Cholestatic Pruritus in Patients with Alagille SyndromeLivmarli (maralixibat)Mirum Pharmaceuticals2021, sold for $110 millionUndisclosed
34. Congenital athymiaRethymic (allogeneic processed thymus tissue-agdc)Enzyvant2021
35. AchondroplasiaVoxzogo (vosoritide)BioMarin Pharmaceutical2021, sold for $110 million in 2022Undisclosed
36. Seizures of genetic epilepsyZtalmy (ganaxolone)Marinus Pharmaceuticals2022, Sold in 2022 for $110 million.Undisclosed
37. Beta thalassemiaZynteglo (betibeglogene autotemcel) Bluebird Bio2022. Sold in 2022 for $102 million.Argenx
38. Sphingomyelinase deficiency (ASMD)Xenpozyme (olipudase alfa) Sanofi2022
39. Cerebral adrenoleukodystrophySkysona (elivaldogene autotemcel) Bluebird Bio2022. Sold in 2023 for $95 millionBristol Myers Squibb

 

  • Creating Hope Act and Pediatric Priority Review Vouchers — Overview
  • Statutory text
  • FDA Guidance on Pediatric Vouchers
  • Pediatric Vouchers Issued and Sold, 2012-present
  • 2020 Testimony in Support of Making the Pediatric Voucher Program Permanent
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President Obama before signing Creating Hope Act
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