Evicenter
P&T meetings

Aqneursa

Ataxia-telangiectasia

Also known as levacetylleucine, IB1001
Manufacturer
IntraBio
206 sources

Section 6 of 6

References

206 sources

  1. Abid et al. (2026) (opens in a new tab)
  2. Akturk et al. (2017) (opens in a new tab)
  3. Amirifar et al. (2019) (opens in a new tab)
  4. Amirifar et al. (2020) (opens in a new tab)
  5. Aqneursa prescribing information (2026) (opens in a new tab)
  6. ASHP AHFS DI Essentials (2025) (opens in a new tab)
  7. Ataxia-Telangiectasia Society (2014) (opens in a new tab)
  8. Ataxia-Telangiectasia Society (2026) (opens in a new tab)
  9. Austin American-Statesman (2024) (opens in a new tab)
  10. Bakhtiar et al. (2021) (opens in a new tab)
  11. Becker-Bense et al. (2023) (opens in a new tab)
  12. Benard et al. (2001) (opens in a new tab)
  13. Beresford et al. (2013) (opens in a new tab)
  14. Beyraghi-Tousi et al. (2024) (opens in a new tab)
  15. Bhatt et al. (2015) (opens in a new tab)
  16. Blom et al. (2019) (opens in a new tab)
  17. Brandsma et al. (2014) (opens in a new tab)
  18. Bremova-Ertl et al. (2024) (opens in a new tab)
  19. Brown and Barañano (2026) (opens in a new tab)
  20. Brueggemann et al. (2022) (opens in a new tab)
  21. Cabana et al. (1998) (opens in a new tab)
  22. California Department of Health Care Access and Information Prescription Drugs Introduced to Market (2026) (opens in a new tab)
  23. CarelonRx DrugInsights (2024) (opens in a new tab)
  24. Chessa et al. (2014) (opens in a new tab)
  25. Chopra et al. (2014) (opens in a new tab)
  26. Churchill et al. (2020) (opens in a new tab)
  27. Churchill et al. (2021) (opens in a new tab)
  28. Cigna Healthcare (2025) (opens in a new tab)
  29. Cirillo et al. (2018) (opens in a new tab)
  30. Cirillo et al. (2022) (opens in a new tab)
  31. ClinicalTrials.gov NCT03759678 (opens in a new tab)
  32. ClinicalTrials.gov NCT03759678 (opens in a new tab)
  33. ClinicalTrials.gov NCT03759678 (opens in a new tab)
  34. ClinicalTrials.gov NCT06193200 (opens in a new tab)
  35. ClinicalTrials.gov NCT06673056 (opens in a new tab)
  36. ClinicalTrials.gov NCT07221292 (opens in a new tab)
  37. ClinicalTrials.gov NCT07380165 (opens in a new tab)
  38. Collyer and Rajan (2024) (opens in a new tab)
  39. Coman et al. (2025) (opens in a new tab)
  40. Crawford et al. (2000) (opens in a new tab)
  41. Crawford et al. (2006) (opens in a new tab)
  42. CTIS 2024-517706-29 (opens in a new tab)
  43. Curant Health (2024) (opens in a new tab)
  44. Curant Health (2026) (opens in a new tab)
  45. Davis et al. (2026) (opens in a new tab)
  46. de Leon et al. (2025) (opens in a new tab)
  47. DelveInsight (2026) (opens in a new tab)
  48. Department of Veterans Affairs Federal Supply Schedule pharmaceutical prices (2026) (opens in a new tab)
  49. Devaney et al. (2017) (opens in a new tab)
  50. Donath et al. (2019) (opens in a new tab)
  51. Donath et al. (2020) (opens in a new tab)
  52. Donath et al. (2022) (opens in a new tab)
  53. Drugs.com Aqneursa price guide (opens in a new tab)
  54. Drugs@FDA NDA 219132 (opens in a new tab)
  55. Dutzmann et al. (2022) (opens in a new tab)
  56. Elitzur et al. (2024) (opens in a new tab)
  57. European Medicines Agency Aqneursa assessment report (2025) (opens in a new tab)
  58. European Medicines Agency Aqneursa EPAR (2026) (opens in a new tab)
  59. European Medicines Agency Aqneursa product information (2026) (opens in a new tab)
  60. European Medicines Agency CHMP agenda (2026) (opens in a new tab)
  61. European Medicines Agency CHMP meeting highlights (2026) (opens in a new tab)
  62. European Medicines Agency orphan designation EU/3/18/2124 (2019) (opens in a new tab)
  63. FDA (2024) (opens in a new tab)
  64. FDA (2026) (opens in a new tab)
  65. FDA Federal Register notice (2024) (opens in a new tab)
  66. FDA integrated review (2024) (opens in a new tab)
  67. FDA NDA approval letter (2024) (opens in a new tab)
  68. FDA Orange Book (2026) (opens in a new tab)
  69. FDA Orphan Drug Designations and Approvals (2018) (opens in a new tab)
  70. FDA supplement approval letter (2026) (opens in a new tab)
  71. Feil et al. (2021) (opens in a new tab)
  72. Félix et al. (2014) (opens in a new tab)
  73. Fields et al. (2021) (opens in a new tab)
  74. Fields et al. (2023) (opens in a new tab)
  75. Fierce Pharma FDA approval report (2026) (opens in a new tab)
  76. Fierce Pharma phase 3 results report (2026) (opens in a new tab)
  77. Günther et al. (2015) (opens in a new tab)
  78. Hasegawa et al. (2019) (opens in a new tab)
  79. Health Research Authority (opens in a new tab)
  80. Hegdekar et al. (2021) (opens in a new tab)
  81. Hensiek et al. (2026) (opens in a new tab)
  82. Hoche et al. (2019) (opens in a new tab)
  83. Horn et al. (2025) (opens in a new tab)
  84. IntraBio AQNEURSA ataxia-telangiectasia website (2026) (opens in a new tab)
  85. IntraBio AQNEURSA Cares healthcare professional brochure (2024) (opens in a new tab)
  86. IntraBio AQNEURSA Cares patient brochure (2024) (opens in a new tab)
  87. IntraBio AQNEURSA healthcare professional website (2026) (opens in a new tab)
  88. IntraBio AQNEURSA patient enrollment form (2026) (opens in a new tab)
  89. IntraBio AQNEURSA prior authorization checklist (2026) (opens in a new tab)
  90. IntraBio CACNA1A trial authorization release (2026) (opens in a new tab)
  91. IntraBio CHMP positive opinion release (2025) (opens in a new tab)
  92. IntraBio EMA variation release (2026) (opens in a new tab)
  93. IntraBio Expanded Access Policy (2026) (opens in a new tab)
  94. IntraBio FDA approval release (2026) (opens in a new tab)
  95. IntraBio further detail release (2026) (opens in a new tab)
  96. IntraBio IB1001-203 protocol (2023) (opens in a new tab)
  97. IntraBio IB1001-203 statistical analysis plan (2024) (opens in a new tab)
  98. IntraBio patient community letter (2024) (opens in a new tab)
  99. IntraBio priority review release (2026) (opens in a new tab)
  100. IntraBio sNDA submission release (2026) (opens in a new tab)
  101. IntraBio topline results release (2026) (opens in a new tab)
  102. IntraBio Virtual Patent Marking (2026) (opens in a new tab)
  103. IRCT IRCT20210413050958N1 (opens in a new tab)
  104. Jackson et al. (2016) (opens in a new tab)
  105. Kaya et al. (2020) (opens in a new tab)
  106. Kaya et al. (2021) (opens in a new tab)
  107. Kelly et al. (2022) (opens in a new tab)
  108. Khan et al. (2024) (opens in a new tab)
  109. Koenig et al. (2025) (opens in a new tab)
  110. Krauthammer et al. (2018) (opens in a new tab)
  111. Kridli et al. (2024) (opens in a new tab)
  112. Lawerman et al. (2017) (opens in a new tab)
  113. Lefton-Greif et al. (2000) (opens in a new tab)
  114. Lefton-Greif et al. (2011) (opens in a new tab)
  115. Leuzzi et al. (2015) (opens in a new tab)
  116. Levy and Lang (2018) (opens in a new tab)
  117. Lindahl et al. (2024) (opens in a new tab)
  118. Lockman et al. (2012) (opens in a new tab)
  119. Lynch et al. (2025) (opens in a new tab)
  120. Magnarelli et al. (2025) (opens in a new tab)
  121. Major et al. (2024) (opens in a new tab)
  122. Mallott et al. (2013) (opens in a new tab)
  123. Mandola et al. (2019) (opens in a new tab)
  124. Martakis et al. (2026) (opens in a new tab)
  125. Martakis et al. supplementary appendix (2026) (opens in a new tab)
  126. McGlashan et al. (2022) (opens in a new tab)
  127. McGrath-Morrow et al. (2010) (opens in a new tab)
  128. McGrath-Morrow et al. (2014) (opens in a new tab)
  129. McGrath-Morrow et al. (2021) (opens in a new tab)
  130. Medicaid.gov State Drug Utilization Data 2025 (2026) (opens in a new tab)
  131. Medicaid.gov State Drug Utilization Data 2026 (2026) (opens in a new tab)
  132. Medical Mutual (2025) (opens in a new tab)
  133. MedlinePlus Genetics (2022) (opens in a new tab)
  134. Mehri et al. (2024) (opens in a new tab)
  135. Micol et al. (2011) (opens in a new tab)
  136. Morrell et al. (1986) (opens in a new tab)
  137. Natale et al. (2021) (opens in a new tab)
  138. National Organization for Rare Disorders (2022) (opens in a new tab)
  139. New York State Medicaid eMedNY (opens in a new tab)
  140. New York State Medicaid list of reimbursable drugs (2026) (opens in a new tab)
  141. NHS England (2013) (opens in a new tab)
  142. Nissenkorn et al. (2013) (opens in a new tab)
  143. Nissenkorn et al. (2016) (opens in a new tab)
  144. Nowak-Wegrzyn et al. (2004) (opens in a new tab)
  145. Oklahoma Health Care Authority Drug Utilization Review Board packet (2025) (opens in a new tab)
  146. openFDA NDC Directory (2026) (opens in a new tab)
  147. Oregon State University Drug Use Research and Management Program (2025) (opens in a new tab)
  148. Padilla et al. (2026) (opens in a new tab)
  149. Panvino et al. (2026) (opens in a new tab)
  150. Park et al. (2024) (opens in a new tab)
  151. Patterson et al. (2025) (opens in a new tab)
  152. Patterson et al. (2026a) (opens in a new tab)
  153. Patterson et al. (2026b) (opens in a new tab)
  154. Pereira et al. (2024) (opens in a new tab)
  155. Perez-Lloret et al. (2021) (opens in a new tab)
  156. Petley et al. (2022) (opens in a new tab)
  157. Potashman et al. (2024a) (opens in a new tab)
  158. Potashman et al. (2024b) (opens in a new tab)
  159. Presterud et al. (2024) (opens in a new tab)
  160. Quince Therapeutics topline results release (2026) (opens in a new tab)
  161. Rehm et al. (2026) (opens in a new tab)
  162. Reiman et al. (2011) (opens in a new tab)
  163. Rothblum-Oviatt et al. (2016) (opens in a new tab)
  164. Ruano et al. (2014) (opens in a new tab)
  165. Russo et al. (2026) (opens in a new tab)
  166. Saberi-Karimian et al. (2023) (opens in a new tab)
  167. Schmitz-Hübsch et al. (2006) (opens in a new tab)
  168. Schmitz-Hübsch et al. (2008) (opens in a new tab)
  169. Schmitz-Hübsch et al. (2010) (opens in a new tab)
  170. Schoenaker et al. (2020) (opens in a new tab)
  171. Schon et al. (2019) (opens in a new tab)
  172. Schroeder et al. (2005) (opens in a new tab)
  173. Shaikh et al. (2013) (opens in a new tab)
  174. Shiloh (2020) (opens in a new tab)
  175. Song et al. (2026) (opens in a new tab)
  176. Stewart et al. (2016) (opens in a new tab)
  177. Stray-Pedersen et al. (2007) (opens in a new tab)
  178. Suarez et al. (2015) (opens in a new tab)
  179. Swift et al. (1986) (opens in a new tab)
  180. Theis et al. (2023) (opens in a new tab)
  181. Tiet et al. (2020) (opens in a new tab)
  182. Tiet et al. (2025) (opens in a new tab)
  183. Tighilet et al. (2015) (opens in a new tab)
  184. Trouillas et al. (1997) (opens in a new tab)
  185. UnitedHealthcare (2026) (opens in a new tab)
  186. van Gool et al. (2025) (opens in a new tab)
  187. van Os et al. (2017a) (opens in a new tab)
  188. van Os et al. (2017b) (opens in a new tab)
  189. van Os et al. (2020) (opens in a new tab)
  190. Vanderkam et al. (2019) (opens in a new tab)
  191. Veenhuis et al. (2021) (opens in a new tab)
  192. Veenhuis et al. (2026) (opens in a new tab)
  193. Verhagen et al. (2009) (opens in a new tab)
  194. Verhagen et al. (2012) (opens in a new tab)
  195. Vibert and Vidal (2001) (opens in a new tab)
  196. Vilozni et al. (2015) (opens in a new tab)
  197. Webber et al. (2026) (opens in a new tab)
  198. Weitering et al. (2025) (opens in a new tab)
  199. Weyer et al. (2007) (opens in a new tab)
  200. WHO Collaborating Centre for Drug Statistics Methodology (2026) (opens in a new tab)
  201. Wölke et al. (2020) (opens in a new tab)
  202. Woods and Taylor (1992) (opens in a new tab)
  203. Woods et al. (1990) (opens in a new tab)
  204. Zannolli et al. (2012) (opens in a new tab)
  205. Zielen et al. (2021) (opens in a new tab)
  206. Zielen et al. (2024) (opens in a new tab)