RT Book, Section
A1 Chan, C. Y. Jennifer
A1 Frei-Jones, Melissa
A2 DiPiro, Joseph T.
A2 Talbert, Robert L.
A2 Yee, Gary C.
A2 Matzke, Gary R.
A2 Wells, Barbara G.
A2 Posey, L. Michael
SR Print(0)
ID 1168070625
T1 Sickle Cell Disease
T2 Pharmacotherapy: A Pathophysiologic Approach, 10e
YR 2017
FD 2017
PB McGraw-Hill Education
PP New York, NY
SN 9781259587481
LK accesspharmacy.mhmedical.com/content.aspx?aid=1168070625
RD 2023/11/29
AB Content  UpdateNovember  26,  2019Voxelotor  Approved  for  Treatment  of  Sickle  Cell  Disease:  Voxelotor  (Oxbryta®)  is  the  first  hemoglobin  S  polymerization  inhibitor  approved  by  the  FDA  and  is  indicated  for  adults  and  children  older  than  12  for  the  treatment  of  sickle  cell  disease.  Sickle  cell  disease  affects  approximately  100,000  individuals  in  the  US  and  is  a  hereditary  condition  which  results  in  the  production  of  sickle,  or  abnormally,  shaped  hemoglobin  and  has  limited  treatment  options.  In  a  phase  3  randomized,  placebo  controlled,  double  blind  trial,  two  doses  of  voxelotor  were  compared  to  placebo.  Those  treated  with  the  1500mg  dose  had  a  significantly  higher  hemoglobin  response  compared  to  placebo,  51%  (95%  CI,  41  -  61)  versus  7%  (95%  CI,  1  -  12).  Adverse  effects  were  similar  across  the  study  groups  and  the  most  common  being  headache  and  diarrhea.