 Home > Active Ingredient News > Blood System > U.S. FDA expands indications for sickle cell disease drug Oxbryta

U.S. FDA expands indications for sickle cell disease drug Oxbryta

 Last Update: 2021-12-27
 Source: Internet
 Author: User

Tags

bystolic 10 mg

fda drug classification

Search more information of high quality chemicals, good prices and reliable suppliers, visit www.echemi.com

The production of sickle hemoglobin is caused by a sickle point mutation in the beta globin gene.
The solubility of this hemoglobin is worse than that of normal fetal or adult hemoglobin
.

Sickle cell disease (SCD) refers to any of the following syndromes, in which a sickle mutation is co-inherited with a mutation on another allele of β-globin, resulting in a reduction or disappearance of normal β-globin production

Global Blood Therapeutics (GBT) recently stated that the FDA has expanded the indications of Oxbryta (voxelotor) to allow it to treat sickle cell disease (SCD) in children under 4 to 12 years of age
.

Oxbryta (voxelotor), a once-daily oral hemoglobin polymerization inhibitor, was initially approved in 2019 for accelerated approval for adults and adolescents with SCD aged 12 years and older

FDA children

This approval is supported by data from the Phase IIa HOPE-KIDS 1 trial, which involved 45 SCD patients aged 4 to 11 years
.

The HOPE-KIDS 1 test has four parts

The average age of the patients was 7 years, slightly more than half of the women, and the average weight was 24 kg
.

The baseline mean hemoglobin was 8.

Blood vessel

The results show that treatment with voxelotor can significantly improve anemia in pediatric patients
.

The average hemoglobin change from baseline at week 24 was 1.

Voxelotor treatment also resulted in a reduction in hemolysis markers at 24 weeks

Original source:

https://firstwordpharma.

https://firstwordpharma.
com/story/5465201 https://firstwordpharma.
com/story/5465201 Leave a message here

This article is an English version of an article which is originally in the Chinese language on echemi.com and is provided for information purposes only. This website makes no representation or warranty of any kind, either expressed or implied, as to the accuracy, completeness ownership or reliability of the article or any translations thereof. If you have any concerns or complaints relating to the article, please send an email, providing a detailed description of the concern or complaint, to service@echemi.com. A staff member will contact you within 5 working days. Once verified, infringing content will be removed immediately.

Related Products

SULODEXIDE 99% Almost White Powder Dideu-01112 Dideu

CAS No.: 57821-29-1

Pharmacy Grade

$61.76/G EXW

Heparin sodium 9041-08-1 vials safe delivery

CAS No.: 9041-08-1

Pharmacy Grade

$1/G EXW

Gadusol Powder Gadusol76663-30-4

CAS No.: 76663-30-4

Industrial Grade

$1-1.01/G FOB

Hot Tags

united states jobs(223)

jobs united states(236)

1 year treasury(277)

1 year subscription(296)

single use technology(195)

announcements today(81)

ups united states(221)

united states logo(227)

80 20 company(94)

healthy food name(42)

Hot Articles

The source of this page with content of products and services is from Internet, which doesn't represent ECHEMI's opinion. If you have any queries, please write to service@echemi.com. It will be replied within 5 days.

Moreover, if you find any instances of plagiarism from the page, please send email to service@echemi.com with relevant evidence.