Positive Early Access to Medicines Scheme (EAMS) Scientific Opinion Awarded to GBT’s Voxelotor for the Treatment of Haemolytic Anaemia Due to Sickle Cell Disease
27 January 2022 - 7:00PM
Positive Early Access to Medicines Scheme (EAMS) Scientific Opinion
Awarded to GBT’s Voxelotor for the Treatment of Haemolytic Anaemia
Due to Sickle Cell Disease
Global Blood Therapeutics (GBT) today announced that the UK’s
Medicines and Healthcare products Regulatory Agency (MHRA) has
awarded a positive scientific opinion under the Early Access to
Medicines Scheme (EAMS) for voxelotor, an oral once-daily tablet
under review by the MHRA for the treatment of haemolytic anaemia
due to sickle cell disease (SCD) in adults and paediatric patients
12 years of age and older as monotherapy or in combination with
hydroxycarbamide. This positive opinion means that those patients
living with SCD and meeting the eligibility criteria can gain
early, pre-license access to voxelotor, while the MHRA completes
its review of the Marketing Authorisation Application (MAA).
The aim of EAMS is to grant early access to medicines that have
received Promising Innovative Medicine (PIM) designation for
patients in the UK with life-threatening or seriously debilitating
conditions prior to marketing authorisation when there is a clear
unmet medical need. Medicines under EAMS that receive marketing
authorisation by the MHRA as well as a positive assessment by the
National Institute of Health and Care Excellence (NICE) also
benefit from accelerated NHS England commissioning.
Following a positive opinion from the Committee for Medicine
Products for Human Use (CHMP) recommending the approval of
voxelotor in the European Union (EU), GBT submitted an application
to the MHRA for a Great Britain Marketing Authorisation using the
European Commission (EC) Decision Reliance Procedure. If approved,
voxelotor would be the first medicine in Great Britain and Europe
that directly inhibits sickle haemoglobin polymerisation, the
molecular cause of sickling and destruction of red blood cells in
SCD.
SCD is a rare genetic condition that affects approximately
15,000 people in the UK.1 People living with SCD and those closest
to them experience a lifelong journey with the disease that
touches every aspect of their lives. Patients experience
progressive, serious complications and morbidities, including
end-organ damage, which lead to decreased quality of life and
early mortality.2 Furthermore, economic disadvantages and health
inequalities experienced by many patients with SCD can have
negative societal impacts in areas such as access to healthcare,
education and employment.3-9
“This decision marks a significant milestone for the sickle cell
community in the UK,” said Arvind Agrawal, UK Medical Director at
GBT. “The EAMS positive scientific opinion is a key step forward to
meeting our goal of providing patients in the UK with the first
oral treatment option that inhibits red blood cell sickling and has
the potential to reduce acute and chronic complications of sickle
cell disease. GBT is delighted with the progress to help eligible
patients have access to this innovation as soon as possible.”
Voxelotor is currently approved in the United States, under the
brand name Oxbryta®, for the treatment of SCD in adults and
children 4 years of age and older.10 The Ministry of Health and
Prevention (MOHAP) in the United Arab Emirates (UAE) has granted
marketing authorisation for voxelotor as an oral treatment for SCD
in adults and children 12 years of age and older.
For more information on the GBT Early Access to Medicines Scheme
please contact EAMSinquiry@gbt.com.
About Sickle Cell Disease Sickle cell disease
(SCD) affects approximately 15,000 people in the UK,1 an estimated
52,000 people in Europe,11 and millions of people throughout the
world, particularly among those whose ancestors are from
sub-Saharan Africa.12 It also affects people of Hispanic, South
Asian, Southern European and Middle Eastern ancestry.12 SCD is a
lifelong inherited rare blood disorder that impacts haemoglobin, a
protein carried by red blood cells that delivers oxygen to tissues
and organs throughout the body.13 Due to a genetic mutation,
individuals with SCD form abnormal haemoglobin known as sickle
haemoglobin. Through a process called haemoglobin polymerisation,
red blood cells become sickled, crescent-shaped and rigid.2,13-14
The recurrent sickling process causes destruction of the red blood
cells and haemolytic anaemia (low haemoglobin due to red blood cell
destruction) and blockages in capillaries and small blood vessels,
which impede the flow of blood and oxygen delivery throughout the
body. The diminished oxygen delivery to tissues and organs can lead
to life-threatening complications, including stroke and
irreversible organ damage.2,14-16 Complications of SCD begin in
early childhood and can include neurocognitive impairment, acute
chest syndrome, and overt stroke, among other serious issues.17
About Voxelotor Tablets Voxelotor is an oral,
once-daily therapy that works by increasing haemoglobin’s affinity
for oxygen. Since oxygenated sickle haemoglobin does not
polymerise, voxelotor inhibits sickle haemoglobin polymerisation
and the resultant sickling and destruction of red blood cells
leading to haemolysis and haemolytic anaemia, which are pathologies
faced by every single person living with sickle cell disease (SCD).
Through addressing haemolytic anaemia and improving oxygen delivery
throughout the body, GBT believes that voxelotor has the potential
to modify the course of SCD. In November 2019, the U.S. Food and
Drug Administration (FDA) granted accelerated approval for Oxbryta
(voxelotor) tablets for the treatment of SCD in adults and children
12 years of age and older, and in December 2021, the U.S. FDA
expanded the approved use of Oxbryta for the treatment of SCD in
patients 4 years of age and older.10
In recognition of the critical need for new SCD treatments,
voxelotor has been granted Priority Medicines (PRIME) designation
from the European Medicines Agency (EMA). Voxelotor was designated
by the European Commission (EC) as an orphan medicinal product for
the treatment of patients with SCD. In addition, the Medicines and
Healthcare products Regulatory Agency (MHRA) in the UK granted
voxelotor a Promising Innovative Medicine (PIM) designation.
About Global Blood Therapeutics Global Blood
Therapeutics, Inc. (GBT) is a biopharmaceutical company dedicated
to the discovery, development and delivery of life-changing
treatments that provide hope to underserved patient communities.
Founded in 2011, GBT is delivering on its goal to transform the
treatment and care of sickle cell disease (SCD), a lifelong,
devastating inherited blood disorder. The company has introduced
Oxbryta (voxelotor) tablets and tablets for oral suspension, the
first FDA-approved medicine that directly inhibits sickle
haemoglobin (HbS) polymerisation, the root cause of red blood cell
sickling in SCD. GBT is also advancing its pipeline programme in
SCD with inclacumab, a P-selectin inhibitor in Phase 3 development
to address pain crises associated with the disease, and GBT021601
(GBT601), the company’s next generation HbS polymerisation
inhibitor. In addition, GBT’s drug discovery teams are working on
new targets to develop the next generation of treatments for SCD.
To learn more, please visit www.gbt.com.
References
- Sickle Cell Society. About Sickle Cell.
https://www.sicklecellsociety.org/about-sickle-cell/. Accessed
January, 2022.
- Kato GJ, et al. Sickle cell disease. Nat Rev Dis Primers.
2018;4:18010
- McClish DK, et al. Health related quality of life in sickle
cell patients: the PiSCES project. Health Qual Life Outcomes.
2005;3:50.
- Daniel LC, et al. Lessons Learned From a Randomized Controlled
Trial of a Family-Based Intervention to Promote School Functioning
for School-Age Children With Sickle Cell Disease. J Pediatr
Psychol. 2015;40:1085-1094.
- Dampier C, et al. Health-related quality of life in adults with
sickle cell disease (SCD): a report from the Comprehensive Sickle
Cell Centers Clinical Trial Consortium. Am J Hematol.
2011;86:203-205.
- Dampier C, et al. Health-related quality of life in children
with sickle cell disease: a report from the Comprehensive Sickle
Cell Centers Clinical Trial Consortium. Pediatr Blood Cancer.
2010;55:485-494.
- Anie KA, et al. Sickle cell disease: Pain, coping and quality
of life in a study of adults in the UK. Br J Health Psychol.
2002;7:331-344.
- Kambasu DM, et al. Health-related quality of life of
adolescents with sickle cell disease in sub-Saharan Africa: a
cross-sectional study. BMC Hematol. 2019;19:9.
- Lubeck D, et al. Estimated Life Expectancy and Income of
Patients With Sickle Cell Disease Compared With Those Without
Sickle Cell Disease. JAMA Netw Open. 2019;2:e1915374.
- Oxbryta (voxelotor) tablets prescribing information. South San
Francisco, Calif. Global Blood Therapeutics, Inc.; December
2021.
- European Medicines Agency.
https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125.
Accessed January, 2022.
- Centers for Disease Control and Prevention website. Sickle Cell
Disease (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html.
Accessed January, 2022.
- National Heart, Lung, and Blood Institute website. Sickle Cell
Disease.
https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease.
Accessed January, 2022.
- Rees DC, et al. Sickle cell disease. Lancet.
2010;376(9757):2018-2031.
- Kato GJ et al. Intravascular hemolysis and the pathophysiology
of sickle cell disease. J Clin Invest. 2017;127:750-760.
- Caboot JB, et al. Hypoxemia in sickle cell disease:
significance and management Paediatr Respir Rev.
2014;15(1):17-23.
- Cooper TE et al. Pharmacological interventions for painful
sickle cell vaso-occlusive crises in adults. Cochrane Database of
Systematic Reviews. 2019.
Contacts: Claudia Nabaie (media Europe)+41 79
906 5814 cnabaie@gbt.com
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