RegulationPriority Review (US), Breakthrough Therapy (US), Fast Track (US), Accelerated Approval (US), Orphan Drug (US), Rare Pediatric Disease (US), Orphan Drug (EU), PRIME (EU)

Structure

Molecular FormulaC19H19N3O3

InChIKeyFWCVZAQENIZVMY-UHFFFAOYSA-N

CAS Registry1446321-46-5

Clinical Trials associated with Voxelotor

NCT06023199 / RecruitingPhase 2IIT

Assessing Functional Capacity in Sickle Cell Patients Receiving Voxelotor

Voxelotor is a novel hemoglobin polymerization inhibitor which has been demonstrated to reduce hemolysis and improve hemoglobin levels. There have been numerous studies examining the clinical impact of voxelotor in sickle cell disease (SCD) patients, but there are few published reports on the effects of treatment on physical function in patients with SCD. The hypothesis to be tested is that anemic SCD patients will have improvements in performance after 6 months of voxelotor treatment.

Start Date23 Oct 2023

Sponsor / Collaborator

Inova Health Care Services

[+1]

NCT05228821 / WithdrawnPhase 4

An Open Label, Single Arm, Multicenter Study to Evaluate the Effect of Voxelotor on Cerebral Blood Flow and Neurocognitive Function in Adolescents and Adults With Sickle Cell Disease

This is an open label, single arm multicenter trial to evaluate the effect of voxelotor treatment on cerebral blood flow (CBF) and neurocognitive function in adolescent and young adult participants (12-30 years of age) with sickle cell disease (SCD).

Start Date09 Aug 2023

Sponsor / Collaborator

Pfizer Inc.

NCT05981365 / CompletedPhase 1

A Phase 1, Open-Label, Two-Part, Fixed-Sequence, Drug-Drug Interaction Study to Evaluate the Effect of Voxelotor on the Pharmacokinetics of Selected CYP and Transporter Probe Substrates in Healthy Participants

This research study is examining multiple doses of voxelotor (a study drug intended for treatment of sickle cell disease) and how it interacts with additional substrates (substrates are drugs or other substances that are metabolized by cytochrome enzymes. The substrates used in this study are FDA approved medications). The study will help to determine the safety and tolerability of the study drugs taken together, as well as the pharmacokinetics (PK) on how your body processes and responds to the combination of the study drug and substrates. Although these drugs are FDA approved, their use in this study is experimental.

Start Date17 Apr 2023

Sponsor / Collaborator

Pfizer Inc.

100 Clinical Results associated with Voxelotor

100 Translational Medicine associated with Voxelotor

100 Patents (Medical) associated with Voxelotor

109

Literatures (Medical) associated with Voxelotor

01 Mar 2024·Blood cells, molecules & diseases

Effect of voxelotor on murine bone marrow and peripheral blood with hematopoietic progenitor cell mobilization for gene therapy of sickle cell disease

Article

Author: Bao, Weili ; Mendelson, Avital ; Liu, Yunfeng ; Shi, Patricia A

In preparation for hematopoietic stem cell mobilization and collection, current ex vivo gene therapy protocols for sickle cell disease require patients to undergo several months of chronic red cell transfusion. For health care equity, alternatives to red cell transfusion should be available. We examined whether treatment with GBT1118, the murine analog of voxelotor, could be a safe and feasible alternative to red cell transfusion. We found that 3 weeks of treatment with GBT1118 increased the percentage of bone marrow hematopoietic stem cells and upon plerixafor mobilization, the percentage of peripheral blood hematopoietic stem cells. Our data suggest that voxelotor should be further explored for its potential safety and utility as preparation for hematopoietic stem cell mobilization and collection.

01 Feb 2024·American journal of hematology

Comparative pharmacovigilance assessment of adverse events associated with the use of hydroxyurea, L‐glutamine, voxelotor, and crizanlizumab in sickle cell disease

Letter

Author: Ataga, Kenneth I ; Hankins, Jane S ; Chen, Ming ; Zhang, Min

Using disproportionality analysis, this study compared the adverse events (AEs) associated with the use of the new agents (e.g., L-glutamine, voxelotor, and crizanlizumab) to the commonly used medication, hydroxyurea, in sickle cell disease. We found that the most frequent drug-related AEs observed in this real-world study were consistent with those in the HOPE (voxelotor) and SUSTAIN (crizanlizumab) trials, but the rates of AEs were lower. Our study demonstrates that the most common AEs and symptoms of an increased risk associated with the individual drugs varied by treatment. Disproportionate reporting signals of drug-related AEs may also capture information that is independent of subjective measures of patient-reported symptoms. Our study highlights the important need for facilitating patient-physician communication in routine clinical care to understand patient-reported symptoms.

30 Jan 2024·Rapid communications in mass spectrometry : RCM

Doping control approach: Identification of equine in vitro metabolites of voxelotor (GBT440), a hemoglobin S polymerization inhibitor

Article

Author: Karakka Kal, Abdul Khader ; Karatt, Tajudheen K ; Perwad, Zubair ; Subhahar, Michael Benedict ; Muhammed Ajeebsanu, Meleparappil ; Philip, Moses

Rationale:

Sickle cell disease, a debilitating genetic disorder affecting numerous newborns globally, has historically received limited attention in pharmaceutical research. However, recent years have witnessed a notable shift, with the Food and Drug Administration approving three innovative disease‐modifying medications. Voxelotor, also known as GBT440, is a promising compound that effectively prevents sickling, providing a safe approach to alleviate chronic hemolytic anemia in sickle cell disease. It is a novel, orally bioavailable small molecule that inhibits hemoglobin S polymerization by enhancing oxygen affinity to hemoglobin. The investigation demonstrated that voxelotor led to an unintended elevation of hemoglobin levels in healthy individuals by increasing serum erythropoietin levels.

Methods:

Voxelotor and its metabolites in an in vitro setting utilizing equine liver microsomes were discussed. Plausible structures of the identified metabolites were inferred through the application of liquid chromatography in conjunction with high‐resolution mass spectrometry.

Results:

Under the experimental conditions, a total of 31 metabolites were detected, including 16 phase I metabolites, two phase II metabolites, and 13 conjugates of phase I metabolites. The principal phase I metabolites were generated through processes such as hydroxylation, reduction, and dissociation. The presence of glucuronide and sulfate conjugates of the parent drug were also observed, along with hydroxylated, reduced, and dissociated analogs.

Conclusions:

The data acquired will accelerate the identification of voxelotor and related compounds, aiding in the detection of their illicit use in competitive sports. It is crucial to emphasize that the metabolites detailed in this manuscript were identified through in vitro experiments and their detection in an in vivo study may not be guaranteed.

News (Medical) associated with Voxelotor

14 Jun 2024

·www.biospace.com

Vertex Presents Positive Long-Term Data On CASGEVY™ (exagamglogene autotemcel) at the 2024 Annual European Hematology Association (EHA) Congress

Results from CLIMB-111, -121 and -131 accepted for oral presentation Data from these trials, with the longest follow-up of more than five years, demonstrate transformative, consistent and durable benefit of CASGEVY™ Safety pro with busulfan conditioning and autologous hematopoietic stem cell transplant BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced longer-term data for CASGEVY™ (exagamglogene autotemcel [exa-cel]) from global clinical trials in people with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, presented at the annual European Hematology Association (EHA) Congress, confirm the transformative, consistent and durable clinical benefits of CASGEVY over time. CASGEVY is the first and only approved CRISPR-based gene-editing therapy. The data being presented are from more than 100 patients (46 SCD; 56 TDT) treated with exa-cel in clinical trials, with the longest follow-up now extending more than 5 years. The efficacy results are consistent with the previously reported primary and key secondary endpoints analyses from these exa-cel studies and continue to demonstrate transformative clinical benefit with durable and stable levels of fetal hemoglobin (HbF) and allelic editing. “The transformative benefit seen in patients with sickle cell disease in the trial is impressive given the significant and cumulative burden of disease faced by people living with this blood disorder,” said Haydar Frangoul, M.D., M.S., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute and HCA Healthcare’s TriStar Centennial Children’s Hospital. “I am eager to offer this therapy and the opportunity of a potential functional cure to my eligible patients.” “The comprehensive data set presented today for adult and adolescent TDT patients adds to the growing body of evidence for CASGEVY, and it is important to now ensure the availability of this innovative treatment to patients in the real world as soon as possible,” said Franco Locatelli, M.D., Ph.D., Professor of Pediatrics at the Catholic University of the Sacred Heart of Rome, Director of the Department of Pediatric Hematology and Oncology at Bambino Gesù Children’s Hospital. “With the longest follow up now more than five years, alongside stable editing and sustained fetal hemoglobin levels, I have conviction in the durable benefit to the patients treated with CASGEVY.” New data presented from CASGEVY pivotal trials In SCD 36/39 (92.3%) evaluable patients (those with at least 16 months of follow-up) were free from vaso-occlusive crises (VOCs) for at least 12 consecutive months (VF12), consistent with the previously reported primary endpoint data. Mean duration of VOC-free was 27.9 months, with a maximum of 54.8 months. 38/39 (97.4%) patients with at least 16 months of follow-up were free from hospitalizations related to VOCs for at least 12 consecutive months (HF12), consistent with the previously reported key secondary endpoint data. In TDT 49/52 (94.2%) evaluable patients (those with at least 16 months of follow-up) were transfusion-independent for at least 12 consecutive months with a mean weighted hemoglobin of at least 9 g/dL (TI12), consistent with the previously reported primary endpoint data. Mean duration of transfusion independence was 31.0 months, with a maximum of 59.4 months. All TDT patients dosed with at least 16 months of follow up are transfusion free. Two of the three patients who did not achieve TI12 in CLIMB-111 achieved TI12 in the long-term follow-up study, CLIMB-131, and have been transfusion independent for over one year. The third has been transfusion free for 3.4 months. Both SCD and TDT patients reported sustained and clinically meaningful improvements in their quality of life, including physical, emotional, social/family and functional well-being, and overall health status. In both SCD and TDT patients, edited levels of BCL11A alleles were stable over time in bone marrow and peripheral blood indicating successful editing in the long-term hematopoietic stem cells. All patients engrafted neutrophils and platelets after exa-cel infusion. The safety pro exa-cel was generally consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant. These longer-term data for CASGEVY from the CLIMB clinical trials will be shared as outlined below: Abstracts S273 and S274 will be oral presentations entitled “Exagamglogene Autotemcel For Severe Sickle Cell Disease,” and “Exagamglogene Autotemcel For Transfusion-Dependent Beta-Thalassemia,” on Sunday, June 16 at 12:15 CEST and 12:30 CEST, respectively. Abstracts P1493 and P1525 will be poster presentations entitled “Health-Related Quality Of Life Improvements After Exagamglogene Autotemcel In Patients With Severe Sickle Cell Disease,” and “Health-Related Quality Of Life Improvements After Exagamglogene Autotemcel In Patients With Transfusion-Dependent Βeta-Thalassemia,” on Friday, June 14 at 18:00 CEST. These presentations will include updated pivotal trial data from patients treated with CASGEVY in CLIMB-111 and CLIMB-121 and followed in CLIMB-131. Vertex will also share five health economics abstracts at the EHA Congress. Abstract P1483 is entitled “Adherence, Treatment Use, and Clinical Outcomes in Patients With Sickle Cell Disease With Recurrent Vaso-Occlusive Crises Treated With L-Glutamine, Voxelotor, or Crizanlizumab in the United States.” Abstract P1506 is entitled “Mortality and Clinical Complications Among Patients with Sickle Cell Disease With Recurrent VOCs in Canada.” Abstract P1507 is entitled “Treatment Utilization and Clinical Complications in Patients with Sickle Cell Disease Receiving Frequent Red Blood Cell Transfusions in the United States.” Abstract P2191 is entitled “Clinical Complications and Treatment Use Among Patients With Sickle Cell Disease With Recurrent Vaso-Occlusive Crises in the Netherlands.” Abstract PB3248 is entitled “Clinical Complications Among Patients With Transfusion-Dependent Beta-Thalassemia in the Netherlands.” About Sickle Cell Disease (SCD) SCD is a debilitating, progressive, life shortening genetic disease. SCD patients report health-related quality of life scores well below the general population and significant health care resource utilization. SCD affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. SCD causes severe pain, organ damage and shortened life span due to misshapen or “sickled” red blood cells. The clinical hallmark of SCD is vaso-occlusive crises (VOCs), which are caused by blockages of blood vessels by sickled red blood cells and result in severe and debilitating pain that can happen anywhere in the body at any time. SCD requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. In Europe, the mean age of death for patients living with SCD is around 40 years. Stem cell transplant from a matched donor is a potentially curative option but is only available to a small fraction of people living with SCD because of the lack of available donors. About Transfusion-Dependent Beta Thalassemia (TDT) TDT is a serious, life-threatening genetic disease. TDT patients report health-related quality of life scores below the general population and significant health care resource utilization. TDT requires frequent blood transfusions and iron chelation therapy throughout a person’s life. Due to anemia, patients living with TDT may experience fatigue and shortness of breath, and infants may develop failure to thrive, jaundice and feeding problems. Complications of TDT can also include an enlarged spleen, liver and/or heart, misshapen bones and delayed puberty. TDT requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. In Europe, the mean age of death for patients living with TDT is 50-55 years. Stem cell transplant from a matched donor is a potentially curative option but is only available to a small fraction of people living with TDT because of the lack of available donors. About CASGEVY™ (exagamglogene autotemcel [exa-cel]) CASGEVY™ is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for certain indications in multiple jurisdictions for eligible patients. About the CLIMB Studies The ongoing Phase 1/2/3 open-label trials, CLIMB-111 and CLIMB-121, are designed to assess the safety and efficacy of a single dose of CASGEVY in patients ages 12 to 35 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now closed for enrollment. Patients will be followed for approximately two years after CASGEVY infusion. Each patient will be asked to participate in the ongoing long-term, open-label trial, CLIMB-131. CLIMB-131 is designed to evaluate the safety and efficacy of CASGEVY in patients who received CASGEVY in other CLIMB studies. The trial is designed to follow patients for up to 15 years after CASGEVY infusion. U.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR CASGEVY (exagamglogene autotemcel) WHAT IS CASGEVY? CASGEVY is a one-time therapy used to treat people aged 12 years and older with: sickle cell disease (SCD) who have frequent vaso-occlusive crises or VOCs beta thalassemia (β-thalassemia) who need regular blood transfusions CASGEVY is made specifically for each patient, using the patient’s own edited blood stem cells, and increases the production of a special type of hemoglobin called hemoglobin F (fetal hemoglobin or HbF). Having more HbF increases overall hemoglobin levels and has been shown to improve the production and function of red blood cells. This can eliminate VOCs in people with sickle cell disease and eliminate the need for regular blood transfusions in people with beta thalassemia. IMPORTANT SAFETY INFORMATION What is the most important information I should know about CASGEVY? After treatment with CASGEVY, you will have fewer blood cells for a while until CASGEVY takes hold (engrafts) into your bone marrow. This includes low levels of platelets (cells that usually help the blood to clot) and white blood cells (cells that usually fight infections). Your doctor will monitor this and give you treatment as required. The doctor will tell you when blood cell levels return to safe levels. Tell your healthcare provider right away if you experience any of the following, which could be signs of low levels of platelet cells: severe headache abnormal bruising prolonged bleeding bleeding without injury such as nosebleeds; bleeding from gums; blood in your urine, stool, or vomit; or coughing up blood Tell your healthcare provider right away if you experience any of the following, which could be signs of low levels of white blood cells: fever chills infections You may experience side effects associated with other medicines administered as part of the treatment regimen for CASGEVY. Talk to your physician regarding those possible side effects. Your healthcare provider may give you other medicines to treat your side effects. How will I receive CASGEVY? Your healthcare provider will give you other medicines, including a conditioning medicine, as part of your treatment with CASGEVY. It’s important to talk to your healthcare provider about the risks and benefits of all medicines involved in your treatment. After receiving the conditioning medicine, it may not be possible for you to become pregnant or father a child. You should discuss options for fertility preservation with your healthcare provider before treatment. STEP 1: Before CASGEVY treatment, a doctor will give you mobilization medicine(s). This medicine moves blood stem cells from your bone marrow into the blood stream. The blood stem cells are then collected in a machine that separates the different blood cells (this is called apheresis). This entire process may happen more than once. Each time, it can take up to one week. During this step rescue cells are also collected and stored at the hospital. These are your existing blood stem cells and are kept untreated just in case there is a problem in the treatment process. If CASGEVY cannot be given after the conditioning medicine, or if the modified blood stem cells do not take hold (engraft) in the body, these rescue cells will be given back to you. If you are given rescue cells, you will not have any treatment benefit from CASGEVY. STEP 2: After they are collected, your blood stem cells will be sent to the manufacturing site where they are used to make CASGEVY. It may take up to 6 months from the time your cells are collected to manufacture and test CASGEVY before it is sent back to your healthcare provider. STEP 3: Shortly before your stem cell transplant, your healthcare provider will give you a conditioning medicine for a few days in hospital. This will prepare you for treatment by clearing cells from the bone marrow, so they can be replaced with the modified cells in CASGEVY. After you are given this medicine, your blood cell levels will fall to very low levels. You will stay in the hospital for this step and remain in the hospital until after the infusion with CASGEVY. STEP 4: One or more vials of CASGEVY will be given into a vein (intravenous infusion) over a short period of time. After the CASGEVY infusion, you will stay in hospital so that your healthcare provider can closely monitor your recovery. This can take 4-6 weeks, but times can vary. Your healthcare provider will decide when you can go home. What should I avoid after receiving CASGEVY? Do not donate blood, organs, tissues, or cells at any time in the future What are the possible or reasonably likely side effects of CASGEVY? The most common side effects of CASGEVY include: Low levels of platelet cells, which may reduce the ability of blood to clot and may cause bleeding Low levels of white blood cells, which may make you more susceptible to infection Your healthcare provider will test your blood to check for low levels of blood cells (including platelets and white blood cells). Tell your healthcare provider right away if you get any of the following symptoms: fever chills infections severe headache abnormal bruising prolonged bleeding bleeding without injury such as nosebleeds; bleeding from gums; blood in your urine, stool, or vomit; or coughing up blood These are not all the possible side effects of CASGEVY. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. General information about the safe and effective use of CASGEVY Talk to your healthcare provider about any health concerns. Please see full Prescribing Information including Patient Information for CASGEVY. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 14 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on LinkedIn, YouTube and Twitter/X. (VRTX-GEN) Vertex Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the statements by Haydar Frangoul, M.D., M.S., and Franco Locatelli, M.D., Ph.D., in this press release, and statements regarding our expectations for and the anticipated benefits of CASGEVY, our plans to share longer-term data for CASGEVY from the CLIMB clinical trials and to share health economics abstracts at the EHA Congress, and our plans for and design of the CLIMB studies. While we believe the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy, and other reasons, and other risks listed under the heading “Risk Factors” in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at and available through the company's website at . You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. View source version on businesswire.com: Contacts Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@vrtx.com Media: mediainfo@vrtx.com or International: +44 20 3204 5275 or U.S.: 617-341-6992 or Heather Nichols: +1 617-839-3607 Source: Vertex Pharmaceuticals Incorporated View this news release online at:

Clinical ResultCell TherapyDrug ApprovalClinical Study

03 May 2024

·firstwordpharma.com

Price cut allows Pfizer’s sickle cell therapy Oxbryta to rollout on NHS

A price cut has allowed Pfizer’s Oxbryta (voxelotor) to be recommended for use on the NHS to treat people with haemolytic anaemia caused by sickle cell disease (SCD), following an initial rejection last year. The once-daily oral drug – which the company gained through its purchase of Global Blood Therapeutics - was approved in the UK in 2022 for people aged 12 and older.However, previous draft guidance from the National Institute for Health and Care Excellence rebuffed the treatment due to uncertainties over supporting evidence. NICE said Friday in final guidance that a cut to Oxbryta’s cost allows some of these reservations to be “mitigated,” with the drug to be funded immediately via NHS England’s Innovative Medicines Fund.Helen Knight, director of medicines evaluation at NICE, remarked “throughout our appraisal…the independent committee has always considered that it has the potential to address the need for effective treatments for SCD.” Knight added “we are therefore delighted that the company has reduced the price of [Onbryxta] to a level that properly reflects the uncertainty in the evidence.”Specifically, NICE said that its appraisal committee agreed that greater uncertainty in the estimates of clinical effectiveness that supported economic modelling for Onbryxta could be accepted in light of “substantial disadvantages” experienced by people with SCD, and a higher cost-effectiveness estimate than usual could be considered acceptable.“By improving the quality of life and reducing the need for hospital care, this new treatment option also has the potential to free up doctors, nurses and other clinicians to better support other patients, so we are acting fast to get it to the front-line immediately through our Innovative Medicines Fund,” commented Amanda Pritchard, NHS chief executive.NICE recommended Oxbryta with or without another SCD treatment hydroxycarbamide, noting that around 4000 people in England are set to benefit from Pfizer’s drug.

Drug ApprovalCell TherapyAccelerated Approval

03 May 2024

·www.pharmaceutical-technology.com

NHS to provide Pfizer’s Voxelotor to treat sickle cell disease

Sickle cell disease is caused by a genetic mutation that results in red blood cells becoming sickle-shaped, leading to haemolytic anaemia. Credit: Victor Josan / Shutterstock.com. The UK’s National Health Service (NHS) has announced plans to offer Pfizer ‘s Voxelotor (Oxbryta), a new treatment option for sickle cell disease patients. The latest development comes after the National Institute for Health and Care Excellence (NICE) granted approval for the treatment in patients aged 12 years and above. The decision will benefit up to 4,000 patients in England with the genetic blood disorder. Voxelotor, which will be available in tablet form for daily intake, can reduce the frequency of blood transfusions and hospital visits. It offers a new treatment option for those who have had severe reactions to treatment or prefer not to undergo blood transfusions. See Also: Psilocybin effective in treating depression BioCryst Pharmaceuticals gets grant for inhibitors of the complement system for disease treatment The NHS will immediately fund Voxelotor through the Innovative Medicines Fund, ensuring rapid access for patients. Clinical trials have demonstrated that Voxelotor could improve anaemia and reduce the necessity for repeated blood transfusions. In trials, 51% of participants treated with Voxelotor experienced an increase in haemoglobin levels, reducing their symptoms and enhancing their overall quality of life. 74% of patients taking the drug reported a significant improvement in their well-being. Sickle cell disease is caused by a genetic mutation that results in red blood cells becoming sickle-shaped, leading to haemolytic anaemia, in which red blood cells are destroyed faster than they can be replaced. NHS chief executive Amanda Pritchard stated: “The NHS has worked hard to make this life-changing treatment available at a fair price for the taxpayer, as part of our wider drive to improve the quality and experience of care for sickle cell patients, and tackle the stigma and inequalities they have told me they face. “By improving the quality of life and reducing the need for hospital care, this new treatment option also has the potential to free up doctors, nurses and other clinicians to better support other patients, so we are acting fast to get it to the frontline immediately through our Innovative Medicines Fund.”

Drug ApprovalClinical ResultAHA

100 Deals associated with Voxelotor

External Link

KEGG	Wiki	ATC	Drug Bank
D11330	-	B03XA	DB14975

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Anemia, Hemolytic	EU	Pfizer Europe MA EEIG	14 Feb 2022
Anemia, Hemolytic	IS	Pfizer Europe MA EEIG	14 Feb 2022
Anemia, Hemolytic	LI	Pfizer Europe MA EEIG	14 Feb 2022
Anemia, Hemolytic	NO	Pfizer Europe MA EEIG	14 Feb 2022
Anemia, Sickle Cell	US	Global Blood Therapeutics, Inc.	25 Nov 2019

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Semicircular Canal Dehiscence	Phase 2	GB	Pfizer Inc.	09 Jan 2020
Hypoxia	Phase 2	US	Global Blood Therapeutics, Inc.	01 Jun 2016
Idiopathic Pulmonary Fibrosis	Phase 2	US	Global Blood Therapeutics, Inc.	01 Jun 2016
Liver Diseases	Phase 1	US	Pfizer Inc.	17 Mar 2017
Renal Insufficiency	Phase 1	US	Global Blood Therapeutics, Inc.	01 Nov 2016

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


EHA2024	Not Applicable	Anemia, Sickle Cell HbSS \| HbSβ0-thalassemia \| Hb	-	Voxelotor 1500 mg QD	hwslsgsryq(kbznmspudp) = CMRO2 were unaffected vuxbhwwjee (froanbzonn )	Positive	14 May 2024
NCT05228834 (CTgov)	Phase 3	Anemia, Sickle Cell	1	Placebo	snovsdjgyq(ibzyzjakkt) = dghdcqdblh udjszpjhqh (hxhblykgsc, ugmuotokcw - ssniwzskoe) View more	-	02 May 2024
NCT04218084 (HOPE-KIDS 2, ASH2023)	Phase 3	Anemia, Sickle Cell	708	Voxelotor	yveyapmopz(ekctndpbnv) = yecupmwpzg ldgdfjfrwp (dtkoexurvu )	-	11 Dec 2023
NCT05199766 (ASH2023)	Phase 2	-	25	Voxelotor 1500mg	nbheexfnmh(xwhonkrlse) = rsqqpthbgj zhkmkohmgw (hxqnlrxing ) View more	-	11 Dec 2023
NCT04930445 (PROSPECT, ASH2023)	Not Applicable	Anemia, Sickle Cell	85	Voxelotor	upqhwxkxvy(dehemhcryp) = vxbifoxlxj fnndwbwmlw (gghjyegvpf )	-	10 Dec 2023
ASH2023	Not Applicable	Anemia, Sickle Cell	19	Voxelotor-treated patients	sgqbawlwaw(mnjjsnesny) = hchcrlddop ehlvpexvbn (ofqpvkvjqv )	-	09 Dec 2023
NCT04400487 (CTgov)	Phase 4	Anemia, Sickle Cell	25	Voxelotor	mnmiscnqyw(pmeocffgkj) = guuqjbgdaj qostyhwogk (ynqzhcbbgo, authgzikqp - lkwrradigo) View more	-	21 Nov 2023
EHA2023	Not Applicable	Polycythemia	15	Voxelotor 1500 mg	aljzkaizrs(ywkjxtzupl) = yvagjzfwdg hgdhhoydtl (ltkwtzccqa ) View more	-	08 Jun 2023
NCT04400487 (ActIVe, EHA2023)	Phase 4	Anemia, Sickle Cell	25	Voxelotor 1500 mg	iiwmglpfzv(xxlyltherk) = lgzthbyxbr ieiugryrac (uejameotuf ) View more	-	08 Jun 2023
EHA2023	Not Applicable	Anemia, Sickle Cell	-	Voxelotor	dgteohradz(axylytcjqg) = rioubsgzpy aupmnjhzst (frlbrobbun ) View more	-	08 Jun 2023

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