Walton Oaks, 25th September 2024 - Pfizer Inc announced today that it is voluntarily withdrawing all lots of OXBRYTA® (voxelotor) for the treatment of sickle cell disease (SCD) at this time, in all markets where it is approved. Pfizer is also discontinuing all active voxelotor clinical trials and expanded access programs worldwide.  
 
Pfizer's decision is based on the totality of clinical data that now indicates the overall benefit of voxelotor no longer outweighs the risk in the approved sickle cell patient population. The data suggest an imbalance in vaso-occlusive crises and fatal events which require further assessment. Pfizer has notified regulatory authorities about these findings and its decision to voluntarily withdraw voxelotor from the market and discontinue distribution and clinical studies while further reviewing the available data and investigating the findings.  
 
“The safety and well-being of patients is of the utmost importance to Pfizer, and we believe this action is in the best interest of patients,” said Aida Habtezion, Chief Medical Officer and Head of Worldwide Medical and Safety at Pfizer. “Our primary concern is for patients who suffer from SCD, which remains a very serious and difficult-to-treat disease with limited treatment options. We advise patients to contact their physicians to discuss alternative treatment while we continue to investigate the findings from our review of the data.” 
 
UK Patients, physicians, pharmacists, or other healthcare professionals with additional questions about voxelotor should contact Pfizer Medical Information 01304 616161. The Company will keep patients, regulatory authorities, investigators and clinicians informed about actions and appropriate next steps for voxelotor. 

ABOUT SICKLE CELL DISEASE 
SCD is a lifelong, debilitating inherited blood disorder in which hemoglobin S polymerization leads to red blood cell (RBC) sickling resulting in vascular inflammation and hemolytic anemia. Vascular inflammation, together with sickled RBC’s can lead to acute pain crises, or vaso-occlusive crises, and progressive end organ damage, including stroke.¹ Complications of SCD begin in early childhood and are associated with shortened life expectancy.¹ Early intervention and treatment of SCD have shown potential to modify the course of this disease, reduce symptoms and events, prevent long-term organ damage, and extend life expectancy.¹
 
Historically, there has been a high unmet need for therapies that address the root cause of SCD and its acute and chronic complications. While rare in developed markets, globally millions of people live with SCD or have the sickle cell trait. SCD occurs particularly among those whose ancestors are from sub-Saharan Africa, though it also occurs in people of Hispanic, South Asian, Southern European and Middle Eastern ancestry. 

ABOUT OXBRYTA^® (voxelotor) 
Voxelotor is an oral, once-daily therapy indicated 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. Voxelotor works by increasing hemoglobin’s affinity for oxygen. Voxelotor inhibits sickle hemoglobin polymerization and the resultant sickling and destruction of red blood cells leading to hemolysis and hemolytic anemia, which are primary pathologies faced by every single person living with SCD.  
 
In 2019, the U.S. Food and Drug Administration (FDA) granted accelerated approval for OXBRYTA tablets for the treatment of SCD in adults and children ages 12 years and older.  
 
Voxelotor was granted Priority Medicines (PRIME) designation by the EMA and designated an orphan medicinal product for the treatment of patients with SCD by the European Commission (EC). In February 2022, the European Commission (EC) granted Marketing Authorization for voxelotor for the treatment of hemolytic anemia due to SCD in adult and pediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide (hydroxyurea). Since its first approval in 2019, voxelotor has been approved in over 35 countries globally.  
 
In July 2022, the MHRA issued marketing authorisation for voxelotor. 

Important safety information  
 The most common adverse reactions include headache (31.8%), diarrhoea (22.7%) and abdominal pain (22.7%). Serious adverse reactions include headache (1.1%) and drug hypersensitivity (1.1%). Permanent discontinuation due to an adverse reaction occurred in 2.3% of patients.² 'For additional safety information, please refer to the SmPC. 
   
Patients taking voxelotor should talk to their doctor if they experience any side effects. This includes any possible side effects not listed in the package leaflet.  
 
▼ This medicine is subject to additional monitoring. This will allow quick identification of new safety information. Side effects can be reported directly via the Yellow Card Scheme at https://yellowcard.mhra.gov.uk/ or search for MHRA Yellow Card in the Google Play or Apple App Store. By reporting side effects, you can help provide more information on the safety of this medicine. 

About Pfizer: Breakthroughs That Change Patients’ Lives
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. In the UK, Pfizer has its business headquarters in Surrey and is a major supplier of medicines to the NHS. To learn more about our commitments, please visit us at www.pfizer.co.uk or follow us on Twitter (@Pfizer_UK), Facebook (@PfizerUK) and Instagram (@pfizeruk).

DISCLOSURE NOTICE: 
The information contained in this release is as of September 25, 2024. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments. 
 
This release contains forward-looking information about a voluntary withdrawal of all lots of OXBRYTA (voxelotor) from worldwide markets, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the future of OXBRYTA (voxelotor), as well as uncertainties related to other sickle cell disease assets in our portfolio; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; risks associated with interim data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of OXBRYTA (voxelotor) or other sickle cell disease assets in our portfolio; uncertainties regarding the impact of COVID-19 on our business, operations and financial results; and competitive developments. 
 
A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023, and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results,” as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov and www.pfizer.co.uk.