Pfizer Inc. (NYSE: PFE) 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 OXBRYTA 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 OXBRYTA 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."
Patients, physicians, pharmacists, or other healthcare professionals with additional questions about OXBRYTA should contact Pfizer Medical Information 1-800-438-1985. The company will keep patients, regulatory authorities, investigators and clinicians informed about actions and appropriate next steps for OXBRYTA.
The company does not anticipate that this event will impact its full-year 2024 financial guidance.
About Sickle Cell Disease
SCD is a lifelong, debilitating inherited blood disorder in which hemoglobin S polymerization leads to red blood cell 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. Complications of SCD begin in early childhood and are associated with shortened life expectancy. 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.
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, there are 4.5 million people living with SCD globally and more than 45 million people living with 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)
OXBRYTA (voxelotor) is an oral, once-daily therapy for patients with sickle cell disease (SCD). OXBRYTA works by increasing hemoglobin's affinity for oxygen. OXBRYTA 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. In December 2021, the FDA expanded the approved use of OXBRYTA for the treatment of SCD in patients 4 years of age and older in the U.S.
OXBRYTA was granted Priority Medicines (PRIME) designation by the European Medicines Agency (EMA) and designated an orphan medicinal product for the treatment of patients with SCD by the European Commission (EC). In February 2022, the EC granted Marketing Authorization for OXBRYTA 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, OXBRYTA has been approved in over 35 countries globally.
Important Safety Information
OXBRYTA should not be taken if the patient has had an allergic reaction to voxelotor or any of the ingredients in OXBRYTA. See the end of the patient leaflet for a list of the ingredients in OXBRYTA. OXBRYTA can cause serious side effects, including serious allergic reactions. Patients should tell their healthcare provider or get emergency medical help right away if they get rash, hives, shortness of breath (difficult breathing) or swelling of the face.
The most common side effects of OXBRYTA include headache, diarrhea, stomach-area (abdominal) pain, nausea, rash or hives, and fever. The most common side effects of OXBRYTA in children ages 4 to less than 12 years of age include fever, vomiting, rash, stomach-area (abdominal) pain, diarrhea, and headache. These are not all the possible side effects of OXBRYTA. Before taking OXBRYTA, patients should tell their healthcare provider about all medical conditions, including if they have liver problems; if they are pregnant or plan to become pregnant as it is not known if OXBRYTA can harm an unborn baby; or if they are breastfeeding or plan to breastfeed as it is not known if OXBRYTA can pass into breastmilk or if it can harm a baby. Patients should not breastfeed during treatment with OXBRYTA and for at least 2 weeks after the last dose.
Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins and herbal supplements. Some medicines may affect how OXBRYTA works. OXBRYTA may also affect how other medicines work and may affect the results of certain blood tests.
Patients are advised to call their doctor for medical advice about side effects. Side effects can be reported to FDA at 1-800-FDA-1088. Side effects can also be reported at 1-833-428-4968.
Full Prescribing Information for OXBRYTA is available at OXBRYTA.com .
