Pfizer voluntarily withdraws all lots of OXBRYTA® (voxelotor) for the treatment of sickle cell disease from the global market

Pfizer Inc. (NYSE: PFE) today announced 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 clinical trials of voxelotor and expanded access programs globally.

Pfizer’s decision is based on the totality of clinical data, which now indicate that the overall benefit of OXBRYTA no longer outweighs the risk in the approved patient population with sickle cell disease. The data suggest an imbalance in vaso-occlusive crises and fatal events that require further evaluation. Pfizer has notified regulatory authorities of these findings and its decision to voluntarily withdraw OXBRYTA from the market and discontinue distribution and clinical studies while it continues to review the available data and investigate the findings.

“The safety and well-being of patients is of paramount 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 suffering 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 treatments as we continue to investigate the findings of our data review.”

Patients, physicians, pharmacists or other healthcare professionals with additional questions about OXBRYTA should contact Pfizer Medical Information at 1-800-438-1985. The company will keep patients, regulators, investigators and clinicians informed of actions and appropriate next steps for OXBRYTA.

The company does not expect this event to impact its full-year 2024 financial guidance.

About sickle cell disease

SCD is a lifelong, debilitating hereditary blood disorder in which polymerization of hemoglobin S leads to sickling of red blood cells, resulting in vascular inflammation and hemolytic anemia. Vascular inflammation, along with sickled RBCs, 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 the 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 large unmet need for therapies that address the root cause of SCD and its acute and chronic complications. Although rare in developed markets, there are 4.5 million people living with SCD worldwide and over 45 million people living with sickle cell disease. SCD is most prevalent in people with ancestral origins in sub-Saharan Africa, although it also occurs in people of Hispanic, South Asian, Southern European, and Middle Eastern descent.

About OXBRYTA^® (voxelotor)

OXBRYTA (voxelotor) is an oral, once-daily therapy for patients with sickle cell disease (SCD). OXBRYTA works by increasing the affinity of hemoglobin for oxygen. OXBRYTA inhibits the polymerization of sickle cell hemoglobin and the resulting sickling and destruction of red blood cells, leading to hemolysis and hemolytic anemia, which are primary pathologies that every person with SCD faces.

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 12 years and older. In December 2021, the FDA expanded the approved use of OXBRYTA for the treatment of SCD in patients 4 years and older in the U.S.

OXBRYTA has been granted Priority Medicines (PRIME) designation by the European Medicines Agency (EMA) and has been designated as an orphan medicinal product by the European Commission (EC) for the treatment of patients with SCD. In February 2022, the EC granted a marketing authorisation for OXBRYTA for the treatment of haemolytic anaemia due to SCD in adult and paediatric patients aged 12 years and older as monotherapy or in combination with hydroxycarbamide (hydroxyurea). Since its first approval in 2019, OXBRYTA has been approved in more than 35 countries worldwide.

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 package 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 seek immediate medical attention if they develop a rash, hives, shortness of breath (difficulty breathing), or swelling of the face.

The most common side effects of OXBRYTA are headache, diarrhea, stomach (abdominal) pain, nausea, rash or hives, and fever. The most common side effects of OXBRYTA in children 4 to less than 12 years of age are fever, vomiting, rash, stomach (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 liver problems; if they are pregnant or planning to become pregnant, because it is not known if OXBRYTA can harm an unborn baby; or if they are breast-feeding or planning to breast-feed, because it is not known if OXBRYTA can pass into breast milk or harm a baby. Patients should not breast-feed during treatment with OXBRYTA and for at least 2 weeks after the last dose.

Patients should tell their healthcare provider about all medications they are taking, including prescription and over-the-counter medications, vitamins, and herbal supplements. Some medications can affect how OXBRYTA works. OXBRYTA can also affect how other medications work and can 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.

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 in 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 leading innovative biopharmaceutical companies, we partner with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For 175 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.Pfizer.com. Additionally, visit us at www.Pfizer.com and follow us on X at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer for more information.

DISCLOSURE NOTICE:

The information in this press release is as of September 25, 2024. Pfizer assumes no obligation to update the forward-looking statements contained in this press release as a result of new information or future events or developments.

This press release contains forward-looking information regarding a voluntary withdrawal of all lots of OXBRYTA (voxelotor) from the global markets, including the anticipated impact on the Company’s full year 2024 guidance, that involves significant 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 relating to the future of OXBRYTA (voxelotor), as well as uncertainties relating to other sickle cell disease assets in our portfolio; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, initiation 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 trials; decisions by regulatory authorities affecting 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; the uncertainties inherent in business and financial planning, including, but not limited to, risks relating to Pfizer’s business and prospects, legal proceedings, adverse developments in Pfizer’s markets or adverse developments in the U.S. or global capital markets, credit markets, the regulatory environment or the economy generally; uncertainties relating to 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 subsequent reports on Form 10-Q, including the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results,” as well as in subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and are available at www.sec.gov And www.pfizer.nl.

Category:Prescription Medicines