Pfizer Halts Sales of Sickle Cell Disease Drug - Implications Explained

The original sentence: "A version of this article first appeared in CNBC's Healthy Returns newsletter, which brings the latest health-care news straight to your inbox. Subscribe here to receive future editions." Rewritten sentence: "This article was initially published in CNBC's Healthy Returns newsletter, providing you with the latest health-care news directly to your inbox. To receive future editions, subscribe here."

Greetings and have a wonderful Tuesday! Today, we're exploring a surprising development from .

The pharmaceutical giant announced the withdrawal of its sickle cell disease therapy, Oxbryta, from worldwide markets, surprising doctors, patients, and investors.

The drug Oxbryta is one of several treatments for an inherited blood disorder. It was granted accelerated approval by the U.S. Food and Drug Administration in 2019, but further trials are needed to confirm its benefits to patients.

Pfizer's $5.4 billion acquisition of Global Blood Therapeutics in 2022 included Oxbryta as one of its main features.

Red blood cells in individuals with sickle cell disease become misshapen half-moons that can get stuck in blood vessels, leading to restricted blood flow and pain crises. This condition affects approximately 100,000 people in the U.S., with a disproportionate number of Black individuals being affected, according to data from the Centers for Disease Control and Prevention.

Pfizer announced on Wednesday that the decision to withdraw Oxbryta was due to new data showing a higher risk of deaths and complications in patients taking the once-daily pill. The company stated in a release that the "totality of clinical data" on Oxbryta now indicates that its overall benefit "no longer outweighs the risk" in the patient population for which the drug is approved.

Pfizer is also discontinuing all studies and access programs related to the treatment.

On Saturday, the FDA advised healthcare professionals to cease prescribing Oxbryta and recommended patients and caregivers to consult their healthcare professional about switching to a different treatment option.

On Thursday, European regulators recommended suspending the marketing authorization of Oxbryta after patients in trials experienced higher rates of pain crises following treatment with the drug.

Concerns are being raised about Oxbryta's withdrawal.

The sudden disappearance of Oxbryta from the market has left doctors, sickle cell disease patients, and patient advocates in a state of confusion and urgency, as they search for more information on the decision and guidance on how to proceed, according to STAT's report on Friday. While discontinuing Oxbryta may put patients at risk, it is unclear what they may experience if they abruptly stop treatment with the drug.

The National Alliance of Sickle Cell Centers advised patients not to abruptly stop taking Oxbryta in a statement last week. Instead, the group urged patients to consult with their doctors and gradually taper off the medication.

The withdrawal of Oxbryta will have a "significant impact" on patients with sickle cell disease, who have been historically underrepresented, according to BMO Capital Markets analyst Evan Seigerman in a research note last week.

The FDA approved two gene therapies for sickle cell disease last year, providing hope for patients. However, health officials have yet to determine how to make these costly treatments accessible to all patients equitably.

The cost of gene therapy Casgevy is $2.2 million per patient, while Lyfgenia's treatment lists for $3.1 million per patient.

Some Wall Street analysts believe that Pfizer's withdrawal of Oxbryta could speed up the timeline for clinical trials on experimental treatments for sickle disease being developed by other companies such as and s.

If mitapivat, the experimental drug of Agios, proves to be effective in reducing pain crises in clinical trials, it is expected to facilitate a smoother regulatory review process, particularly given the growing patient need for an alternative to Pfizer's drug, according to Piper Sandler analyst Christopher Raymond in a research note last week.

The financial impact of Pfizer withdrawing Oxbryta is "somewhat modest," according to Guggenheim analysts in a note last week.

Despite modest sales of $328 million last year, analysts predict Oxbryta sales to reach $750 million by the end of the decade based on FactSet consensus estimates.

Pfizer's decision to withdraw Oxbryta may raise questions about the company's ability to grow in the future, particularly as it faces several drug patent expirations and other challenges to its current growth drivers, according to Guggenheim. Additionally, the withdrawal of Oxbryta raises questions about what will happen to Pfizer's other sickle cell disease treatment in development, GBT-601.

The oral drug acquired by Pfizer through the Global Blood Therapeutics deal is viewed as a potential successor to Oxbryta.

Rewritten sentence: Please provide any tips, suggestions, story ideas, and data to Annika at [email protected].

A new bill has been introduced by senators to strengthen health-care cybersecurity after significant attacks.

On Thursday, Senators Ron Wyden and Mark Warner presented a bill to set stringent cybersecurity guidelines in the health-care industry.

The "Health Infrastructure Security and Accountability Act" proposes that the Department of Health and Human Services will develop and enforce new standards for health plans, providers, business associates, and clearinghouses.

Healthcare data is highly sensitive and valuable, making it an attractive target for malicious actors. Over the past 14 years, the number of cyberattacks in the healthcare industry has been steadily increasing, with a record 725 data breaches reported in 2020, according to The HIPAA Journal.

In 2024, the journal reported 491 data breaches of more than 500 health records, including the ransomware attack on Change Healthcare that disrupted the health-care industry.

The company, which is owned by Healthcare, provides payment and revenue cycle management tools, as well as other solutions such as electronic prescription software. It processes over 15 billion billing transactions annually, and one in three patient records passes through its systems, according to its website.

On February 21, hackers breached Change Healthcare's information technology systems, causing UnitedHealth to shut down the affected systems. This left many doctors unable to fill prescriptions or receive payment for their services, forcing them to use their personal savings to keep their practices running.

In May, UnitedHealth CEO Andrew Witty testified before the Senate Finance Committee and apologized for the attack. Later that day, he estimated that data from approximately one-third of Americans could have been compromised.

As per Wyden's statement in a release on Thursday, legislation is being proposed due to the failure of megacorporations like UnitedHealth in basic cybersecurity, resulting in harm to American families.

The Health Insurance Portability and Accountability Act (HIPAA) safeguards patient data, and organizations face penalties for violations. Under the new legislation, Wyden and Warner propose to eliminate the current limit on HIPAA fines, allowing regulators to enforce stricter cybersecurity standards on large corporations.

Before this legislation can be enacted, it must first pass through both houses of Congress and receive presidential approval.

You can read a full copy of the legislative text here.

Rewritten sentence: Please provide any tips, suggestions, story ideas, and data to Ashley at [email protected].