Pfizer withdraws sickle cell anemia drug from the market

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Hello and happy Tuesday! Today, we are analyzing a shocking movement of Pfizer.

The pharmaceutical giant announced last week that it would voluntarily withdraw its sickle cell anemia therapy, Oxbryta, from global markets, to the surprise of doctors, patients and investors.

Here's why the drug is important: Oxbryta is one of at least six treatments for this inherited blood disorder. The drug first gained accelerated approval from the U.S. Food and Drug Administration in 2019, requiring more trials to confirm its benefits for patients.

Oxbryta was one of the centerpieces of Pfizer's $5.4 billion acquisition of Global Blood Therapeutics in 2022.

Sickle cell anemia causes red blood cells to turn into misshapen crescents that get stuck inside blood vessels, which can restrict blood flow and cause what is known as a pain crisis. It affects approximately 100,000 people in the United States, many of whom are black, according to data from the Centers for Disease Control and Prevention.

The company said Wednesday that the decision to withdraw Oxbryta was based on data showing an increased risk of deaths and complications in patients treated with the once-daily pill. In a statement, Pfizer said the “totality of the 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.

As part of that measure, Pfizer will also suspend all studies and access programs related to the treatment.

On Saturday, the FDA urged health care professionals to stop prescribing Oxbryta. The agency also said patients and caregivers should contact their health care professional to stop the medication and begin another treatment option.

European regulators also said Thursday that patients in trials had higher rates of pain crises after starting treatment with Oxbryta than before taking it. Those regulators recommended suspending the marketing authorization of the drug.

All that may seem simple. But Oxbryta's withdrawal raises concerns.

His sudden absence from the market It leaves doctors, sickle cell patients and patient advocates scrambling for more information about the decision and guidance on what they should do next, STAT reported Friday. And while taking Oxbryta could put patients at risk, it's not entirely clear what they might experience if they abruptly stop taking the drug.

In a statement last week, the National Alliance of Anthropology Centers urged patients not to abruptly stop taking Oxbryta. The group, which supports health centers that administer treatments for the disorder, urged all patients currently taking Oxbryta to make an appointment with their doctor and develop a plan to taper off the medication.

The withdrawal of Oxbryta will be a “significant blow” to sickle cell patients “who have historically been underserved,” BMO Capital Markets analyst Evan Seigerman wrote in a research note last week.

Last year, the FDA approved two gene therapies to treat sickle cell anemia, a landmark decision that gave hope to patients suffering from this debilitating disease. But health officials have so far struggled to find a way to provide equitable access to the expensive treatments.

Vertex Pharmaceuticals'Casgevy gene therapy costs $2.2 million per patient, and Blue Bird BiographyLyfgenia treatment costs $3.1 million per patient.

Other companies like Agios Pharmaceuticals and therapeutic fulcrumNew experimental treatments for sickle cell anemia are being developed. In particular, some Wall Street analysts said Pfizer's withdrawal of Oxbryta could speed up the clinical trial timeline for those rival drugs.

If Agios' experimental drug, mitapivat, shows a benefit in reducing pain crises in clinical trials, “we anticipate that this will allow for easier regulatory review, especially now that we take into account the increased demands of patients who already they can't access the Pfizer drug,” said analyst Piper Sandler. Christopher Raymond said in a research note last week.

Meanwhile, the financial impact of Oxbryta's withdrawal is “somewhat modest for a company the size of Pfizer,” Guggenheim analysts said in a note last week.

They said Oxbryta sales have been relatively modest for the company, amounting to $328 million last year. But analysts noted that Oxbryta's sales were expected to rise to about $750 million by the end of the decade, citing FactSet consensus estimates.

Pfizer's decision will likely raise questions about the company's ability to grow through the end of the decade, when it faces several drug patent expirations and “other challenges to its current growth engines,” according to Guggenheim. Analysts also said Oxbryta's withdrawal raises questions about what will happen to Pfizer's other sickle cell treatment in development, GBT-601.

That oral drug, which Pfizer also acquired through the Global Blood Therapeutics deal, is considered a successor to Oxbryta.

Feel free to send any tips, suggestions, story ideas, and facts to Annika at [email protected].

Senate Finance Committee Chairman Ron Wyden, D-Ore., and Sen. Mark Warner, D-Va., introduced a bill Thursday that aims to establish “tough” new cybersecurity standards within the security sector. medical attention.

Under the proposed legislation, the Department of Health and Human Services would be responsible for developing and enforcing new standards for health plans, providers, business partners and clearinghouses. The bill is called the “Health Infrastructure Security and Accountability Act,” according to a statement.

Patient data is inherently sensitive and valuable, which can make it an attractive (and often lucrative) target for bad actors. The number of cyberattacks on healthcare has been on an upward trend over the past 14 years, with a record 725 data breaches reported last year, according to The HIPAA Journal.

As of Aug. 31, the magazine said 491 data breaches of more than 500 medical records had been reported in 2024. This includes the massive ransomware attack on the Change Healthcare clearinghouse that shook the healthcare industry this spring.

Change Healthcare is owned by UnitedHealth Groupand offers revenue and payment cycle management tools, as well as other solutions such as e-prescribing software. The company processes more than 15 billion billing transactions annually and 1 in 3 patient records pass through its systems, according to its website.

On February 21, UnitedHealth discovered that hackers compromised part of Change Healthcare's information technology systems. UnitedHealth shut down affected systems, leaving many doctors with no way to fill prescriptions or receive payment for their services. Many providers took thousands of dollars out of their personal savings to keep their practices afloat.

UnitedHealth CEO Andrew Witty testified before the Senate Finance Committee about the attack in May, where he apologized to those affected. At a hearing later that afternoon, Witty estimated that the data of about a third of Americans could have been compromised.

“Megacorporations like UnitedHealth are failing Cybersecurity 101, and American families are suffering as a result,” Wyden said in a statement Thursday announcing the proposed legislation.

Patient data is protected by the Health Insurance Portability and Accountability Act, or HIPAA, and organizations can be fined for violations. As part of the new bill, Wyden and Warner said they would remove the existing cap on HIPAA fines so regulators can force large companies to adhere to new cybersecurity standards.

There is still a long way to go before this legislation can become a reality. It must pass both houses of Congress and be approved by the president before it can become law.

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

Please feel free to send any tips, suggestions, story ideas, or information to Ashley at [email protected].