Pfizer withdraws its sickle cell disease drug from the market

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Hello and happy Tuesday! Today, we reveal a shocking move from… Pfizer.

The pharmaceutical giant announced last week that it would voluntarily withdraw its sickle cell disease treatment, Oxpreta, 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 the hereditary blood disorder. The drug first received accelerated approval from the US Food and Drug Administration in 2019, which requires more trials to confirm its benefits for patients.

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

Sickle cell disease causes red blood cells to become deformed into half-moons that get stuck inside blood vessels, which can restrict blood flow and cause what are known as pain crises. It affects nearly 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 on Wednesday that the decision to withdraw Oxbryta was based on data showing a higher risk of death and complications in patients treated with the once-daily pill. Pfizer said in a statement that the “overall clinical data” for Oxpreta now indicate that its overall benefit “no longer outweighs the risks” in the patient population for which the drug is approved.

As part of the move, Pfizer is also halting all studies and access programs related to the treatment.

The US Food and Drug Administration on Saturday urged healthcare professionals to stop prescribing Oxpreta. The agency also said patients and caregivers should contact their health care professional about stopping the drug and starting another treatment option.

European regulators also said Thursday that patients in the trials had higher rates of pain crises after starting treatment with Oxpreta than they did before taking it. These regulators recommended suspending the drug's marketing authorization.

This may all seem cut and dry. But Oxpreta's withdrawal raises concerns.

Its sudden absence from the market leaves doctors, sickle cell patients and patient advocates scrambling for more information about the decision and guidance on what to do next, STAT reported Friday. Although taking Oxbryta can put patients at risk, it is not entirely clear what they might experience if they suddenly stop treatment with the drug.

In a statement last week, the National Alliance of Sickle Cell Centers urged patients not to suddenly stop taking Oxpreta. The group, which supports health centers offering treatments for the disorder, urged all patients currently taking Oxpreta to make an appointment with their doctor and develop a plan to taper off the drug.

Oxbryta's withdrawal would be a “huge blow” to patients with sickle cell disease who have been “historically underserved,” BMO Capital Markets analyst Evan Segerman wrote in a research note last week.

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

Vertex PharmaceuticalsCasgevy's gene therapy costs $2.2 million per patient Bluebird BioLyfgenia's Lyfgenia treatment lists a cost of $3.1 million per patient.

Other companies like Agios Pharmaceuticals and Therapeutic fulcrumResearchers are developing new experimental treatments for sickle cell disease. It is worth noting that some Wall Street analysts said that Pfizer's withdrawal from Oxpreta may lead to an acceleration of the timeline for clinical trials on those competing drugs.

If Agios' experimental drug, Metapivat, shows benefit in reducing pain crises in clinical trials, “we expect this will enable easier regulatory review, especially now given the greater demands from patients who no longer have access to Pfizer's drug,” says analyst Piper. Sandler. Christopher Raymond said in a research note last week.

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

Oxbryta sales were relatively modest for the company, at $328 million last year, they said. But analysts noted that Oxbryta sales are expected to rise to about $750 million by the end of the decade, citing consensus FactSet 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 the Oxbryta withdrawal raises questions about what will happen to Pfizer's other sickle cell treatment, GBT-601.

The oral drug, which Pfizer also acquired through the Global Blood Therapeutics deal, is seen as a successor to Oxbryta.

Feel free to send any tips, suggestions, story ideas and data to Annikakim at annikakim.constantino@nbcuni.com.

Senate Finance Committee Chairman Ron Wyden, D-Ore., and Sen. Mark Warner, D-Virginia, on Thursday introduced a bill aimed at setting “stringent” new standards for cybersecurity within the health care sector.

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 press release.

Patient data is inherently sensitive and valuable, which can make it a tempting – and often lucrative – target for bad actors. The number of cyberattacks in healthcare has risen over the past 14 years, with 725 data breaches reported last year, according to HIPAA.

As of August 31, the magazine said there have been 491 data breaches of more than 500 health records reported in 2024. This includes a massive ransomware attack against the Change Healthcare clearinghouse that rocked the healthcare industry this spring.

Change Healthcare is owned by UnitedHealth GroupIt offers payment cycle and revenue management tools as well as other solutions such as electronic prescription 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 had compromised part of Change Healthcare's IT systems. UnitedHealth shut down the affected systems, leaving many doctors without a way to fill prescriptions or get paid 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 people affected. At a hearing later that afternoon, Whitty estimated that data from about a third of Americans may have been compromised.

“Giant companies like UnitedHealth are failing to enforce Cybersecurity 101, and American families are suffering as a result,” Wyden said in a statement issued 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 current cap on HIPAA fines so regulators can force larger companies to adhere to new cybersecurity standards.

There is still a long way to go before this legislation becomes a reality. The law needs to pass through both houses of Congress and receive the president's approval before it can be signed into law.

You can read the full version of the legislative text here.

Feel free to send any tips, suggestions, story ideas, and pitches to Ashley at ashley.capoot@nbcuni.com.