Pfizer pulls sickle cell disease drug from markets

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Hello and happy Tuesday! Today, we’re unpacking a shocking move from Pfizer. 

The pharmaceutical giant last week announced it would voluntarily withdraw its sickle cell disease therapy, Oxbryta, from worldwide 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 inherited blood disorder. The drug first won accelerated approval from the U.S. Food and Drug Administration in 2019, which requires further trials to confirm its benefits to patients. 

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

Sickle cell disease causes red blood cells to become misshapen half-moons that get stuck inside blood vessels, which can restrict blood flow and cause what are known as pain crises. It impacts roughly 100,000 people in the U.S., many of whom are Black, according to data from the Centers for Disease Control and Prevention.

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

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

The FDA on Saturday urged healthcare professionals to stop prescribing Oxbryta. The agency also said patients and caregivers should contact their healthcare professional about stopping the drug and starting another treatment option. 

European regulators on Thursday also said patients in trials had higher rates of pain crises after they started treatment with Oxbryta than they did before taking it. Those regulators recommended suspending the drug’s marketing authorization. 

That all may sound cut and dry. But Oxbryta’s withdrawal is raising concerns. 

Its sudden absence from the market leaves doctors, sickle cell disease patients and patient advocates scrambling for more information on the decision and guidance on what they should do next, STAT reported on Friday. And while taking Oxbryta could put patients at risk, it is not entirely clear what they may experience if they abruptly stop treatment with the drug. 

In a statement last week, the National Alliance of Sickle Cell 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 for gradually tapering off the medication.  

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

The FDA last year approved two gene therapies to treat sickle cell disease, a landmark decision that gave hope to patients who suffer from the debilitating disease. But health officials have so far struggled to find a way to provide equitable access to the costly treatments. 

Vertex Pharmaceuticals‘ gene therapy Casgevy costs $2.2 million per patient, and Bluebird Bio‘s treatment Lyfgenia lists for $3.1 million per patient. 

Other companies such as Agios Pharmaceuticals and Fulcrum Therapeutics are developing new experimental treatments for sickle disease. Notably, some Wall Street analysts said Pfizer’s withdrawal of Oxbryta could accelerate the timeline for clinical trials on those rival drugs. 

If Agios’ experimental drug, mitapivat, shows a benefit in reducing pain crises in clinical trials, “We anticipate this will enable an easier regulatory review, especially now considering the greater demands from patients who can no longer access Pfizer’s drug,” Piper Sandler analyst Christopher Raymond said in a research note last week. 

Meanwhile, the financial impact of the Oxbryta withdrawal is “somewhat modest for a company of Pfizer’s size,” 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 the analysts noted that Oxbryta sales were expected to increase to around $750 million by the end of the decade, citing FactSet consensus estimates. 

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

That 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 Annika at [email protected].

Senate Finance Committee Chairman Ron Wyden, D-Ore., and Sen. Mark Warner, D-Va., on Thursday introduced a bill that aims to establish “tough” new cybersecurity standards 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 associates and clearinghouses. The bill is called the “Health Infrastructure Security and Accountability Act,” according to a release. 

Patient data is inherently sensitive and valuable, which can make it an enticing – and often lucrative – target for bad actors. The number of health-care cyberattacks has been trending upward over the last 14 years, with a record 725 data breaches reported last year, according to The HIPAA Journal.  

As of August 31, the journal said 491 data breaches of more than 500 health records had been reported in 2024. This includes the massive ransomware attack against the clearinghouse Change Healthcare that shook the health-care industry this spring. 

Change Healthcare is owned by UnitedHealth Group, and it offers payment and revenue cycle management tools as well as other solutions like electronic prescription software. The company processes more than 15 billion billing transactions annually, and 1 in 3 patient records passes 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 the impacted 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 in front of the Senate Finance Committee about the attack in May, where he apologized to the people impacted. In a subsequent hearing that afternoon, Witty estimated that data from around one-third of Americans could have been compromised.

“Megacorporations like UnitedHealth are flunking Cybersecurity 101, and American families are suffering as a result,” Wyden said in a release 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 that regulators can actually compel big companies to adhere to the new cybersecurity standards.

There’s still a long road ahead before this piece of legislation could become a reality. It needs to pass through both chambers of Congress and get approved by the president before it can be signed into law.  

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

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