Pfizer pulls sickle cell disease drug from markets

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

The pharmaceutical giant last week announced it will 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 essential: Oxbryta is one in every of no less than 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 substantiate its advantages to patients. 

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

Sickle cell disease causes red blood cells to change into misshapen half-moons that get stuck inside blood vessels, which may restrict blood flow and cause what are referred to as pain crises. It impacts roughly 100,000 people within the U.S., lots of whom are Black, based on data from the Centers for Disease Control and Prevention.

The corporate on Wednesday said the choice to withdraw Oxbryta was based on data showing a better 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 profit “not outweighs the chance” within the patient population for which the drug is approved. 

As a part of that move, Pfizer can also be 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 skilled about stopping the drug and starting one other treatment option. 

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

That each one 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 choice and guidance on what they need to do next, STAT reported on Friday. And while taking Oxbryta could put patients in danger, it will not be entirely clear what they could experience in the event that they abruptly stop treatment with the drug. 

In a statement last week, the National Alliance of Sickle Cell Centers urged patients to not 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 regularly really fizzling out the medication.  

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

The FDA last yr approved two gene therapies to treat sickle cell disease, a landmark decision that gave hope to patients that suffer from the debilitating disease. But health officials have to this point struggled to search out a solution 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 corporations equivalent to Agios Pharmaceuticals and Fulcrum Therapeutics are developing latest experimental treatments for sickle disease. Notably, some Wall Street analysts said Pfizer’s withdrawal of Oxbryta could speed up the timeline for clinical trials on those rival drugs. 

If Agios’ experimental drug, mitapivat, shows a profit in reducing pain crises in clinical trials, “We anticipate this may enable a neater regulatory review, especially now considering the greater demands from patients who can not 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 an organization of Pfizer’s size,” Guggenheim analysts said in a note last week. 

They said Oxbryta sales have been relatively modest for the corporate, amounting to $328 million last yr. However the analysts noted that Oxbryta sales were expected to extend to around $750 million by the tip of the last decade, citing FactSet consensus estimates. 

Pfizer’s decision will likely raise questions around the corporate’s ability to grow through the tip of the last decade when it faces several drug patent expirations and “other challenges to their current growth drivers,” based on Guggenheim. The analysts also said the Oxbryta withdrawal raises questions on what’s going to occur 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.

Be happy to send any suggestions, suggestions, story ideas and data to Annika at annikakim.constantino@nbcuni.com.

Senate Finance Committee Chairman Ron Wyden, D-Ore., and Sen. Mark Warner, D-Va., on Thursday introduced a bill that goals to determine “tough” latest cybersecurity standards inside the health-care sector. 

Under the proposed laws, the Department of Health and Human Services could be liable for developing and enforcing latest standards for health plans, providers, business associates and clearinghouses. The bill known as the “Health Infrastructure Security and Accountability Act,” based on a release. 

Patient data is inherently sensitive and helpful, which may make it an attractive – and infrequently lucrative – goal for bad actors. The variety of health-care cyberattacks has been trending upward over the past 14 years, with a record 725 data breaches reported last yr, based on The HIPAA Journal.  

As of August 31, the journal said 491 data breaches of greater than 500 health records had been reported in 2024. This includes the huge 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 in addition to other solutions like electronic prescription software. The corporate processes greater than 15 billion billing transactions annually, and 1 in 3 patient records passes through its systems, based on its website.

On February 21, UnitedHealth discovered that hackers compromised a part of Change Healthcare’s information technology systems. UnitedHealth shut down the impacted systems, leaving many doctors with no solution to fill prescriptions or receives a commission for his or her services. Many providers took 1000’s of dollars out of their personal savings to maintain their practices afloat. 

UnitedHealth CEO Andrew Witty testified in front of the Senate Finance Committee concerning 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 might have been compromised.

“Megacorporations like UnitedHealth are flunking Cybersecurity 101, and American families are suffering in consequence,” Wyden said in a release Thursday announcing the proposed laws. 

Patient data is protected by the Health Insurance Portability and Accountability Act, or HIPAA, and organizations might be fined for violations. As a part of the brand new bill, Wyden and Warner said they’d remove the present cap on HIPAA fines in order that regulators can actually compel big corporations to stick to the brand new cybersecurity standards.

There’s still an extended road ahead before this piece of laws could change into a reality. It must go through each chambers of Congress and get approved by the president before it will probably be signed into law.  

You’ll be able to read a full copy of the legislative text here.  

Be happy to send any suggestions, suggestions, story ideas and data to Ashley at ashley.capoot@nbcuni.com.