Pfizer pulls sickle cell disease drug from market | Wilnesh News

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

The pharmaceutical giant announced last week that it would voluntarily Withdrawal of sickle cell disease treatmentOxbryta, from global markets – surprising doctors, patients and investors.

Here’s why this drug is so important: Oxbryta is one of at least six drugs that treat inherited blood disorders. Drug wins first Accelerate approval Approved by the U.S. Food and Drug Administration in 2019, further trials are needed to confirm its benefit to patients.

Oxbryta is one of Pfizer’s core products $5.4 billion acquisition Global Blood Therapeutics Companies 2022.

Sickle cell disease causes red blood cells to turn into misshapen half-moon shapes and become stuck in blood vessels, restricting blood flow and causing What is a so-called pain crisis?. It reportedly affects around 100,000 people in the United States, many of them black data From the Centers for Disease Control and Prevention.

The company said Wednesday that the decision to withdraw Oxbryta was based on data showing patients taking the drug once daily were at higher risk of death and complications. in a releasePfizer said the “full clinical data” for Oxbryta now show that its overall benefits “no longer outweigh the risks” in the patient population for which the drug was approved.

As part of this measure, Pfizer has also halted all research and access programs related to the treatment.

FDA Saturday Healthcare professionals are urged to Stop prescribing Oxbryta. The agency also said patients and caregivers should contact their health care professional about stopping the medication and starting another treatment option.

European regulators also said Thursday that patients in the trial Higher incidence of pain crises After they started treatment with Oxbryta compared to before taking it. These regulatory agencies recommended suspending the drug’s marketing authorization.

This all may sound boring. But Oxbryta’s withdrawal raises concerns.

suddenly disappeared from the market Leaving doctors, sickle cell patients and patient advocates scrambling for more information and guidance on what to do next, STAT report on Friday. While taking Oxbryta may put patients at risk, it’s 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 Oxbryta. The organization, which supports health centers treating the disease, urges all patients currently taking Oxbryta to make an appointment with their doctor and make a plan to gradually taper off the medication.

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

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

Vertex Pharmaceuticals‘Gene therapy Casgevy costs $2.2 million per patient, and blue bird creatureLyfgenia’s treatment costs $3.1 million per patient.

Other companies such as Agios Pharmaceuticals and Pivot therapyWe are developing new experimental treatments for sickle disease. Notably, some Wall Street analysts say Pfizer’s withdrawal of Oxbryta could speed up clinical trial timelines for those rival drugs.

If Agios’ experimental drug mitapivat shows benefit in reducing pain crises in clinical trials, “we expect this will make regulatory review easier, especially given the greater need among patients who can no longer access Pfizer’s medicine,” Piper Sandler analyst Christopher Raymond said in a research note last week.

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

They said Oxbryta’s sales were relatively modest, at $328 million last year. But analysts, citing FactSet consensus estimates, say Oxbryta’s sales are expected to rise to about $750 million by the end of the decade.

Guggenheim said Pfizer’s decision could raise questions about the company’s ability to grow by the end of the decade as it faces multiple drug patent expirations and “other challenges to its current growth momentum.” Analysts also said Oxbryta’s withdrawal raised questions about the impact of GBT-601, another sickle cell disease treatment Pfizer is developing.

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

Please feel free to send Annika any tips, suggestions, story ideas and data: annikakim.constantino@nbcuni.com.

On Thursday, Senate Finance Committee Chairman Ron Wyden (D-Ore.) and Sen. Mark Warner (D-Virginia) introduced a bill The aim is to establish “rigorous” new cybersecurity standards in the healthcare 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 Safety and Accountability Act, according to a press release.

Patient data is inherently sensitive and valuable, making it an attractive and often lucrative target for bad actors. According to statistics, the number of healthcare cyberattacks has been on the rise over the past 14 years, with a record 725 data breaches reported last year HIPAA Magazine.

The magazine said that as of August 31, 491 data breaches of more than 500 health records had been reported in 2024.

The owners of Changing Healthcare are UnitedHealth Groupwhich provides payment and revenue cycle management tools and other solutions such as e-prescribing software. The company processes more than 15 billion billing transactions annually, and one-third of patient records pass through its system, according to its website.

February 21, UnitedHealth Discover Hackers compromised some of Change Healthcare’s information technology systems. UnitedHealth shut down the affected systems, leaving many doctors unable to fill prescriptions or get paid for their services. Many healthcare providers are taking thousands of dollars from personal savings to keep their businesses afloat.

UnitedHealth CEO Andrew Witty testified before the Senate Finance Committee in May about the attack and apologized to those affected. At a subsequent hearing that afternoon, Whitty estimated that about one-third of Americans may have had their data compromised.

“Large companies like UnitedHealth fail to pass cybersecurity 101, and American families suffer because of it,” Wyden said in a press release announcing the proposed legislation Thursday.

Patient data is protected by the Health Insurance Portability and Accountability Act (HIPAA), and organizations can be fined for violations. As part of the new bill, Wyden and Warner said they would lift existing HIPAA fine caps so regulators can actually force large companies to comply with new cybersecurity standards.

There is still a long way to go before this legislation becomes a reality. It would need to pass both houses of Congress and receive presidential approval before it can be signed into law.

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

Please feel free to send any tips, suggestions, story ideas and data to Ashley at ashley.capoot@nbcuni.com.