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Hello and happy Tuesday! Today we reveal a shocking move by Pfizer.
The pharmaceutical giant announced last week that it would voluntarily withdraw its sickle cell anemia drug Oxbryta from global markets, to the surprise of doctors, patients and investors.
That's why the drug is important: Oxbryta is one of at least six drugs used to treat the congenital blood disease. The drug first received accelerated approval from the U.S. Food and Drug Administration in 2019, requiring further studies 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 develop into misshapen crescents that get stuck in blood vessels, which can restrict blood flow and lead to so-called pain crises. According to the Centers for Disease Control and Prevention, it affects about 100,000 people in the United States, including many Black people.
The company said Wednesday that the decision to stop Oxbryta was based on data showing a higher risk of death and complications in patients treated with the once-daily pill. In a press release, Pfizer said the “aggregate of clinical data” on Oxbryta now shows that its overall benefit “no longer outweighs the risk” in the patient population for which the drug is approved.
As part of this move, Pfizer is also halting all trials and access programs related to the treatment.
The FDA on Saturday asked healthcare professionals to stop prescribing Oxbryta. The agency also said patients and caregivers should contact their doctor about stopping the medication and starting another treatment option.
European regulators also said Thursday that patients in trials suffered more pain crises after starting treatment with Oxbryta than before taking it. These regulators recommended that the drug's marketing authorization be suspended.
This may all sound trite. But Oxbryta's withdrawal is cause for concern.
His sudden absence from the market Doctors, sickle cell anemia patients and patient advocates are seeking more information about the decision and guidance on what they should do next, STAT reported Friday. And while taking Oxbryta could pose a risk to patients, 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 Sickle Cell Centers urged patients not to abruptly stop taking Oxbryta. The group, which supports health centers providing treatments for the condition, urged all patients currently taking Oxbryta to make an appointment with their doctor and develop a plan for tapering the drug.
Oxbryta's withdrawal is a “severe blow” to patients with sickle cell anemia “who have been historically underserved,” BMO Capital Markets analyst Evan Seigerman wrote in a research note last week.
The FDA approved two gene therapies to treat sickle cell anemia last year, a groundbreaking decision that offers hope to patients suffering from the debilitating disease. But health authorities have so far struggled to find a way to ensure equal access to the costly treatments.
Vertex PharmaceuticalsCasgevy's gene therapy costs $2.2 million per patient Bluebird OrganicThe treatment offered by Lyfgenia costs $3.1 million per patient.
Other companies such as Agios Pharmaceuticals And Fulcrum Therapeutics develop new experimental treatments for sickle disease. In particular, some Wall Street analysts said Pfizer's withdrawal of Oxbryta could speed up the clinical trial schedule of these rival drugs.
If Agios' experimental drug mitapivat shows benefit in relieving pain crises in clinical trials, “we expect this will allow for easier regulatory review, particularly given greater demand from patients who no longer have access to Pfizer's drug.” “Analyst Piper Sandler Christopher Raymond said in a research note last week.
Meanwhile, the financial impact of the Oxbryta withdrawal “will be modest for a company the size of Pfizer,” Guggenheim analysts said in a note last week.
They said Oxbryta sales were relatively modest for the company, totaling $328 million last year. However, analysts noted that Oxbryta sales are expected to rise to around $750 million by the end of the decade, citing FactSet consensus estimates.
Guggenheim said Pfizer's decision will likely raise questions about the company's ability to grow through the end of the decade, when several drug patents expire and “other challenges exist to their current growth drivers.” The analysts also said Oxbryta's withdrawal raises questions about what will happen to GBT-601, Pfizer's other sickle cell anemia drug in development.
This oral drug, which Pfizer also acquired as part of the Global Blood Therapeutics deal, is considered the successor to Oxbryta.
Feel free to send tips, suggestions, story ideas and data to Annika at annikakim.constantino@nbcuni.com.
Latest developments in healthcare: Senators introduce new bill to strengthen cybersecurity in healthcare after serious attacks
Senate Finance Committee Chairman Ron Wyden, D-Ore., and Sen. Mark Warner, D-Va., introduced a bill Thursday aimed at establishing “strict” new cybersecurity standards in 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 “Healthcare 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 criminals. The number of healthcare cyberattacks has trended upward over the past 14 years, with a record 725 data breaches reported last year, according to The HIPAA Journal.
As of August 31, the magazine said there were 491 reported data breaches in more than 500 health records in 2024. This also includes the massive ransomware attack on the clearinghouse Change Healthcare, which rocked the healthcare industry this spring.
Change Healthcare belongs UnitedHealth Groupand it offers payment and revenue cycle management tools, as well as other solutions such as electronic prescription software. According to its website, the company processes more than 15 billion billing transactions annually and one in three patient records passes through its systems.
On February 21, UnitedHealth discovered that hackers had compromised a portion of Change Healthcare's information technology systems. UnitedHealth has shut down the affected systems, leaving many doctors unable to fill prescriptions or get paid for their services. Many providers have drained thousands of dollars from their personal savings to keep their practices afloat.
UnitedHealth CEO Andrew Witty testified before the Senate Finance Committee about the attack in May and apologized to those affected. In a subsequent hearing that afternoon, Witty estimated that about a third of Americans' data may have been compromised.
“Megacorporations like UnitedHealth are failing Cybersecurity 101, and American families are suffering because of it,” Wyden said Thursday in a news release announcing the proposed legislation.
Patient data is protected by the Health Insurance Portability and Accountability Act (HIPAA) and organizations can be fined for violations. Under the new bill, Wyden and Warner said they would remove the existing cap on HIPAA fines so that regulators can actually force large companies to comply with the new cybersecurity standards.
There is still a long way to go before this law can become a reality. It must pass both chambers of Congress and be approved by the president before it can take effect.
You can read a full copy of the law here.
Feel free to send tips, suggestions, story ideas and data to Ashley at ashley.caroot@nbcuni.com.
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