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A version of this article first appeared in CNBC’s Healthy Returns newsletter, which delivers the latest healthcare news straight to your inbox. To receive future editions, subscribe here.
Hello and Happy Tuesday! Today we unravel a shocking move from. pfizer.
The pharmaceutical giant surprised doctors, patients and investors last week by announcing it would voluntarily withdraw its sickle cell disease drug Oxbrita from the global market.
Here’s why this drug is important: Oxbrita is one of at least six treatments for inherited blood disorders. The drug first received accelerated approval from the U.S. Food and Drug Administration in 2019, but further trials are needed to confirm its benefits for patients.
Oxbrita was one of the highlights of Pfizer’s $5.4 billion acquisition of Global Blood Therapeutics in 2022.
Sickle cell disease causes red blood cells to become misshapen, crescent-shaped, and can get stuck in blood vessels, restricting blood flow and causing so-called pain attacks. It affects about 100,000 people in the United States, many of them Black, according to data from the Centers for Disease Control and Prevention.
The company said Wednesday that its decision to discontinue Oxbrita was based on data showing an increased risk of death and complications in patients treated with the once-daily pill. Pfizer said in a release that the “totality of clinical data” about Oxbrita shows that its overall benefits “no longer outweigh the risks” in the patient populations for which the drug is approved.
As part of its move, Pfizer will also halt all research and access programs related to the treatment.
The FDA on Saturday urged health care professionals to stop prescribing Oxbrita. The agency also said patients and caregivers should contact their health care professional about discontinuing the drug and starting another treatment option.
European regulators also announced Thursday that patients in the trial had a higher incidence of pain attacks after starting treatment with Oxbrita than before taking it. These regulatory agencies recommended that the drug’s marketing authorization be suspended.
It all may sound dry. But Oxbrita’s withdrawal has raised concerns.
Its sudden disappearance from the market has doctors, sickle cell patients and patient advocates scrambling for more information about the decision and guidance on what to do next, STAT reported Friday. . And while taking Oxbrita may put patients at risk, it’s not entirely clear what patients would experience if they abruptly stopped treatment with the drug.
In a statement last week, the National Federation of Sickle Cell Centers urged patients not to suddenly stop taking Oxbrita. The group, which supports health centers treating the disease, urged all patients currently taking Oxbrita to talk to their doctor and come up with a plan to gradually taper off the drug.
Oxbrita’s withdrawal would be a “significant blow” to patients with sickle cell disease, who have been “historically undertreated,” BMO Capital Markets analyst Evan Sagerman said last week. stated in the research note.
Last year, the FDA approved two gene therapies to treat sickle cell disease, a landmark decision that offers hope to patients suffering from the debilitating disease. But health officials have so far struggled to find ways to provide equitable access to expensive treatments.
vertex pharmaceuticalsGene therapy Kasgeby costs $2.2 million per patient; bluebird bioThe treatment, Lifgenia, is listed at $3.1 million per patient.
other companies etc. Agios Pharmaceuticals and Fulcrum TherapeuticThey are developing new experimental treatments for sickle disease. Notably, some Wall Street analysts have said that Pfizer’s withdrawal from Oxbrita could speed up clinical trial timelines for these competing drugs.
If Agios’ experimental drug mitapivat proves effective in reducing pain symptoms in clinical trials, “this could lead to regulatory review, especially given the increased demand from patients who no longer have access to Pfizer’s drug.” “We expect it to become easier,” said analyst Piper Sandler. Christopher Raymond said in a research note last week.
Meanwhile, Guggenheim analysts said in a note last week that the financial impact of Oxbrita’s withdrawal is “rather modest for a company of Pfizer’s size.”
Oxbrita’s sales have been relatively modest for the company, with sales reaching $328 million last year, they said. But analysts noted, citing FactSet consensus forecasts, that Oxbrita’s sales are expected to rise to about $750 million by the end of 2010.
Guggenheim said Pfizer’s decision is likely to cast doubt on the company’s ability to grow through the end of 2010 as it faces the expiration of several drug patents and “other challenges to its current growth engine.” Analysts also said Oxbrita’s withdrawal raises questions about what will happen to Pfizer’s other sickle cell disease drug, GBT-601, in development.
The oral drug, which Pfizer also acquired through a deal with Global Blood Therapeutics, is seen as a successor to Oxbrita.
Please feel free to send tips, suggestions, story ideas, and data to Annika at annikakim.constantino@nbcuni.com.
Latest Healthcare Tech: After Massive Attack, Senators Introduce New Bill to Strengthen Healthcare Cybersecurity
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 bill, the Department of Health and Human Services would be responsible for developing and enforcing new standards for health plans, health care providers, business partners, and clearinghouses. The bill is called the “Health Infrastructure Security and Accountability Act,” according to the release.
Patient data is inherently sensitive and valuable, making it an attractive and often lucrative target for bad actors. According to HIPAA Journal, the number of cyberattacks in the healthcare sector has been on the rise for the past 14 years, with a record 725 data breaches reported last year.
The magazine said that as of August 31, 491 data breaches of more than 500 medical records had been reported in 2024. This includes the massive ransomware attack on information exchange company Change Healthcare that rocked the healthcare industry this spring.
Change Healthcare is owned by united health groupoffers payment and revenue cycle management tools, as well as other solutions such as e-prescription software. The company processes more than 15 billion billing transactions annually, with one in three patient records passing through its systems, according to its website.
On February 21, UnitedHealth discovered that hackers had compromised some of Change Healthcare’s information technology systems. UnitedHealth shut down affected systems, leaving many doctors with no way to fill prescriptions or receive reimbursement. Many providers have taken thousands of dollars out of personal savings to stay in business.
UnitedHealth CEO Andrew Whitty testified about the attack before the Senate Finance Committee in May and apologized to those affected. At a subsequent hearing that afternoon, Whitty estimated that the data of about one-third of Americans may have been compromised.
“Giant companies like UnitedHealth are ignoring Cybersecurity 101, and American families are suffering as a result,” Wyden said in a Thursday release announcing the bill.
Patient data is protected by the Health Insurance Portability and Accountability Act (HIPAA), and organizations that violate it can be subject to fines. As part of the new bill, Wyden and Warner said they would eliminate existing caps on HIPAA fines so that regulators can actually force large companies to comply with new cybersecurity standards.
There is still a long way to go before this bill becomes a reality. To be signed into law, it must pass both houses of Congress and be approved by the president.
The full text of the law can be read here.
Feel free to send tips, suggestions, story ideas, and data to Ashley at ashley.capoot@nbcuni.com.