FDA and Safety - Pharma Rep Focus

'It was a good lesson': FDA reconsiders CAR-T boxed warning on secondary cancers, Peter Marks says

Fierce Pharma

NOVEMBER 1, 2024

An FDA announcement last year of an investigation into the risk of patients developing secondary cancers following the treatment of CAR-T cell therapies sent shock waves across the industry. | said in an interview with Fierce Pharma.

FDA

FDA Safety Pharma Patients

‘Serial Killing’ Cell Therapy From Autolus Lands FDA Approval in Blood Cancer

MedCity News

NOVEMBER 10, 2024

Autolus Therapeutics’ Aucatzyl is now FDA approved for treating advanced cases of B-cell precursor acute lymphoblastic leukemia. While it goes after the same target as Gilead Sciences’ Tecartus, Autolus engineered its CAR T-therapy with properties that could improve safety, efficacy, and durability.

FDA

FDA Engineering Safety Biopharma

FDA Pushes Out Decision for Cytokinetics Heart Drug to Review Proposed Safety Plan

MedCity News

MAY 2, 2025

The FDA asked Cytokinetics for the Risk Evaluation and Mitigation Strategy after it had already accepted the application for aficamten, a potential competitor to Bristol Myers Squibbs heart drug Camzyos. Analysts say this development could signal a change in FDA thinking about risk management for the broader drug class.

Safety

Safety FDA Management Biopharma

With FDA Nod in Lung Cancer, Dizal Now Aims to Best a J&J Drug and Succeed Where Takeda Fell Short

MedCity News

JULY 3, 2025

Dizal Pharmaceutical’s Zegfrovy is now FDA approved for treating non-small cell lung cancer driven by a particular rare genetic signature. Johnson & Johnson drug Rybrevant already addresses this genetic signature as an earlier line of therapy, but Dizal executives contend their drug offers the potential for better safety and efficacy.

FDA

FDA Safety Pharmaceutical Biopharma

Pharma Must Speak Up for Patient Safety

World of DTC Marketing

JUNE 21, 2025

Yet, astonishingly, the very institutions tasked with protecting and managing our healthcare can sometimes become a threat to patient safety. Consider the frustrating reality of slow-moving, poorly informed decisions made by agencies such as the FDA, CMS, or CDC. It’s a basic principle: the healthcare system exists to serve patients.

Safety

Safety Pharma Patients Government

FDA Just Made Some Key Regulatory Changes to Make Cancer Cell Therapies More Accessible

MedCity News

JUNE 27, 2025

The FDA has eliminated a safety monitoring requirement and eased certain restrictions that had been in place since CAR T-therapies first reached the market in 2017. The post FDA Just Made Some Key Regulatory Changes to Make Cancer Cell Therapies More Accessible appeared first on MedCity News.

FDA

FDA Safety Marketing Patients

Why Biosimilars Struggle Despite FDA Approval

World of DTC Marketing

JULY 6, 2025

Yet, despite FDA approval and being clinically equivalent to their branded counterparts, biosimilars have not lived up to the promise of becoming blockbuster disrupters. Facebook Twitter LinkedIn Tumblr Bluesky Email Reddit WhatsApp The post Why Biosimilars Struggle Despite FDA Approval appeared first on World of DTC Marketing.

FDA

FDA Physicians Manufacturing Insurance

From Public to Private: Do Laid-Off FDA and CDC Employees Have the Skills Pharma and Biotech Need?

World of DTC Marketing

JUNE 6, 2025

With reports of budget constraints and layoffs hitting federal agencies like the FDA and CDC, many talented public health professionals are suddenly finding themselves out of work. What Laid-Off Government Employees Bring to the Table 1. Mission-Driven Perspective Former federal employees are typically motivated by improving health outcomes.

Pharma

Pharma FDA Ethics Government

Preclinical Safety Signal Prompts FDA Clinical Hold on Neumora Schizophrenia Drug

MedCity News

APRIL 15, 2024

The FDA clinical hold keeps Neumora Therapeutics from catching up to the field of biopharmaceutical companies pursuing the same target for schizophrenia. The post Preclinical Safety Signal Prompts FDA Clinical Hold on Neumora Schizophrenia Drug appeared first on MedCity News.

Safety

Safety FDA Leads Biopharma

AstraZeneca to go on defense for Andexxa as FDA questions bleeding reversal agent's safety ahead of adcomm

Fierce Pharma

NOVEMBER 20, 2024

As a council of outside experts prepares to meet Thursday to discuss AstraZeneca’s Andexxa, the FDA has flagged “major safety findings” linked to the British drugmaker’s bleeding reversal agent.

Safety

Safety FDA

Intercept's latest NASH bid in jeopardy after FDA questions drug's efficacy, safety

Fierce Pharma

MAY 17, 2023

Intercept's latest NASH bid in jeopardy after FDA questions drug's efficacy, safety aliu Wed, 05/17/2023 - 12:29

Safety

Safety FDA

Novartis Tries Again in Huntington’s, Putting Up $1B to Partner on a PTC Therapeutics Drug

MedCity News

DECEMBER 2, 2024

Novartis is securing global rights to a PTC Therapeutics Huntington’s disease drug candidate that has encouraging Phase 2 data that will be discussed with the FDA to plan a pivotal study. Last year, Novartis discontinued its Huntington’s program after safety issues emerged in Phase 2 testing.

Safety

Safety FDA Biopharma Pharma

FDA Calls for New Safety Warning for the Class of CAR T Cancer Therapies

MedCity News

JANUARY 23, 2024

Two months after announcing an inquiry into reports of new cancers in patients treated with CAR T-cell therapies, the FDA is directing makers of these therapies to add new safety warnings to product labels describing this risk. Companies have 30 calendar days to comply.

Safety

Safety FDA Patients Biopharma

Eisai Alzheimer’s Drug Wins Full FDA Approval, But With Sterner Safety Alert

MedCity News

JULY 6, 2023

Six months after Esai Alzheimer’s disease drug Leqembi received accelerated FDA approval, the agency has awarded the drug full approval. While the agency’s regulatory decision sets precedent for other Alzheimer’s drugs, it also flags serious safety risks.

Safety

Safety FDA Biopharma Pharma

Gilead's Tecartus gets revised safety demand amid FDA's push for CAR-T boxed warnings

Fierce Pharma

JANUARY 24, 2024

. | The FDA’s letter demanding a labeling change for Gilead Sciences’ Tecartus temporarily went missing on the agency’s website Tuesday. Turns out, the FDA didn’t drop the CD19 CAR-T from a classwide safety alert list. Instead, it's adjusted the wording of a proposed boxed warning.

Safety

Safety FDA

Morale at the FDA Has Hit Rock Bottom: What It Means for Pharma

World of DTC Marketing

JUNE 20, 2025

Low morale within the Food and Drug Administration (FDA) is more than just an internal issue—it sends shockwaves through the pharmaceutical industry, affecting regulatory timelines, decision-making, innovation, and public trust. Low morale at the FDA can disrupt these plans significantly: 1. What Does This Mean for Pharma?

FDA

FDA Pharma Food and Drug Administration Pharmaceutical

Enabling Clinical Trial Innovation Through AI Regulation at FDA

MedCity News

JULY 29, 2024

As the FDA stakes its claim as the primary regulator of AI in healthcare, it must navigate the delicate balance between fostering innovation and ensuring safety and responsibility. The post Enabling Clinical Trial Innovation Through AI Regulation at FDA appeared first on MedCity News.

FDA

FDA Safety Healthcare Healthcare

FDA wants classwide boxed warning on all commercial CAR-T therapies amid secondary cancer safety probe

Fierce Pharma

JANUARY 22, 2024

As many have feared, the FDA’s investigation into secondary T-cell cancers following treatment with existing CAR-T therapies is poised to lead to a classwide black box warning. |

FDA

FDA Safety Leads

Inside the FDA: Staff Morale and Public Health Concerns

World of DTC Marketing

JUNE 13, 2025

What are FDA staffers thinking? And while the outside world may only see the final decisions and policy announcements, it’s the rank-and-file FDA staffers who live with the day-to-day fallout. Whistleblowers in the FDA are rare, not because there aren’t problems, but because the consequences of speaking out can be career-ending.

FDA

FDA Safety Doctors Food

FDA Approval of Bristol Myers Drug Makes It the First Novel Schizophrenia Med in Decades

MedCity News

SEPTEMBER 27, 2024

Bristol Myers Squibb’s Cobenfy treats schizophrenia by going after a different target than currently available antipsychotic drugs, which is intended to offer better efficacy and safety. The post FDA Approval of Bristol Myers Drug Makes It the First Novel Schizophrenia Med in Decades appeared first on MedCity News.

FDA

FDA Safety Pharma Biopharma

As FDA probes CAR-T safety, expert meeting for Bristol's Abecma could serve as key guidepost

Fierce Pharma

NOVEMBER 29, 2023

The revelation of an FDA investigation< | As industry watchers deliberate the implications from the FDA's investigation into secondary cancer risks from CAR-T medicines, an upcoming expert meeting could offer precious clarity from drug regulators.

FDA

FDA Safety Medicine

Akebia Recovers From Rejection to Win FDA Approval for Anemia Drug

MedCity News

MARCH 31, 2024

Approval of Akebia Therapeutics’ Vafseo is based on additional post-marketing safety data from Japan, where the drug has been used since 2020. The FDA rejected the drug two years ago due to concerns about cardiovascular safety.

FDA

FDA Safety Marketing Biopharma

Clarity will help stem the problem of ‘winners and losers’ in medical device safety and security

MedCity News

JUNE 22, 2022

The Clarifying Remanufacturing to Protect Patient Safety Act was introduced by Reps. Scott Peters (D-CA), Kim Schrier (D-WA), and John Joyce (R- PA) to clarify the meaning of medical device remanufacturing and empower the FDA to educate company technicians so they know if they’ve crossed the line into a regulated activity. .

Safety

Safety Medical Education FDA

Trial Data Shows ‘IUD for Men’ Is Effective for 2 Years

MedCity News

APRIL 24, 2025

Contraline released promising new trial data showing that its male contraceptive gel has demonstrated safety and efficacy in the first two trial participants to reach the two-year mark. The company is aiming to earn the products FDA approval by the end of 2028, according to CEO Kevin Eisenfrats.

Safety

Safety FDA

J&J, Legend’s Carvykti slapped with FDA black-box warning over secondary cancer risk

Fierce Pharma

DECEMBER 22, 2023

As the FDA investigates a safety signal for approved CAR-T therapies, the agency has upgraded a warning about secondary cancers resulting from use of Johnson & Johnson and Legend Biotech’s Carv | As the FDA investigates a safety signal for approved CAR-T therapies, the agency has elevated a warning about secondary cancers resulting from use of (..)

FDA

FDA Safety

As FDA puts CAR-T safety under microscope, researchers dig into adverse events record of BCMA therapies

Fierce Pharma

DECEMBER 9, 2023

A new study examined side effect reports of BMCA immunotherapies from the FDA Adverse Event Reporting System, hoping to help inform doctors in their treatment decisions. BCMA-targeted therapies are transforming care for multiple myeloma patients. But these immunotherapies also come with various potentially dangerous side effects. |

Side effects

Side effects FDA Safety Doctors

FDA Pauses Verve’s Trial Plan for Gene-Editing Therapy for High Cholesterol

MedCity News

NOVEMBER 7, 2022

The FDA placed a clinical hold on Verve Therapeutics’ application to begin human testing of its gene-editing therapy for an inherited form of high cholesterol. No details about the FDA’s questions or concerns were disclosed; so far, no serious safety problems have been reported in the U.K.

FDA

FDA Safety Biopharma Pharma

Cell therapy bigwigs endorse CAR-T in Nature Medicine article amid FDA safety probe

Fierce Pharma

JANUARY 6, 2024

A group of cell therapy experts, including CAR-T pioneers Drs. Bruce Levine and Carl June, wrote in support of continued use of CAR-T therapy.

Safety

Safety Medicine FDA

FDA issues first recommendations on AI for drug development

European Pharmaceutical Review

JANUARY 7, 2025

The US Food and Drug Administration (FDA) has for the first time, issued draft guidance on using artificial intelligence (AI) intended to support a regulatory decision about a drug or biological product. The guidelines address the safety, effectiveness or quality of these medicines.

Food and Drug Administration

Food and Drug Administration FDA Food Medical

Amid high-profile CAR-T safety probe, FDA’s Peter Marks offers first glimpse at data under review

Fierce Pharma

JANUARY 25, 2024

But questions remained unanswered.

Safety

Safety FDA Medicine Patients

Intercept's Ocaliva approval in jeopardy after FDA flags concerns in advisory committee briefing doc

Fierce Pharma

SEPTEMBER 11, 2024

Ahead of an advisory committee meeting slated for Friday, the FDA unleashed damning briefing documents calling into question the effectiveness and safety of Intercept’s rare liver disease drug Ocaliva. For Intercept Pharmaceuticals’ beleaguered liver disease drug Ocaliva, the hits just keep coming.

FDA

FDA Safety Pharmaceutical Marketing

Liver injury leads FDA to pause Sanofi’s tests of MS drug acquired in $3.7B deal

MedCity News

JUNE 30, 2022

The FDA placed a partial clinical hold on late-stage tests of Sanofi multiple sclerosis drug tolebrutinib after some patients developed drug-induced liver injury. tests are paused, Sanofi said clinical testing in other countries is continuing with additional safety monitoring.

FDA

FDA Leads Safety Patients

Regeneron's high-dose Eylea hit with surprise FDA rejection over manufacturing hiccups

Fierce Pharma

JUNE 27, 2023

Despite Regeneron’s impressive high-dose Eylea data, its high-stakes launch is delayed thanks to an unexpected FDA rejection. | In a surprise decision, the FDA issued powerhouse Eylea's high-dose version a complete response letter. But the company said it has nothing to do with the drug's efficacy or safety.

FDA

FDA Manufacturing Safety

FDA approves novel therapy for chronic skin condition

European Pharmaceutical Review

APRIL 21, 2025

For the first time in over ten years, the US Food and Drug Administration (FDA) has approved a new targeted treatment for chronic spontaneous urticaria. Why is the FDA’s decision to approve Dupixent as a new treatment for chronic spontaneous urticaria significant?

FDA

FDA Food and Drug Administration Food Safety

FDA approves first-of-a-kind oral therapy for rare disease

European Pharmaceutical Review

JULY 7, 2025

The US Food and Drug Administration (FDA) has approved the first oral on-demand therapy for acute attacks of hereditary angioedema (HAE) in individuals aged 12 years and older. The post FDA approves first-of-a-kind oral therapy for rare disease appeared first on European Pharmaceutical Review.

Food and Drug Administration

Food and Drug Administration FDA Pharmaceutical Physicians

Eisai Alzheimer’s Drug Data Show Slowing of Decline But Safety Questions Linger

MedCity News

NOVEMBER 30, 2022

While an accelerated approval decision is expected in early 2023, the latest trial data are key because they represent the confirmatory study that could support an application for full FDA approval.

Safety

Safety FDA Biopharma Pharma

With new gene therapy data in hand, Sarepta talks with FDA about approval pathways

MedCity News

JULY 6, 2022

Sarepta Therapeutics has more clinical data showing the safety and efficacy of its experimental gene therapy for Duchenne muscular dystrophy. A pivotal Phase 3 test is already underway and could post data next year but the company is also talking with regulators about the possibility of a submission under the accelerated approval pathway.

Safety

Safety FDA Biopharma Pharma

B. Braun, Baxter launch separate recalls over particulate concerns and excess endotoxin levels, respectively

Fierce Pharma

AUGUST 15, 2024

So far in August, the FDA has announced two separate drug recalls on its website: one related to particulate concerns and the other thanks to high endotoxin levels. | So far in August, the FDA has announced two separate drug recalls on its website: one related to particulate concerns and the other thanks to high endotoxin levels.

Side effects

Side effects Safety FDA

After initial failure, Intercept Pharma feels new data can get NASH drug approval

MedCity News

JULY 7, 2022

Two years after the stinging FDA rejection of its drug for the fatty liver disease NASH, Intercept Pharmaceuticals has more safety and efficacy data from a pivotal study that could support resubmission of a new drug application. The biotech said it will meet with the FDA later this month.

Safety

Safety Pharma FDA Pharmaceutical

CMS Will Cover Approved Alzheimer’s Drugs, But It Still Wants More Evidence

MedCity News

JUNE 1, 2023

Medicare will cover Alzheimer’s disease drugs granted traditional FDA approval, CMS announced. But the decision keeps the spirit of an earlier coverage determination, which called for the gathering of more evidence about the safety and efficacy of these therapies.

Safety

Safety FDA Biopharma Pharma

RSV vaccines may raise risk of Guillain-Barré syndrome, FDA preliminary analysis finds

Fierce Pharma

MARCH 4, 2024

. | After a preliminary analysis found a potentially higher risk of Guillain-Barré syndrome following RSV vaccination in older adults, GSK and Pfizer said they will conduct further safety studies.

Safety

Safety FDA

Astellas touts 2-year trial of geographic atrophy drug Izervay but shares few details

Fierce Pharma

SEPTEMBER 19, 2023

Gaining FDA approval last month for its geographic atro | Astellas reported results of a phase 3 trial that assessed Izervay’s effectiveness and safety over a 24-month period.

Safety

Safety FDA

Astellas Drug Acquired in $5.9B Deal Wins FDA Approval in Vision-Loss Disorder

MedCity News

AUGUST 7, 2023

The regulatory decision comes as safety concerns emerge around the first therapy for the disease, an Apellis Pharmaceuticals product. Izervay, a drug developed by Astellas Pharma subsidiary Iveric Bio, is now the second approved therapy for the degenerative vision-loss disorder geographic atrophy.

Pharmaceutical products

Pharmaceutical products Safety FDA Pharma

Drug companies know how to play the FDA drug approval process

World of DTC Marketing

JULY 13, 2021

SUMMARY: The FDA has many puzzled as to why they approve some drugs with questionable data and ask for more data on other drugs. The FDA approves drugs on the potential to save a life, the cost of the drugs is never considered in the process. Drug companies know how to play the system. Can we make sense of this?

FDA

FDA Medicine Healthcare Healthcare

'It was a good lesson': FDA reconsiders CAR-T boxed warning on secondary cancers, Peter Marks says

‘Serial Killing’ Cell Therapy From Autolus Lands FDA Approval in Blood Cancer

Trending Sources

FDA Pushes Out Decision for Cytokinetics Heart Drug to Review Proposed Safety Plan

With FDA Nod in Lung Cancer, Dizal Now Aims to Best a J&J Drug and Succeed Where Takeda Fell Short

Pharma Must Speak Up for Patient Safety

FDA Just Made Some Key Regulatory Changes to Make Cancer Cell Therapies More Accessible

Why Biosimilars Struggle Despite FDA Approval

From Public to Private: Do Laid-Off FDA and CDC Employees Have the Skills Pharma and Biotech Need?

Preclinical Safety Signal Prompts FDA Clinical Hold on Neumora Schizophrenia Drug

AstraZeneca to go on defense for Andexxa as FDA questions bleeding reversal agent's safety ahead of adcomm

Intercept's latest NASH bid in jeopardy after FDA questions drug's efficacy, safety

Novartis Tries Again in Huntington’s, Putting Up $1B to Partner on a PTC Therapeutics Drug

FDA Calls for New Safety Warning for the Class of CAR T Cancer Therapies

Eisai Alzheimer’s Drug Wins Full FDA Approval, But With Sterner Safety Alert

Gilead's Tecartus gets revised safety demand amid FDA's push for CAR-T boxed warnings

Morale at the FDA Has Hit Rock Bottom: What It Means for Pharma

Enabling Clinical Trial Innovation Through AI Regulation at FDA

FDA wants classwide boxed warning on all commercial CAR-T therapies amid secondary cancer safety probe

Inside the FDA: Staff Morale and Public Health Concerns

FDA Approval of Bristol Myers Drug Makes It the First Novel Schizophrenia Med in Decades

As FDA probes CAR-T safety, expert meeting for Bristol's Abecma could serve as key guidepost

Akebia Recovers From Rejection to Win FDA Approval for Anemia Drug

Clarity will help stem the problem of ‘winners and losers’ in medical device safety and security

Trial Data Shows ‘IUD for Men’ Is Effective for 2 Years

J&J, Legend’s Carvykti slapped with FDA black-box warning over secondary cancer risk

As FDA puts CAR-T safety under microscope, researchers dig into adverse events record of BCMA therapies

FDA Pauses Verve’s Trial Plan for Gene-Editing Therapy for High Cholesterol

Cell therapy bigwigs endorse CAR-T in Nature Medicine article amid FDA safety probe

FDA issues first recommendations on AI for drug development

Amid high-profile CAR-T safety probe, FDA’s Peter Marks offers first glimpse at data under review

Intercept's Ocaliva approval in jeopardy after FDA flags concerns in advisory committee briefing doc

Liver injury leads FDA to pause Sanofi’s tests of MS drug acquired in $3.7B deal

Regeneron's high-dose Eylea hit with surprise FDA rejection over manufacturing hiccups

FDA approves novel therapy for chronic skin condition

FDA approves first-of-a-kind oral therapy for rare disease

Eisai Alzheimer’s Drug Data Show Slowing of Decline But Safety Questions Linger

With new gene therapy data in hand, Sarepta talks with FDA about approval pathways

B. Braun, Baxter launch separate recalls over particulate concerns and excess endotoxin levels, respectively

After initial failure, Intercept Pharma feels new data can get NASH drug approval

CMS Will Cover Approved Alzheimer’s Drugs, But It Still Wants More Evidence

RSV vaccines may raise risk of Guillain-Barré syndrome, FDA preliminary analysis finds

Astellas touts 2-year trial of geographic atrophy drug Izervay but shares few details

Astellas Drug Acquired in $5.9B Deal Wins FDA Approval in Vision-Loss Disorder

Drug companies know how to play the FDA drug approval process

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