Pharmaceutical Technology

MAY 2, 2023

There are more than 20 marketed ICIs with approvals across a very wide spectrum of solid tumour indications. While initially only approved for metastatic disease, ICIs have now moved into earlier disease settings, reducing the risk of disease progression and relapse. Cost-related unmet needs also scored highly.

Genetic Engineering 130

Genetic Engineering Antibody Clinical Trials Clinical Trials 130

World of DTC Marketing

SEPTEMBER 7, 2022

In pharma, growth depends on new products with hefty price tags when over 80% of voters want lower costs for their prescription drugs. According to JAMA , “in a cross-sectional study, between May 1, 2016, and May 31, 2021, there were 207 cancer drug approvals in oncology and malignant hematology. ” The U.S.

Pharma Companies 210

Pharma Companies Marketing Drugs Production 210

XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). The FDA approval came after several delays and a narrow advisory committee vote of eight to six in favor of the gene therapy’s risk-benefit profile.

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Gene Therapy Gene Protein FDA Approval 98

XTalks

AUGUST 31, 2022

In this episode, Ayesha discussed the FDA approval of Axsome Therapeutics’ rapid-acting oral treatment Auvelity for the treatment of major depressive disorder (MDD). The approval makes Auvelity the first and only rapid-acting oral medication for depression, and the first and only oral NMDA receptor antagonist approved for MDD.

Gene Therapy 52

Gene Therapy FDA Approval Life Science Gene 52

XTalks

APRIL 18, 2024

Conversely, a negative result suggests a low probability of amyloid pathology, guiding healthcare providers in clinical decision-making and potentially ruling out Alzheimer’s disease in symptomatic patients. There are currently no FDA-authorized blood tests for detecting Alzheimer’s disease.

Clinical Trials 52

Clinical Trials Clinical Trials In-Vitro Protein 52

Rethinking Clinical Trials

NOVEMBER 1, 2022

Duke Clinical Research Institute. On March 21, 2020, in response to the COVID-19 pandemic, PCORI contacted leadership at Duke Clinical Research Institute and PCORnet and a decision was made to focus on the space of healthcare workers. The HERO Program was fully approved and began recruiting participants on April 22, 2020.

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Trials Clinical Research Vaccination Vaccine 130

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

Rethinking Clinical Trials

NOVEMBER 21, 2023

Three healthcare delivery systems participated in the study: UC Health Clinics, Denver Health, and VA Eastern Colorado HCS Clinics. Patients could text back with clinical questions or questions about the study. responded with a question for a clinical pharmacist. About 50% of participants responded with a question, and 9.2%

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Clinical Trials Clinical Trials Trials Development 141

Pharmaceutical Technology

FEBRUARY 10, 2023

On November 22, 2022, the FDA approved CSL Behring’s Hemgenix (etranacogene dezaparvovec), the first gene therapy treatment for hemophilia B, with a staggering manufacturer price of $3.5 This is not the first treatment to come with a high price tag.

Gene Therapy 263

Gene Therapy Marketing Gene Drugs 263

XTalks

JULY 12, 2021

The US Food and Drug Administration’s (FDA) recent approval of Biogen’s Alzheimer’s drug Aduhelm (aducanumab) was met with a lot of backlash, prompting the health agency to narrow its recommendation of the drug for mild forms of the disease. All of this for a drug that has shown questionable clinical benefits, critics say.

Drugs 98

Drugs Clinical Trials Clinical Trials Trials 98

Rethinking Clinical Trials

JULY 27, 2022

Vice Dean for Clinical Research. The key clinical questions of the ACTIV-6 study are: how to help someone feel better faster with newly diagnosed mild-moderate COVID-19 and how to prevent hospitalizations or death in someone with newly diagnosed mild-moderate COVID-19? ? ? ? ? ? ? ? ? ? ? ? ?. Speaker. Susanna Naggie, MD, MHS.

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Trials Pharmacy Medicine Clinical Trials 169

Pharmaceutical Technology

JANUARY 17, 2023

Despite its high price tag, bimagrumab’s unique features set it apart from currently marketed therapies. Mounjaro is anticipated to launch in the US later this year or by early 2024. Bimagrumab is an activin receptor type 2B (ACVR2B or EC 2.7.11.30) antagonist that offers a unique mechanism of action within the obesity market.

Development 130

Development Compliance Trials Marketing 130

Rethinking Clinical Trials

JUNE 29, 2022

Real-world evidence (RWE) is clinical evidence derived from analysis of RWD regardless of study design. With the 21st Century Cures Act of 2016, the FDA established a program to evaluate the potential use of real-world evidence to support new indications for drugs and satisfy post-approval study requirements. pctGR, @Collaboratory1.

Big Data 130

Big Data Clinical Trials Clinical Trials Drugs 130

Pharmaceutical Technology

APRIL 13, 2023

Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER). If approved, exa-cel would be the first FDA-approved gene therapy based on CRISPR editing.

Gene Therapy 147

Gene Therapy Gene FDA Approval Licensing 147

pharmaphorum

JANUARY 9, 2023

First introduced in 2020, the global Managed Access Programme (gMAP) has provided Zolgensma (onasemnogene abeparvovec) free of charge to nearly 300 children with the genetic disorder across 36 countries where the therapy has not yet received approval or in which no formal access pathway exists.

Gene Therapy 119

Gene Therapy Genetics Gene Clinical Trials 119

Pharmaceutical Technology

APRIL 5, 2023

More than a decade after the first patient was treated with a CAR-T therapy, six therapies relying on the same principles have been approved by the US Food and Drug Administration (FDA) and marketed to thousands of patients. It will be six years since the US FDA approval of Kymriah in August this year.

Gene Therapy 147

Gene Therapy Gene Engineer Production 147

Rethinking Clinical Trials

MAY 25, 2023

Low Molecular Weight Heparin for Thromboprophylaxis: A Randomized Clinical Trial of Over 12,000 Orthopedic Trauma Patients (PREVENT CLOT) was conducted through METRC at 21 centers across the country. The study required an FDA exemption because Aspirin is not approved for this indication.

Trials 130

Trials Clinical Trials Clinical Trials Compliance 130

World of DTC Marketing

JUNE 23, 2021

One agency FDA adviser, resigning from his committee post in protest, called it “probably the worst drug approval decision in recent U.S. Source: STAT News Our healthcare system can’t continue to support drugs that don’t have good clinical outcomes behind them. Not to mention the $56,000 annual price tag.

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Drugs FDA Approval Doctors Doctor 218

pharmaphorum

OCTOBER 3, 2022

Amylyx Pharmaceuticals’ Relyvrio – a new treatment for amyotrophic lateral sclerosis (ALS) approved by the FDA on Thursday, after review of the data from its phase 2 trial – was the next day set at a list price of $158,000 per year in the US, sparking outcry. ALS affects over 30,000 people in the US.

Drugs 52

Drugs Generic Drugs FDA Approval Trials 52

pharmaphorum

AUGUST 21, 2022

Shares in Axsome Therapeutics have rocketed on FDA approval of its depression therapy Auvelity (formerly AXS-05) – a year after its approval was held up by the regulator. Auvelity is the first drug Axsome has taken through to regulatory approval, but could be the first of a string of new products for the company, according to Tabuteau.

Drugs 98

Drugs FDA Approval Production Marketing 98

Rethinking Clinical Trials

MARCH 1, 2023

They are both approved and used widely in lung cancer, and combining two existing agents is logistically and clinically simpler. S1800A was a Phase II randomized study of ramucirumab plus pembrolizumab versus standard of care for NSCLC patients previously treated with immunotherapy performed within the Lung-MAP platform. .

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Clinical Trials Clinical Trials Trials Packaging 130

pharmaphorum

OCTOBER 21, 2021

Analysts were predicting sales of Biogen’s recently approved Alzheimer’s therapy Aduhelm may start to gather a little momentum in the third quarter, but the drug seems to be going into reverse. Vounatsos also insisted that Aduhelm’s price tag has not been a factor discouraging treatment with the drug.

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Sales Doctors Doctor Gene Therapy 122

Drug Discovery World

JANUARY 30, 2024

Lentivirus strategies are one of the most widely used tools in biomedical research and clinical testing, says Stella Vnook, CEO, Likarda. “Dr He said: “This is something to celebrate as patients will have additional therapeutic options available to them, in some cases the therapies represent the first therapeutic option for patients.

Gene Therapy 71

Gene Therapy Manufacturing Gene Clinical Trials 71

Drug Discovery World

OCTOBER 31, 2023

UK regulatory authorities have approved the first trial of a gene therapy for young children with Hunter syndrome. The drug was developed over eight years by Brian Bigger, Professor of Cell and Gene Therapy at The University of Manchester.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

Drug Discovery World

DECEMBER 20, 2022

The therapy was also recently approved by the FDA for patients in the US. . The clinical development of etranacogene dezaparvovec was led by uniQure and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialise the treatment. . World’s most expensive drug.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

XTalks

FEBRUARY 27, 2024

Iovance Biotherapeutics’ Amtagvi (lifileucel) won US Food and Drug Administration (FDA) approval last week for the treatment of advanced melanoma, making it the first individualized tumor-infiltrating lymphocyte (TIL) therapy and the first T-cell therapy for a solid tumor to win US regulatory approval.

FDA Approval 52

FDA Approval Manufacturing In-Vivo Trials 52

pharmaphorum

JUNE 8, 2021

The FDA “has failed in its responsibility to protect patients and families from unproven treatments with known harms” in approving Biogen’s Alzheimer’s disease drug Aduhelm. We decided to use AA as there is an expectation of clinical benefit. Accelerated approval is probably a reasonable middle ground here.

Drugs 98

Drugs Marketing FDA Approval Trials 98

Rethinking Clinical Trials

APRIL 27, 2023

The REB approved the MyTEMP request to use an altered patient consent process because the research was deemed of minimal risk to patients. Dixon, PhD MSc) appeared first on Rethinking Clinical Trials. degrees Celsius. Am I delivering great medicine when I’m not sure what to do here when there is practice variability?

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Trials Clinical Trials Clinical Trials Medicine 130

pharmaphorum

NOVEMBER 16, 2021

“We look forward to the full technology appraisal guidance (TAG) which is due to be published in early December 2021,” he added. Cenobamate was originally developed by South Korean biotech SK Biopharmaceuticals, and was approved as Xcopri in the US in 2019. The drug made almost $17 million in US sales in the third quarter.

Drugs 98

Drugs Medicine Clinical Trials Clinical Trials 98

XTalks

FEBRUARY 8, 2024

Pharma commercials at Super Bowl 2024 will include ones for newly approved menopause drug Veozah and a unique partnership between antacid giant TUMS and sports betting company Draft Kings. Bayer is preparing to file for FDA approval based on strong Phase III data it shared last month. In its first year on the market, Veozah earned 1.3

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Life Science Marketing Branding Drugs 105

pharmaphorum

DECEMBER 17, 2020

Her videos cover a host of topics from the science behind the immune system, side effects caused by the shots and vaccine hesitancy and the clinical and regulatory path to approval. She told the BBC that her approach on TikTok is “come for the entertainment, but stay for the science.” Dr Austin Chiang.

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Scientist Vaccination Vaccine Medicine 97

XTalks

JUNE 9, 2021

Biogen has been working vigorously over the past couple of years to get its potentially disease-modifying drug Aduhelm (aducanumab) approved for Alzheimer’s disease, and it has finally happened. This Monday, the US Food and Drug Administration (FDA) granted accelerated approval to the drug for the treatment of the disease.

Drugs 98

Drugs Clinical Trials Clinical Trials Trials 98

pharmaphorum

NOVEMBER 19, 2020

Lucentis (ranibizumab) was first FDA approved in 2006 for wet age-related macular degeneration (wet AMD) and has picked up approvals in related eye conditions ever since, although its patent expired in June.

FDA Approval 64

FDA Approval Marketing Drugs Clinical Development 64

Rethinking Clinical Trials

OCTOBER 13, 2022

HandiCAP Trial, Pragmatic Clinical Trial. We planned this trial with three centers and then the third center did not get the approval by the ethics committee, so we had to include 12 centers. Melanie Meersch-Dini, MD. Department of Anesthesiology, Intensive Care and Pain Medicine. University Hospital Münster, Germany. Key Points.

Trials 130

Trials Clinical Trials Clinical Trials Nurses 130

pharmaphorum

AUGUST 18, 2022

bluebird bio’s Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment for patients with beta thalassaemia who require regular blood transfusions. An FDA advisory committee recommended approval of Zynteglo on the strength of that data in June.

Gene Therapy 52

Gene Therapy FDA Approval Gene Clinical Trials 52

The Pharma Data

JANUARY 31, 2021

The phase 1 clinical trial was a randomized, double-blind and placebo-controlled study in 150 adult and elderly participants. These findings give confidence that Clover’s COVID-19 vaccine candidates are suitable for further clinical development.” About Trimer-Tag© Technology. . CHENGDU, China , Feb.

Clinical Trials 52

Clinical Trials Clinical Trials Vaccination Vaccine 52

pharmaphorum

JULY 25, 2022

The EMA’s human medicines committee has recommended approval of Johnson & Johnson’s Tecvayli as a fourth-line therapy for multiple myeloma , joining a growing group of BCMA-targeted therapies for the blood cancer.

Antibody 52

Antibody Medicine Drugs Trials 52