Clinical Research Informer

bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Pharmaceutical Technology

SEPTEMBER 30, 2022

After several setbacks, bluebird bio bounces back with two major FDA gene therapy approvals. Last month, Zynteglo (betibeglogene autotemcel), or beti-cel, was approved as a one-time potentially curative gene therapy for patients with beta-thalassaemia who require regular blood transfusions.

Gene Therapy

Gene Therapy FDA Approval Gene Genetic Disease

Biogen, Eisai Alzheimer’s drug lecanemab fast tracked by FDA

pharmaphorum

DECEMBER 24, 2021

Biogen and Eisai head towards the end of the year with some much-needed good news in their Alzheimer’s programmes, as the FDA awards a fast-track designation to lecanemab, their follow-up to recently approved Aduhelm. The post Biogen, Eisai Alzheimer’s drug lecanemab fast tracked by FDA appeared first on.

Drugs

Drugs FDA Approval Trials Marketing

Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years

Pharmaceutical Technology

JUNE 29, 2022

The US Food and Drug Administration (FDA) put a high-profile bluebird bio trial for sickle cell disease on partial clinical hold, and advisory panels deliberated over decisions involving gene therapies for amyotrophic lateral sclerosis (ALS), cerebral adrenoleukodystrophy (CALD), and beta-thalassemia.

Drugs

Drugs FDA Approval Marketing Development

The FDA and Aduhelm: WTF?

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. Biogen’s Aduhelm won FDA approval after discovering that some patients on higher dosages had displayed improvement of.39 Not to mention the $56,000 annual price tag.

Drugs

Drugs FDA Approval Doctors Doctor

Sarepta’s Elevidys Reaches Finish Line as First Gene Therapy Approved for Duchenne Muscular Dystrophy

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). After the vote, the FDA extended the decision date by one month due to concerns it raised in briefing documents before the committee meeting.

Gene Therapy

Gene Therapy Gene Protein FDA Approval

Roche’s Early Alzheimer’s Blood Test Gets FDA Breakthrough Device Designation

XTalks

APRIL 18, 2024

Roche has received Breakthrough Device designation from the US Food and Drug Administration (FDA) for its Elecsys pTau217 early Alzheimer’s blood test. There are currently no FDA-authorized blood tests for detecting Alzheimer’s disease.

Clinical Trials

Clinical Trials Clinical Trials In-Vitro Protein

New Rare Disease Drugs and Research Advancements

XTalks

FEBRUARY 27, 2024

The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies. In 2020, 31 out of 53 novel drug approvals were for rare or orphan diseases. Both therapies were awarded their approvals on the same day in December 2023.

Research

Research Drugs Gene Therapy Clinical Trials

FDA Trims Broad Label for Biogen’s Alzheimer’s Drug Amidst Harsh Criticism

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. head of research and development at Biogen, in a statement from the company.

Drugs

Drugs Clinical Trials Clinical Trials Trials

Amtagvi (Lifileucel) Scores Landmark Win as First FDA-Approved T-Cell Therapy for a Solid Tumor

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. months of follow-up, 43.5

FDA Approval

FDA Approval Manufacturing In-Vivo Trials

Bluebird Bio’s Skysona Receives FDA Approval and Becomes World’s Most Expensive Drug

XTalks

SEPTEMBER 27, 2022

With a list price of $3 million and a recent accelerated US Food and Drug Administration (FDA) approval, bluebird bio’s gene therapy Skysona (elivaldogene autotemcel, eli-cel), has officially become the world’s most expensive drug. Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia.

FDA Approval

FDA Approval Gene Therapy Drugs Gene

Exit Aduhelm, enter lecanemab. Biogen and Eisai have another go

pharmaphorum

MAY 10, 2022

With Aduhelm largely sidelined, Biogen and Eisai are hoping for a better fortunes with their next Alzheimer’s disease therapy lecanemab, which is now fully filed with the FDA. The post Exit Aduhelm, enter lecanemab. Biogen and Eisai have another go appeared first on.

FDA Approval

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Biogen updates on its confirmatory Aduhelm trial

pharmaphorum

DECEMBER 17, 2021

Biogen and partner Eisai have said they will provide details next March for the confirmatory trial that will be needed to upgrade the drug’s accelerated approval for controversial Alzheimer’s drug Aduhelm to a full one.

Trials

Trials Sales Marketing FDA Approval

FDA slammed over Aduhelm OK as Biogen takes heat for $56k price tag

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. Accelerated approval is probably a reasonable middle ground here. Broad label questioned.

Drugs

Drugs Marketing FDA Approval Trials

Biogen’s Aducanumab Becomes First Drug Approved for Alzheimer’s in Nearly 20 Years Despite Controversy

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

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You can’t afford to get cancer

World of DTC Marketing

DECEMBER 13, 2021

As cancer survival rates rise, so do the price tags of life-saving treatments. 11 of the 12 cancer drugs that the Food and Drug Administration approved in 2012 were priced at more than $100,000 per year. OPENING: American cancer patients spent more than $21 billion on their care in 2019. billion included out-of-pocket costs of $16.22

Drugs

Drugs FDA Approval Medicine Containment

Life Sciences 2022 Year in Review

XTalks

DECEMBER 21, 2022

The past year has been a strong one for life sciences industries, from pivotal gene therapy approvals to continued innovations in the biotech and medical device spaces. Gene Therapy Approvals. Bluebird bio is a notable mention as it secured FDA approvals for two of its gene therapies, Zynteglo and Skysona.

Life Science

Life Science Gene Therapy Gene Clinical Trials

Gilead tags Merck for first-line triple negative breast cancer trial

pharmaphorum

OCTOBER 29, 2021

Take two recently-approved immunotherapies for triple negative breast cancer (TNBC), put them together, and what do you get? Trodelvy is also approved to treat metastatic urothelial cancer in the US, and Gilead has just reported third-quarter sales of $101 million for the drug, which were actually a little lower than analyst expectations.

Trials

Trials Sales FDA Approval Production

What challenges does the advanced therapies sector face?

Drug Discovery World

JANUARY 30, 2024

He said: “Increasing the level of automation and standardisation is not only the way to reduce manufacturing costs, but also paves the way for follow-on approvals once a platform technology is part of an approval. Fostering open innovation and collaboration could help address this issue.

Gene Therapy

Gene Therapy Manufacturing Gene Clinical Trials

AAIC 2021: Six Ways the Approval of Aducanumab Will Change Alzheimer’s Clinical Trials

XTalks

AUGUST 23, 2021

AAt this year’s Alzheimer’s Association International Conference ( AAIC ), entire sessions were dedicated to discussions of Biogen’s recently-approved, controversial drug Aduhelm (aducanumab), but their focus wasn’t what you’d expect. Now that [aducanumab] is approved, how will this affect clinical trials going forward?”.

Clinical Trials

Clinical Trials Clinical Trials Trials Drugs

Unveiling Potential: Exploring Alpha Emitter Radiopharmaceuticals

XTalks

JANUARY 19, 2024

Regulatory bodies have demonstrated preparedness for the surge in radiopharmaceuticals as evidenced by inaugural Chemistry, Manufacturing and Controls (CMC) Development and Readiness Pilot (CDRP) programs to expedite commercial manufacturing, US Food and Drug Administration (FDA) approvals and marketing authorizations.

DNA

DNA Radiology FDA Approval Development

HP&M Attorneys Receive Accolades: Top Lawyer Under 40 and WWL: Life Sciences 2023 Global Elite Thought Leader

FDA Law Blog

JUNE 27, 2023

Amongst his accomplishments, Law360 considered the role James has played in leveraging little-used pathways to FDA approval for often first-ever drugs to treat rare diseases (e.g., James’s status as a 15-year champion for bringing patient voices to the table with FDA and industry decision-makers was also considered.

Life Science

Life Science FDA Approval Drugs Development

PFIZER REPORTS STRONG FIRST-QUARTER 2021 RESULTS

The Pharma Data

MAY 4, 2021

up 28%, primarily driven by continued strong demand in the metastatic and non-metastatic castration-resistant prostate cancer indications, as well as the metastatic castration-sensitive prostate cancer indication, which was approved in the U.S. The FDA action converts the 2018 accelerated approval to full approval.

Vaccination

Vaccination Vaccine Production Sales

Clinical Research Informer

bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Biogen, Eisai Alzheimer’s drug lecanemab fast tracked by FDA

Trending Sources

Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years

The FDA and Aduhelm: WTF?

Sarepta’s Elevidys Reaches Finish Line as First Gene Therapy Approved for Duchenne Muscular Dystrophy

Roche’s Early Alzheimer’s Blood Test Gets FDA Breakthrough Device Designation

New Rare Disease Drugs and Research Advancements

FDA Trims Broad Label for Biogen’s Alzheimer’s Drug Amidst Harsh Criticism

Amtagvi (Lifileucel) Scores Landmark Win as First FDA-Approved T-Cell Therapy for a Solid Tumor

Bluebird Bio’s Skysona Receives FDA Approval and Becomes World’s Most Expensive Drug

Exit Aduhelm, enter lecanemab. Biogen and Eisai have another go

Biogen updates on its confirmatory Aduhelm trial

FDA slammed over Aduhelm OK as Biogen takes heat for $56k price tag

Biogen’s Aducanumab Becomes First Drug Approved for Alzheimer’s in Nearly 20 Years Despite Controversy

You can’t afford to get cancer

Life Sciences 2022 Year in Review

Gilead tags Merck for first-line triple negative breast cancer trial

What challenges does the advanced therapies sector face?

AAIC 2021: Six Ways the Approval of Aducanumab Will Change Alzheimer’s Clinical Trials

Unveiling Potential: Exploring Alpha Emitter Radiopharmaceuticals

HP&M Attorneys Receive Accolades: Top Lawyer Under 40 and WWL: Life Sciences 2023 Global Elite Thought Leader

PFIZER REPORTS STRONG FIRST-QUARTER 2021 RESULTS

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