Clinical Research Informer

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

Pharmaceutical Technology

SEPTEMBER 30, 2022

Spark Therapeutics’ Luxturna, indicated for inherited retinal disease (IRD), was the first gene therapy to be approved, in 2017, with a price tag of $850,000 for each eye. Prior to bluebird's approvals, there were only two FDA-approved gene therapies for inherited conditions on the market.

Gene Therapy

Gene Therapy FDA Approval Gene Genetic Disease

Grand Rounds August 19, 2022: Inclusion and Diversity in Clinical Trials: Actionable Steps to Drive Lasting Change (Gerald Bloomfield, MD, MPH; Michelle Kelsey, MD)

Rethinking Clinical Trials

AUGUST 23, 2022

There is a national priority to increase diversity in clinical trials, from regulatory, funding agencies, industry, and others, so that the participant population reflects the U.S. In systematic reviews, community involvement and partnership with community-based organizations are universally cited as key to success. Key Points.

Clinical Trials

Clinical Trials Clinical Trials Trials Medicine

Axsome bounces back as FDA clears depression drug

pharmaphorum

AUGUST 21, 2022

Axsome’s drug was given a breakthrough designation from the FDA, reflecting the need for new treatment options in patients at increased risk of self harm or suicide, and was also given a priority review.

Drugs

Drugs FDA Approval Production Marketing

J&J gets first approval for multiple myeloma bispecific Tecvayli

pharmaphorum

AUGUST 25, 2022

A marketing application for teclistamab is also under review at the FDA, with a decision expected later this month or in early September. Tecvayli previously scored a priority medicines (PRiME) designation from the EMA and a breakthrough-therapy tag from the FDA.

Antibody

Antibody Marketing Drugs Medicine

Second unanimous FDA adcomm vote boosts bluebird bio

pharmaphorum

JUNE 12, 2022

Beti-cel has already been approved for marketing in Europe as Zynteglo, with a price tag of around $1.8 million price tag, saying its ability to help patients reach sustained transfusion independence justified its high price. In April, influential US cost-effectiveness watchdog ICER endorsed beti-cel’s proposed $2.1

Gene Therapy

Gene Therapy Gene Marketing Trials

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

XTalks

SEPTEMBER 27, 2022

The record-breaking price tag of Skysona is not surprising for one-time gene therapies like it. The price tag reflects “the clinical benefit [it] provides as an urgently needed treatment option to slow the progression of neurologic dysfunction in children impacted by progressive, irreversible and fatal rare disease,” Obenshain added.

FDA Approval

FDA Approval Gene Therapy Drugs Gene

Clover Biopharmaceuticals Announces Publication of Phase 1 Clinical Trial Data for its Adjuvanted COVID-19 Vaccine Candidates in The Lancet

The Pharma Data

JANUARY 31, 2021

Utilizing Clover’s proprietary Trimer-Tag© technology, S-Trimer is a trimeric SARS-CoV-2 spike (S)-protein subunit vaccine candidate. About Trimer-Tag© Technology. Trimer-Tag© is an innovative drug development platform which allows the production of novel, covalently-trimerized fusion proteins. —- End —-.

Clinical Trials

Clinical Trials Clinical Trials Vaccination Vaccine

Clover biopharmaceuticals raises $230 M; BrainStorm after ALS; BARDA grants $5.65 M LightDeck for SARS-CoV-2 antigen test; Libtayo for first-line advanced NSCLC subset

Delveinsight

FEBRUARY 23, 2021

The company plans to use the capital to expand its pipeline of protein-based vaccines and biologic cancer therapies utilizing its innovative and proprietary Trimer-Tag technology platform. This approval comes as the third one that followed a Priority Review by the FDA.

In-Vitro

In-Vitro Vaccination Vaccine Licensing

CHMP backs J&J’s myeloma bispecific Tecvayli, ahead of US decision

pharmaphorum

JULY 25, 2022

Tecvayli (teclistamab) is an off-the-shelf bispecific antibody targeting both BCMA and CD3 which is also under review at the FDA and if approved will slot into J&J’s myeloma therapy portfolio alongside Darzalex (daratumumab) – an antibody directed at CD38 – and BCMA-targeted CAR-T therapy Carvykti (ciltacabtagene autoleucel). .”

Antibody

Antibody Medicine Drugs Trials

bluebird unveils $2.8m price for gene therapy Zynteglo on FDA approval

pharmaphorum

AUGUST 18, 2022

million price tag attached to the therapy when it was first made available in Europe, and is also well above the proposed $2.1 That payback option was also factored into the ICER review, though notably over a five-year period. bluebird has opted to launch it at a cost of $2.8 million, which compares to the $1.7

Gene Therapy

Gene Therapy FDA Approval Gene Clinical Trials

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

pharmaphorum

MAY 10, 2022

The two partners have also requested a priority review for the Biologics License Application (BLA) for lecanemab, which if granted by the FDA could result in an approval before the end of the year.

FDA Approval

FDA Approval Trials Clinical Trials Clinical Trials

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

pharmaphorum

JUNE 8, 2021

That was the damning conclusion of the influential Institute for Clinical and Economic Review (ICER) in the US to yesterday’s approval of Aduhelm (aducanumab), the first new treatment for Alzheimer’s since 2003, which it says has “no evidentiary basis.”

Drugs

Drugs Marketing FDA Approval Trials

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

XTalks

FEBRUARY 27, 2024

Addressing the high price tag, Iovance’s commercial chief Jim Ziegler said, “Payers have expressed their appreciation for the value proposition for Amtagvi.” After addressing the assay issue, the FDA accepted its application in May 2023 under priority review.

FDA Approval

FDA Approval Manufacturing In-Vivo Trials

Deerfield/ Dana-Farber’s USD 130 Million Cancer Research Deal; FDA-Approval to BMS’ Breyanzi & TG Therapeutics’ Ukoniq; EU Marketing Rights for ViiV Healthcare’s Rukobia

Delveinsight

FEBRUARY 9, 2021

Breyanzi will come with a price tag of USD 410,300 wholesale price, said the company, justifying it after taking into consideration several factors including medical and clinical value, patient value, and societal value. The drug received priority review for the MZL.

FDA Approval

FDA Approval Marketing Research Protein

Life Sciences 2022 Year in Review

XTalks

DECEMBER 21, 2022

Despite the two blockbuster gene therapy approvals, the company sold one of its two priority review vouchers to Argenx SE for $102 million in November to raise some much-needed funds. The company is a leader in the gene therapy space and had been facing significant financial challenges for the past several years.

Life Science

Life Science Gene Therapy Gene Clinical Trials

Advanced therapies and the high-profile pricing dilemma

pharmaphorum

NOVEMBER 16, 2022

Previously, the discovery of small molecule treatments had been the priority, with their development and manufacturing process being an established formula. However, with a price tag of €1.58 However, a recently released report from the organisation could blow the price tag of Zolgensma out of the water. At more than $2.1

Gene Therapy

Gene Therapy Gene Marketing Development

Salesforce Platform Capabilities

Cloudbyz

JUNE 1, 2023

Security: Security is a priority at all Salesforce data centers. Users can also collaborate on files, add comments, and tag colleagues. They can review login attempts, IP addresses, and devices used for authentication. This includes common file types like PDFs, Microsoft Word documents, and image files.

Compliance

Compliance Development Branding Production

PFIZER REPORTS STRONG FIRST-QUARTER 2021 RESULTS

The Pharma Data

MAY 4, 2021

TicoVac (Tick-borne Encephalitis Vaccine) — In February 2021, Pfizer announced that the FDA accepted for Priority Review a Biologics License Application (BLA) for its tick-borne encephalitis (TBE) vaccine, as submitted for active immunization to prevent TBE in individuals 1 year of age and older. mg and norethindrone acetate 0.5

Vaccination

Vaccination Vaccine Production Sales

Clinical Research Informer

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

Grand Rounds August 19, 2022: Inclusion and Diversity in Clinical Trials: Actionable Steps to Drive Lasting Change (Gerald Bloomfield, MD, MPH; Michelle Kelsey, MD)

Trending Sources

Axsome bounces back as FDA clears depression drug

J&J gets first approval for multiple myeloma bispecific Tecvayli

Second unanimous FDA adcomm vote boosts bluebird bio

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

Clover Biopharmaceuticals Announces Publication of Phase 1 Clinical Trial Data for its Adjuvanted COVID-19 Vaccine Candidates in The Lancet

Clover biopharmaceuticals raises $230 M; BrainStorm after ALS; BARDA grants $5.65 M LightDeck for SARS-CoV-2 antigen test; Libtayo for first-line advanced NSCLC subset

CHMP backs J&J’s myeloma bispecific Tecvayli, ahead of US decision

bluebird unveils $2.8m price for gene therapy Zynteglo on FDA approval

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

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

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

Deerfield/ Dana-Farber’s USD 130 Million Cancer Research Deal; FDA-Approval to BMS’ Breyanzi & TG Therapeutics’ Ukoniq; EU Marketing Rights for ViiV Healthcare’s Rukobia

Life Sciences 2022 Year in Review

Advanced therapies and the high-profile pricing dilemma

Salesforce Platform Capabilities

PFIZER REPORTS STRONG FIRST-QUARTER 2021 RESULTS

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