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. Shortly after this, the FDA announced the accelerated approval of bluebird's Skysona (elivaldogene autotemcel), or eli-cel, on 19 September.

Gene Therapy

Gene Therapy FDA Approval Gene Genetic Disease

STAT+: Pharmalittle: Vertex exec says prices for gene therapies are ‘in the ballpark’; insulin access in poor countries remains spotty

STAT News

OCTOBER 6, 2022

million tag as cost effective for a Bluebird Bio drug called Zynteglo for the blood disorder beta thalassemia, Arbuckle said in an interview. Bluebird also recently said it would charge $3 million for Skysona, the gene therapy it just launched for a rare brain-wasting disease.

Gene Therapy

Gene Therapy Gene Insulin Drugs

Can gene therapies for haemophilia defend their high price tags?

Pharmaceutical Technology

NOVEMBER 9, 2022

Reimbursement has been a major obstacle in the EU for expensive gene therapies, including bluebird bio’s beta-thalassemia therapy Zynteglo. The European Commission withdrew the marketing authorisation of Zynteglo, at the request of bluebird bio, in March 2022. million price tag. million ($1.8 million at the time).

Gene Therapy

Gene Therapy Gene FDA Approval Medicine

ICER says bluebird bio’s $2.1m gene therapy is cost-effective

pharmaphorum

APRIL 14, 2022

US cost-effectiveness watchdog ICER has handed bluebird bio some good news ahead of its FDA advisory committee meeting for rare blood disorder gene therapy beti-cel in June, by endorsing its proposed $2.1 million price tag. The post ICER says bluebird bio’s $2.1m

Gene Therapy

Gene Therapy Gene Marketing Clinical Trials

FDA Approval of First Rapid-Acting Oral Depression Drug + Approval of $2.8 Million Gene Therapy for Rare Blood Disorder – Xtalks Life Science Podcast Ep. 75

XTalks

AUGUST 31, 2022

Bluebird bio was awarded the approval for its gene therapy Zynteglo (beti-cel), which is a one-time treatment for the rare blood disorder. Hear more about the therapy, some of the setbacks bluebird has had to face on the road to its approval and why the treatment has a steep price tag.

Gene Therapy

Gene Therapy FDA Approval Life Science Gene

Second unanimous FDA adcomm vote boosts bluebird bio

pharmaphorum

JUNE 12, 2022

bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene therapy candidates that are key to its future as a commercially viable concern. Beti-cel has already been approved for marketing in Europe as Zynteglo, with a price tag of around $1.8

Gene Therapy

Gene Therapy Gene Marketing Trials

bluebird bio to split into oncology and gene therapy specialists

pharmaphorum

JANUARY 11, 2021

US biotech bluebird bio has announced plans to split into two this year, with a separate oncology business spinning off as the company prepares to bring its products to market. Meanwhile the as-yet unnamed oncology company will spin off under the leadership of bluebird’s current chief executive Nick Leschly. million price tag.

Gene Therapy

Gene Therapy Gene Genetic Disease Manufacturing

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

DECEMBER 13, 2023

The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel). bluebird expects cell collection for the first Lyfgenia patients to begin in the first quarter of 2024.

Gene Therapy

Gene Therapy FDA Approval Gene In-Vivo

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

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. bluebird has opted to launch it at a cost of $2.8 The post bluebird unveils $2.8m

Gene Therapy

Gene Therapy FDA Approval Gene Clinical Trials

J&J gets first approval for multiple myeloma bispecific Tecvayli

pharmaphorum

AUGUST 25, 2022

It joins a number of other BCMA-directed therapies for multiple myeloma, including GSK’s first-to-market antibody-drug conjugate Blenrep (belantamab mafodotin), Bristol-Myers Squibb/bluebird bio’s CAR-T therapy Abecma (idecabtagene vicleucel) and J&J/Legend Biotech’s CAR-T Carvykti (ciltacabtagene autoleucel).

Antibody

Antibody Marketing Drugs Medicine

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

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

pharmaphorum

JULY 25, 2022

.” GSK’s antibody-drug conjugate (ADC) Blenrep (belantamab mafodotin) was the first BCMA-targeting drug to reach the market in 2020, and has since been joined by Carvykti as well as Bristol-Myers Squibb and bluebird bio rival CAR-T therapy Abecma (idecabtagene vicleucel).

Antibody

Antibody Medicine Drugs 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. Related: Bluebird’s $2.8M Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia.

FDA Approval

FDA Approval Gene Therapy Drugs Gene

Bluebird’s $2.8M Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia

XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Bluebird has a total of three gene therapies in its pipeline.

Gene Therapy

Gene Therapy FDA Approval Gene Clinical Trials

bluebird bio ‘baffled’ after NICE rejects beta-thalassaemia gene therapy

pharmaphorum

FEBRUARY 16, 2021

It’s back to the drawing board for bluebird bio and its discussions with NICE, which has rejected its beta thalassaemia gene therapy Zynteglo for regular NHS use in first draft guidance. The post bluebird bio ‘baffled’ after NICE rejects beta-thalassaemia gene therapy appeared first on. million in Europe.

Gene Therapy

Gene Therapy Gene Medicine Development

Roctavian okay sets up another gene therapy test for Europe

pharmaphorum

AUGUST 26, 2022

BioMarin will no doubt have an eye on the earlier experience of bluebird bio, which secured EU approval for rare disease gene therapy Zynteglo (betibeglogene autotemcel) in 2019 for beta thalassaemia with a price tag of around €1.6 million spread over five years.

Gene Therapy

Gene Therapy Gene Antibody Marketing

Life Sciences 2022 Year in Review

XTalks

DECEMBER 21, 2022

Manufacturer: bluebird bio, Inc. Manufacturer: bluebird bio Inc. Bluebird bio is a notable mention as it secured FDA approvals for two of its gene therapies, Zynteglo and Skysona. Currently, CSL Behring’s one-dose Hemophilia B gene therapy Hemgenix holds the title at $3.5 million for a single infusion.

Life Science

Life Science Gene Therapy Gene Clinical Trials

CHMP backs J&J’s myeloma CAR-T therapy Carvykti

pharmaphorum

MARCH 27, 2022

The decision comes less than a month after Carvykti was approved by the FDA as a fifth-line therapy for multiple myeloma, teeing up a tussle in the market with Bristol-Myers Squibb and bluebird bio spinout 2seventy’s Abecma (idecabtagene autoleucel) which was approved for a similar indication last year.

Antibody

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Clinical Research Informer

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

STAT+: Pharmalittle: Vertex exec says prices for gene therapies are ‘in the ballpark’; insulin access in poor countries remains spotty

Trending Sources

Can gene therapies for haemophilia defend their high price tags?

ICER says bluebird bio’s $2.1m gene therapy is cost-effective

FDA Approval of First Rapid-Acting Oral Depression Drug + Approval of $2.8 Million Gene Therapy for Rare Blood Disorder – Xtalks Life Science Podcast Ep. 75

Second unanimous FDA adcomm vote boosts bluebird bio

bluebird bio to split into oncology and gene therapy specialists

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

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

J&J gets first approval for multiple myeloma bispecific Tecvayli

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

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

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

Bluebird’s $2.8M Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia

bluebird bio ‘baffled’ after NICE rejects beta-thalassaemia gene therapy

Roctavian okay sets up another gene therapy test for Europe

Life Sciences 2022 Year in Review

CHMP backs J&J’s myeloma CAR-T therapy Carvykti

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