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.

FDA 115

FDA Insurance Patients Marketing 115

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

FDA 52

FDA Insurance Patients Medical 52

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).

Safety 111

Safety Pharmaceutical Patients Medicine 111

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).

FDA 95

FDA Marketing Safety Patients 95

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).

Medicine 52

Medicine FDA Recruitment Patients 52

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.

Food and Drug Administration 98

Food and Drug Administration FDA Marketing Pharmaceutical 98

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.

FDA 109

FDA Marketing Competition Patients 109