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). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

FDA 59

FDA 59

STAT

OCTOBER 6, 2022

… Multimillion-dollar prices for a rival’s treatments with the potential to cure rare diseases are “in the right ballpark,” according to Stuart Arbuckle, chief operating officer at Vertex Pharmaceuticals, which is preparing to market its own treatment for sickle-cell disease , Bloomberg News tells us. Even

70

70

pharmaphorum

JANUARY 11, 2021

While bluebird has conducted pioneering work in gene therapy for blood disorders and in cancer cell therapy, products have been delayed by issues with filing data for the FDA. The gene therapy firm will be focused on its most important therapies in beta-thalassemia, cerebral adrenoleukodystrophy and sickle cell disease in the US And Europe.

FDA 66

FDA 66

pharmaphorum

JUNE 12, 2022

Cases of cancer associated with insertional mutagenesis, caused when the viral vectors used to deliver the gene therapy to host cells, have been seen with bluebird’s gene therapies but have been more apparent in trials involving its CALD and sickle cell disease candidates than beti-cel.

FDA 59

FDA 59

pharmaphorum

FEBRUARY 22, 2021

Last week, Bluebird said it was pausing its Zynteglo (betibeglogene autotemcel) programme in Europe after a phase 1/2 trial in patients with sickle cell disease (SCD) – another red blood cell disorder – turned up two cases of blood cancers, one of acute myeloid leukaemia (AML) and another of myelodysplastic syndrome (MDS).

Immunization 52

Immunization 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.

Pharmaceutical Companies 98

Pharmaceutical Companies Pharmaceutical Companies FDA 98

The Checkup by Singlecare

MARCH 21, 2023

Opioids should not be used as first-line therapy for short-term or chronic pain —with the exception being in end-of-life or palliative care, during active cancer treatment, and with sickle cell disease. Belbuca is a good alternative to opioid treatment that needs to be taken around the clock, but it may come with a hefty price tag.

Dosage 52

Dosage Controlled Substances Labelling Packaging 52

The FDA Law Blog

JUNE 27, 2023

Duchenne, Friederichs’s Ataxia, Sickle Cell Disease, Chagas, ALS). In addition to Mr. Karst, ten other HP&M Directors were tagged as “Recommended” by WWL: Life Sciences 2023: Ricardo Carvajal Robert A. Dormer Jeffrey N. Gibbs Paul M. Hyman Alan M. Kirschenbaum Allyson B. Mullen Frank J. Sasinowski Jeffrey K.

FDA 81

FDA 81

The Checkup by Singlecare

MAY 8, 2023

It’s important to note that the CDC’s recommendations do not apply to pain related to sickle cell disease, cancer, or patients receiving palliative or end-of-life care. Belbuca is a good alternative to opioid treatment that needs to be taken around the clock, but it may come with a hefty price tag. fibromyalgia ).

Controlled Substances 52

Controlled Substances Dosage Labelling FDA 52

STAT

DECEMBER 15, 2023

… Bluebird Bio claimed a large insurer has agreed to cover its newly approved sickle cell disease gene therapy, easing some investor worries about resistance from other payers over the high price of the treatment, Reuters says. But Bluebird’s $3.1

Insurance 95

Insurance FDA 95