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). The standard of care for the condition includes blood transfusions, hydroxyurea and iron chelation.

FDA 59

FDA 59

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. Beti-cel offers another potentially curative treatment for a much broader range of patients.

FDA 62

FDA 62

pharmaphorum

JUNE 12, 2022

The panel voted 13 to 0 on the question of whether beti-cel’s benefits outweighed the risks of the gene therapy in patients with beta thalassaemia who are dependent on blood transfusions, in a dream result for the biotech which just a few weeks ago was expressing doubts about its ability to continue as an operating concern.

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FDA 57

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).

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Immunization 52

pharmaphorum

MAY 17, 2021

C5 inhibitor Soliris – which has a list price in the US of more than $500,000 per year – has been a standard therapy for the rare disorder since 2007, and was joined on the market by longer-acting follow-up Ultomiris in 2018 which launched with a price tag of around $450,000 per year. billion and $1.1

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FDA Labelling Vaccines 52

The Checkup by Singlecare

NOVEMBER 2, 2023

These drugs mimic GLP-1, a hormone responsible for regulating appetite and food intake, leading to weight loss and improved control of blood sugar levels in people with Type 2 diabetes. These are the benefits of GLP-1 drugs , beyond blood sugar control. Diabetes Blood glucose control was the first known benefit of GLP-1 drugs.

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Insurance FDA Adverse Reactions Hospitals 52

The Checkup by Singlecare

JANUARY 23, 2024

Various factors can lead to a bump on the head, such as head trauma , infections, cysts , or abnormal cell growth, and although head bumps might cause some discomfort, they usually aren’t a cause for alarm. This can lead to the formation of a scalp hematoma —a localized collection of blood—that causes a visible bump on the head.

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