STAT

AUGUST 2, 2023

The first drug purporting to slow the advance of Alzheimer’s disease is likely to cost the U.S. Besides the $26,500 annual price tag, treatment could cost U.S. Besides the $26,500 annual price tag, treatment could cost U.S. Medicare and Medicaid patients will make up 92% of the market for the drug, called Leqembi.

Insurance Coverage 76

Insurance Coverage Insurance 76

pharmaphorum

JANUARY 9, 2023

First introduced in 2020, the global Managed Access Programme (gMAP) has provided Zolgensma (onasemnogene abeparvovec) free of charge to nearly 300 children with the genetic disorder across 36 countries where the therapy has not yet received approval or in which no formal access pathway exists.

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pharmaphorum

NOVEMBER 19, 2021

Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company. million price tag make it is the most expensive treatment ever approved for NHS funding.

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Pharmaceutical Technology

MAY 2, 2023

While initially only approved for metastatic disease, ICIs have now moved into earlier disease settings, reducing the risk of disease progression and relapse. All currently approved CAR-Ts are autologous, with the patient’s T-cells being genetically engineered to target antigens expressed by the cancerous cells.

Immunization 52

Immunization Vaccines 52

pharmaphorum

JANUARY 11, 2021

Under the plans the company’s rare disease drugs will remain under the aegis of bluebird with current genetic disease president Andrew Obenshain taking the reins as CEO. million price tag. Zynteglo is already in beta-thalassemia in Europe, where the company will seek to expand access despite its hefty $1.8

FDA 60

FDA 60

Pharmaceutical Technology

JULY 14, 2022

It is estimated that there are currently more than 7,000 orphan diseases, many of which are considered life-threatening and most of which have a genetic basis. Despite this high number, orphan diseases are rare by definition, affecting around one in 2,000 people as defined by the European Union. Go-to-market strategies.

Pharmaceutical Companies 59

Pharmaceutical Companies Pharmaceutical Companies Hospitals 59

pharmaphorum

DECEMBER 23, 2022

price tag of $475,000 when it was first launched in 2017 – and it becomes apparent that these may not be desirable treatment options for every patient and in every setting. Add to this the considerable cost of these medications – the first approved CAR-T, Novartis’ Kymriah (tisagenlecleucel), had a U.S. CellCentric, a U.K-based

Immunization 52

Immunization Hospitals Vaccines 52

pharmaphorum

AUGUST 18, 2022

.” The gene therapy specialist’s chief executive Andrew Obenshain said the approval “marks a watershed moment for the field of gene therapy” and is “ushering in a new era in which gene therapy has the potential to transform existing treatment paradigms for diseases that currently carry a lifelong burden of care.”

FDA 52

FDA Insurance 52

pharmaphorum

JUNE 2, 2021

The cost-effectiveness agency’s initial assessment is that Evrysdi is simply too expensive at its current price to be provided to the roughly 1,500 people with the rare genetic disorder who might be eligible to receive it. Zolgensma was backed for type 1 SMA in draft guidance issued last month, even though the gene therapy’s £1.79

Documentation 52

Documentation Communication 52

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). million in Europe.

Immunization 52

Immunization 52

Roots Analysis

JANUARY 28, 2024

Velocity: Big Data needs to be handled promptly as businesses generate data at an unprecedented speed, driven by the growing adoption of Internet of Things (IoT), and technologies, such as RFID tags, sensors, and smart meters are used to deal with the high velocity of data in real-time. It is loosely arranged into categories using meta tags.

Health and Personal Care Stores 40

Health and Personal Care Stores Medical Records Hospitals 40

PharmaShots

MARCH 9, 2023

She developed various mobile technologies and pioneered early electronic labels including NFC tags, which helped Thin Film Electronics win the Wall Street Innovation Award and the Global Technology Award. Other Affiliations: She holds New York State Department of Health certification in Molecular Genetics and Molecular Oncology.

FDA 40

FDA Pharmaceutical Companies Pharmaceutical Companies Immunization 40

European Pharmaceutical Review

MAY 11, 2023

Rare, genetic diseases like SMA are prime targets for gene therapy, but by their very nature, early signs can be difficult to recognise, delaying diagnosis and treatment. 1 This is assuming a $1M price tag for all gene therapies. In reality, this will vary by disease area and therapy.

FDA 72

FDA 72

pharmaphorum

DECEMBER 7, 2021

Scientists in the US have found that people who use Viagra for erectile dysfunction seem to have a lower risk of developing Alzheimer’s disease – a link that they say should be examined more closely in clinical trials.

Compounding 52

Compounding Insurance FDA 52

European Pharmaceutical Review

NOVEMBER 10, 2022

With the new modalities used to treat disease and address previously undruggable targets, advanced drug delivery systems are critical to ensure targeted delivery, and to maximise safety and efficacy. BT-474 cell line was gene-edited to incorporate a green fluorescent protein (GFP) tag into late endosomes/lysosomes (green).

Labelling 98

Labelling Compounding 98

European Pharmaceutical Review

DECEMBER 27, 2023

Bridging therapy has been shown to correlate with an inferior response to CAR T-cell therapy due to both progression of the chemotherapy-refractory malignant disease during the manufacturing period and increased toxicity. This is in addition to its considerable price tag. 2021;138(24):2499–513. Schultz LM, Baggott C, Prabhu S, et al.

Adverse Reactions 86

Adverse Reactions Immunization Medical Schools Chemotherapy 86