pharmaphorum

JANUARY 9, 2023

” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1 Pause in UK access. million in the US and just under €2 million in Europe, which the company says is justified by its dramatic benefits when given as a one-shot therapy to patients with SMA. .

Gene Therapy 118

Gene Therapy Genetics Gene Clinical Trials 118

Drug Discovery World

OCTOBER 31, 2023

UK regulatory authorities have approved the first trial of a gene therapy for young children with Hunter syndrome. The post First gene therapy trial for Hunter syndrome opens in Manchester, UK appeared first on Drug Discovery World (DDW).

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

Pharmaceutical Technology

JULY 14, 2022

Due to the high price tags associated with these speciality medicines, innovators have naturally favoured big markets with high GDP such as the US and EU-5 (Germany, France, Spain, Italy, and the UK). The choice of where to launch an orphan drug is an important and difficult decision.

Drugs 147

Drugs Medicine Marketing Development 147

pharmaphorum

APRIL 26, 2021

In 2019 Cognitant and Addenbrook’s Hospital in Cambridge, UK piloted a programme that aimed to educate patients to improve adherence to rheumatoid arthritis medicines. It comprises more than 170 companies and employs 20,000 people across 20 countries worldwide.

Development 121

Development Medicine Production Marketing 121

Drug Discovery World

AUGUST 15, 2022

The event will take place on 4-5 Oct, London Excel, UK and topics will include: . Kanza also works on her own research following her PhD in the digitisation of scientific research, smart labs and the lab of the future, and semantic tagging of scientific data. Click here to register.

Drugs 52

Drugs Research 52

Pharmaceutical Technology

MAY 2, 2023

High-prescribing physicians surveyed by GlobalData across eight major markets (8MM: US, France, Germany, Italy, Spain, UK, Japan, and China) identified a broad range of unmet needs in the IO space (see Figure 1). Despite the significant advances that have been made in the IO field, a huge level of unmet need remains.

Genetic Engineering 130

Genetic Engineering Antibody Clinical Trials Clinical Trials 130

XTalks

JUNE 8, 2021

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. With a price tag of over $2.5 Baby Arthur: First UK Patient. Why is Zolgensma So Expensive?

Gene Therapy 110

Gene Therapy Gene Protein Trials 110

pharmaphorum

APRIL 20, 2021

Novartis has secured backing from NICE for its relapsing multiple sclerosis (RMS) therapy Kesimpta in the UK, just two weeks after the drug was approved by the national drugs regulator. . There are around 130,000 people with MS in the UK, with 7,000 new diagnoses a year.

Sales 110

Sales Medicine Drugs Marketing 110

pharmaphorum

JUNE 2, 2021

NICE has rejected routine NHS funding for Roche’s Evrysdi (risdiplam) for spinal muscular atrophy drug in draft guidance, a decision which doesn’t come as a surprise to patient association SMA UK. “NICE’s initial ‘no’ to approving risdiplam for NHS funding is not unexpected,” said SMA UK this morning.

Gene Therapy 52

Gene Therapy Gene Drugs Medicine 52

pharmaphorum

APRIL 5, 2022

Oxford, UK-based Immunocore said the approval is based on the phase 3 IMCgp100-202 trial, which showed that Kimmtrak achieved an unprecedented improvement in overall survival when added to current first-line therapy for mUM. Uveal melanoma is a rare cancer of the eye, which in the majority of patients is localised.

Sales 52

Sales Protein Drugs Trials 52

XTalks

APRIL 18, 2024

Previously, Roche secured an FDA breakthrough tag for another one of its Alzheimer’s blood tests that detects levels of pTau-181 and ApoE4 (apolipoprotein e4). However, the guidance doesn’t say whether it could serve as a study’s primary endpoint on its own.

Clinical Trials 52

Clinical Trials Clinical Trials In-Vitro Protein 52

pharmaphorum

APRIL 14, 2022

million price tag. The gene therapy has already been approved for marketing in the EU and UK, where it was launched at a cost of around $1.7 The FDA is currently reviewing beti-cel – also known as Zynteglo (betibeglogene autotemcel) – as a treatment for patients with beta thalassaemia who require regular blood transfusions.

Gene Therapy 63

Gene Therapy Gene Marketing Clinical Trials 63

pharmaphorum

SEPTEMBER 15, 2020

“Digitally native brands are typically agile businesses from the ground up, and agility and flexibility are part of their core makeup,” says Joe Holmes, vice president, marketing services, US at Tag. Jeremy Richter, Tag. Joe began his life at Tag in the UK, bringing efficiencies to his clients, working across all conceivable channels.

Branding 129

Branding Marketing Pharma Companies Life Science 129

XTalks

DECEMBER 13, 2023

XTALKS WEBINAR: Cryopreservation Best Practices for Cell & Gene Therapy Source Material Live and On-Demand: Monday, January 22, 2024, at 11am EST (4pm GMT/UK) Register for this free webinar to learn about cryopreservation, a vital step in the manufacturing of today’s cell and gene therapies. How do Casgevy and Lyfgenia Work?

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

pharmaphorum

FEBRUARY 22, 2021

One UK company that could be affected greatly by that is Oxford BioMedica , which has built a business around its LentiVector delivery platform used in Kymriah and other emerging therapies, according to Jefferies. million in Europe. The post Zynteglo halt re-ignites viral vector safety concerns; analysts appeared first on.

Gene Therapy 52

Gene Therapy Gene Gene Sequencing Gene Expression 52

pharmaphorum

NOVEMBER 24, 2022

One issue has been the cost of the drug, with health technology assessment agency NICE in the UK and the ICER organisation in the US both concluding its price tag means it is not a cost-effective option for health systems.

Trials 52

Trials Sales Drugs Marketing 52

XTalks

OCTOBER 26, 2021

The treatment came with an initial price tag of $39,000 per vial, which amounts to a total of $575,00 each year. Two sisters living in the UK with AIP described their experience having the disease in a BBC article. However, Alnylam recently offered a confidential discount just before NICE’s latest recommendation came out.

Gene Silencing 98

Gene Silencing Gene Clinical Trials Clinical Trials 98

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. Professional reception of the findings.

Development 52

Development Drugs Clinical Development Antibody 52

pharmaphorum

DECEMBER 2, 2020

It was at this time they set their site at Sandwich in the UK, initially just to finish processing compounds imported from America, but due to tariffs on imported products the company rapidly expanded the plant to accommodate producing medicines from scratch. For all the latest Pfizer news follow pharmaphorum’s Pfizer tag.

Sales 128

Sales Production Drugs Marketing 128

pharmaphorum

NOVEMBER 27, 2022

In recent years, the UK pharmaceutical industry has relied heavily on China and India as providers of active pharmaceutical ingredients. Supply chain challenges take many forms in pharma – from capacity issues, to managing excess safety stock, and ensuring accurate supplier communication. Reducing capacity issues.

Production 105

Production Manufacturing Drugs Marketing 105

XTalks

FEBRUARY 27, 2024

Addressing the high price tag, Iovance’s commercial chief Jim Ziegler said, “Payers have expressed their appreciation for the value proposition for Amtagvi.” Vogt said Iovance set Amtagvi’s price based on its value as the first drug approved in the post-PD-1 melanoma setting, and the company’s analysis of relevant benchmarks.

FDA Approval 52

FDA Approval Manufacturing In-Vivo Trials 52

Druggist

OCTOBER 25, 2020

Calamine lotion available in the UK for management of chickenpox symptoms contain a mixture of calamine (15%) and zinc oxide (5%). Nevertheless, calamine lotion is licensed in the UK for symptomatic relief of skin conditions including itchiness. Buy on Amazon Price incl.

Production 52

Production Pharmacy Licensing Licensing 52

pharmaphorum

FEBRUARY 16, 2021

In this first draft guidance NICE raised a series of issues with Zynteglo, which bluebird has already agreed to supply at a confidential discount from its hefty price tag, which is around €1.57 million in Europe. NICE said that data came from a small sample of patients and is using its standard discount rate of 3.5%

Gene Therapy 89

Gene Therapy Gene Medicine Development 89

XTalks

AUGUST 19, 2022

Live and On-Demand: Tuesday, October 4, 2022, at 11am EDT (4pm BST/UK). Zynteglo’s hefty price tag of $2.8 XTALKS WEBINAR: How Agile Clinical Trials Unlock Universal Access to Rare Disease Research. The cells are prepared outside of the body ( ex vivo ) and then infused into the patient.

Gene Therapy 95

Gene Therapy FDA Approval Gene Clinical Trials 95

XTalks

FEBRUARY 27, 2024

Despite high price tags of a couple of million dollars, gene therapy makers say they are working to ensure patients are able to access the treatments through patient assistance programs developed in conjunction with the support of governments. Sarepta hopes to clarify Elvidys’ effectiveness in older children in a confirmatory trial.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

The Pharma Data

MARCH 20, 2021

Manager, Weatherall Institute, Oxford UK. L volumes, even for notoriously difficult liquid classes like the 100% acetonitrile used to store the Tandem Mass Tags (TMT). If you want to do a lot. of Covid tests, clever. little machines [like. the MANTIS] can easily beat big impressive looking robots. – Neil Ashley, Core Facility.

Reagent 52

Reagent RNA Bioinformatics Medicine 52

Drug Discovery World

NOVEMBER 9, 2023

Gail Calvert, Field Application Scientist, Sartorius UK, on: ‘Leveraging integrated and advanced technologies for successful cell line development’. The two luncheon presentations include: Maurice Brozzo, Global Antibody Specialist, Charles River, on: ‘The antibody discovery journey: from next-generation libraries to efficacy and safety’.

Engineer 52

Engineer Antibody Protein In-Vivo 52

The Pharma Data

MAY 5, 2021

UK Spinraza Data Review Shows Benefits Of NICE Flexibility ( Pink Sheet ). AbbVie’s Humira regains top pharma TV spender spot, while sibling brands Rinvoq and Skyrizi tag along ( Fierce ). . Connecticut becomes the first state with half of all adults fully vaccinated amid slowing pace of U.S.

Vaccination 52

Vaccination Vaccine Gene Therapy Contract Packaging 52

Pharmaceutical Technology

NOVEMBER 30, 2022

The UK and US are both undergoing an obesity crisis. The Health Survey for England 2019 reported that 28% of adults in the UK were obese and 36.2% The company’s weight loss drug Saxenda (liraglutide) won an approval for chronic weight management in December 2014, and Wegovy (semaglude) got the same tag in 2021. compared to a 5.1%

Drugs 264

Drugs Manufacturing Hormones Contract Manufacturing 264

Pharmaceutical Technology

NOVEMBER 30, 2022

The UK and US are both undergoing an obesity crisis. The Health Survey for England 2019 reported that 28% of adults in the UK were obese and 36.2% The company’s weight loss drug Saxenda (liraglutide) won an approval for chronic weight management in December 2014, and Wegovy (semaglude) got the same tag in 2021. compared to a 5.1%

Drugs 246

Drugs Manufacturing Hormones Contract Manufacturing 246

Pharmaceutical Technology

MARCH 17, 2023

In some cases, she said, “[drug] innovation would have been otherwise hindered by their price tag alone”. Watt explained that for newer, first-in-class drugs these types of agreements can also give healthcare systems safety in their decisions to approve certain drugs. Click here to watch this webinar and access the presentation.

Marketing 257

Marketing Drugs Research Analyst Manufacturing 257

Pharmacy Checkers

JANUARY 30, 2020

Tagged the “pharma bro” for the cavalier attitude he fronted about his greed, Martin is actually currently behind bars. For those Americans who might have been caught needing Daraprim and unable to afford it, Daraprim was available to order from the UK for a 99% discount. pill in the UK. but it’s now apparently down to $1.33/pill

Drugs 54

Drugs Generic Drugs Cosmetics FDA Approval 54