Pharmaceutical Technology

JUNE 12, 2023

Quell Therapeutics and AstraZeneca have entered a collaboration, exclusive option and licence deal for the development, manufacturing and commercialisation of engineered T-regulator (Treg) cell therapies for autoimmune diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Engineer 130

Engineer Gene Therapy Clinical Trials Clinical Trials 130

Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Peter Marks, head of FDA’s CBER – the organization responsible for regulating gene therapies.

Gene Therapy 52

Gene Therapy Development Gene Drugs 52

pharmaphorum

AUGUST 20, 2020

US biotech BioMarin has hit back with a filing for a rare disease drug, after suffering what was described by analysts as a “major setback” when the FDA rejected its gene therapy for haemophilia A. It also has three other gene therapies in its pipeline for phenylketonuria, hereditary angioedema, and hypertrophic cardiomyopathy.

Gene Therapy 55

Gene Therapy Drugs Regulation Gene 55

pharmaphorum

OCTOBER 12, 2021

Sales were $20 million and $0.5 Gocovri could see its sales momentum rise following its approval for a second indication by the FDA earlier this year. per share subject to Gocovri meeting sales targets. per share subject to Gocovri meeting sales targets. million, respectively, in the second quarter of this year.

Sales 93

Sales Gene Therapy Production Marketing 93

XTalks

MARCH 28, 2024

In November, the FDA extended its decision deadline by three months to allow extra time for regulators to review additional information submitted by Italfarmaco as part of a new drug application. It will also be going up against its breakthrough gene therapy Elevidys (delandistrogene moxeparvovec), which received FDA approval last year.

FDA Approval 105

FDA Approval Genetics Gene Therapy Gene 105

Pharmaceutical Technology

OCTOBER 27, 2022

The increasing regulation of biotech and manufacturing deals in the US could prompt foreign companies such as Chinese contract development and manufacturing organisations (CDMOs) to seek alternative targets in Europe or elsewhere. F-Star has key locations and subsidiaries based in the US.

Manufacturing 267

Manufacturing Contract Manufacturing Gene Therapy Gene 267

pharmaphorum

AUGUST 8, 2022

As we reported this morning, the deal gives Pfizer already-approved SCD therapy Oxbryta (voxelator) – which industry watchers reckon could see a dramatic uptick in sales with Pfizer’s marketing muscle – plus a phase 3 antibody candidate, a phase 1 follow-up to Oxbryta that could offer improved dosing.

Marketing 109

Marketing Sales Gene Editing Antibody 109

XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies. Here are four life sciences trends to look out for in 2023.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

pharmaphorum

MARCH 9, 2022

The XTEND-1 trial also showed that BIVV001 was better at reducing the annualised rate of bleeding episodes than patients’ previous therapies, according to Sanofi and Sobi, which said they will now talk to regulators to chart a path forward to approval.

Sales 98

Sales Protein Gene Therapy Regulation 98

XTalks

AUGUST 3, 2023

While most post-marketing activities are typically oriented towards marketing, sales, and commercialization of the drug, pharmacovigilance is fundamentally patient-centric, focusing on the safety and efficacy of pharmaceutical products once they reach the market.

Regulation 97

Regulation Marketing Life Science Drugs 97

pharmaphorum

JANUARY 4, 2022

Spinal muscular atrophy (SMA) therapy Spinraza has been a key earner for Biogen in recent years, but with sales now heading into reverse, the company is looking to extend its franchise. The arrival of Zolgensma was followed by a decline in Spinraza’s sales during 2020, particularly in the US.

Sales 52

Sales Gene Therapy Marketing Drugs 52

pharmaphorum

JULY 21, 2021

Novartis has revealed that the FDA has accepted its resubmission for cholesterol-lowering drug Leqvio – rejected unexpectedly by the US regulator last year – and has set a new action date of 1 January 2022. Meanwhile, sales of gene therapy Zolgensma for spinal muscular atrophy shot up more than 50% to $315 million.

Sales 52

Sales Gene Therapy RNA Production 52

The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52

The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. This route of administration has the potential to open up access for older patients to all the benefits of gene therapy.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

pharmaphorum

AUGUST 12, 2020

The FDA has started its review of Israeli biotech Protalix BioTherapeutics and partner Chiesi’s Fabry disease therapy pegunigalsidase alfa, setting up a possible approval by 27 January. Market research firm Optima Insights has predicted that sales of Fabry disease drugs will more than double from around $1.8 billion last year to $3.8

Drugs 97

Drugs Genetic Disease Gene Therapy Trials 97

The Pharma Data

MAY 3, 2021

Chilean regulator sees no clouds over Sputnik vaccine despite Brazilian rejection ( Reuters ). Canada’s regulator holds Johnson & Johnson vaccine over Emergent link ( NYTimes ). Six patients with dementia went to Mexico for an unproven gene therapy, a biotech CEO claims ( STAT ).

Gene Therapy 52

Gene Therapy Vaccination Vaccine Bioequivalency 52

Drug Discovery World

SEPTEMBER 14, 2023

Excluding Covid-19 medicines, total revenue increased 16% and product sales increased 15%. For Sanofi, second quarter 2023 sales growth of 3.3% AstraZeneca saw total revenue reach $22,295m, up 4% despite a decline of $2,181m from Covid-19 medicines. at CER and business EPS growth of 8.1% Specialty care grew 11.8% Vaccines were up 9.1%

Drugs 59

Drugs Vaccination Vaccine Medicine 59

pharmaphorum

JANUARY 13, 2023

Cell and gene therapies – big opportunities, big challenges. Everyone agrees that cell and gene therapies are poised for an explosion, with far more drugs in the pipeline than are currently approved, and the FDA predicts it will clear 10 to 20 per year by 2025. But enough vagaries. Let’s talk trends.

Clinical Trials 98

Clinical Trials Clinical Trials Gene Therapy Trials 98

XTalks

AUGUST 2, 2023

Most of the company’s revenue comes from the manufacture and sale of biopharmaceutical products. billion in pharmaceutical sales from 2021. In contrast, several drugs experienced sale declines, such as Remicade (infliximab), an old immunology medication that continues to decline due to the rise of biosimilars since 2016.

Sales 98

Sales Manufacturing FDA Approval Life Science 98

XTalks

MARCH 22, 2023

The cholesterol-lowering statin received its biggest boost in sales when a study found it was linked to a lower risk of heart attacks among those with heart disease. More potential patients equal more prescriptions which equals higher sales and bigger profits. million, making it the most expensive drug in the world.

Life Science 97

Life Science Drugs FDA Approval Vaccination 97

Pharmaceutical Technology

APRIL 24, 2023

billion in Spikevax sales in 2022, and Comirnaty generating $37 million for Pfizer in 2022. GlobalData predicts that Comirnaty’s sales will increase by more than 20% between 2024 and 2027. Conde says the use of nanoparticle delivery for gene therapies has become more common over recent years.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147

The Pharma Data

MAY 5, 2021

Affinia Therapeutics raises $110 million for gene therapy treatments ( STAT ). Startup sets out to tackle the gene therapy manufacturing crisis ( Endpoints ). Zimmer sees Q1 sales growth after CEO warned of down quarter ( MedTech Dive ). Medtech. Medtech.

Vaccination 52

Vaccination Vaccine Gene Therapy Contract Packaging 52

pharmaphorum

NOVEMBER 30, 2022

As an industry, pharma has specific needs; it is driven by targeted regulations and rules and has not been well served by technology. Prioritisation and the evolution of the sales, medical, and marketing triumvirate. Veeva’s strategy is to help simplify that with excellence in and connection between sales, medical, and marketing.

Life Science 98

Life Science Pharma Companies Sales Marketing 98

Pharmaceutical Technology

DECEMBER 9, 2022

The move is particularly timely given the significant growth expected in global sales of cell and gene therapies. The deal allows the company to provide proprietary technology and knowledge steeped in fast-evolving genomic techniques that support effective decision-making and R&D program design.

Development 130

Development Production Manufacturing Vaccination 130

pharmaphorum

APRIL 16, 2021

Approval by regulators such as the FDA or EMA, as reported by the regulators themselves. Achievement, meaning commercial performance in the context of sales uptake, as measured by the IQVIA sales audits. We measured the pandemic impact on the launch of NAS at four levels.

Sales 88

Sales Marketing Medicine Doctors 88

The Pharma Data

OCTOBER 21, 2020

Nasdaq: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, today announced the pricing of its public offering of 4,687,500 shares of its common stock at a public offering price of $40.00 BOSTON, Oct. 22, 2020 (GLOBE NEWSWIRE) — Replimune Group, Inc.

Gene Therapy 40

Gene Therapy Immune Response Sales Gene 40

The Pharma Data

DECEMBER 17, 2020

Prior to SpringWorks, Smith was the executive vice president of Gene Therapy at Bamboo Therapeutics. Burger has held executive sales positions with two software-based medical device startups where he led commercialization strategy. Immune Regulation – U.K.-based based Immune Regulation Ltd.

Development 52

Development Gene Therapy Engineer Life Science 52

pharmaphorum

NOVEMBER 23, 2022

We looked at the areas of rare diseases, cell and gene therapy, vaccines – loads of different perspectives. But it’s a highly regulated industry, with significant risks associated with any non-compliant material that’s been approved. There is still a struggle in pharma, an increasing demand for promotional materials.

Life Science 98

Life Science Marketing Medicine Production 98

pharmaphorum

APRIL 28, 2022

When a company, a big pharma company, calculates the financial impact, they also consider the sales force that they need, the marketing. It’s a different business model, if you will,” Miquel said. “So So that is the trick. We as the rare disease, we’re catering to very small patient populations, but also specialists.

Pharma Companies 57

Pharma Companies Branding Development Marketing 57

pharmaphorum

FEBRUARY 16, 2022

The 1 January 2021 saw the UK’s regulator, the MHRA, formally join Project Orbis. Project Orbis is a framework for concurrent reviews and approval of promising cancer treatments by regulators in the US, Australia, Canada, Singapore, Switzerland, Brazil and Israel, as well as the MHRA.

Marketing 52

Marketing Life Science Medicine Gene Therapy 52

Drug Discovery World

NOVEMBER 13, 2023

Jeremy Fry, Dr, Director of Sales, ProImmune, on: ‘Mastering immunogenicity and biologics development’. Michael Traxlmayr, PhD, Group leader, CD Laboratory for Next-Generation CAR T Cells, University of Natural Resources & Life Sciences, on: ‘Novel concepts to regulate CAR T cell activity with small molecule drugs in vivo’.

Protein 59

Protein Antibody Engineer Scientist 59

Drug Discovery World

MARCH 29, 2023

No dynamic analysis is provided to support the review’s two-year across-the-board RDP reduction, despite the projected impact (28% reduction in non-contested sales of RDP-protected products, and a 15% decline in gross profits).” Effects analyses are missing for several more of the review’s proposals, Meulenbelt and Koutsoupia confirm.

Clinical Trials 52

Clinical Trials Clinical Trials Production Medicine 52

XTalks

AUGUST 30, 2022

Global sales of over-the-counter and prescription products related to central nervous system (CNS) disease added up to $86 billion in 2019. We know that a lot of different pathologies, especially as we’ve entered the era of molecular medicine and gene therapies, cause many of the same symptoms and work through final common pathways.

Trials 83

Trials Drug Delivery Drugs Gene 83