pharmaphorum

AUGUST 25, 2022

The double-headed antibody binds to CD3 on T cells and redirects them to BCMA-expressing myeloma cells, with the aim of stimulating an immune attack on the tumour, which remains incurable despite a slew of new therapies reaching the market in recent years.

FDA 88

FDA Marketing Safety Patients 88

Pharmaceutical Technology

NOVEMBER 9, 2022

On November 2, the Institute for Clinical and Economic Review (ICER) released its updated evidence aimed at measuring the clinical effectiveness and cost of the two haemophilia gene therapies. Known by the brand name Roctavian, BioMarin’s haemophilia A therapy valoctocogene roxaparvovec could be fairly priced in the range of $1.95–1.96

Safety 111

Safety Pharmaceutical Patients Medicine 111

Pharmaceutical Technology

APRIL 5, 2023

More than a decade after the first patient was treated with a CAR-T therapy, six therapies relying on the same principles have been approved by the US Food and Drug Administration (FDA) and marketed to thousands of patients. More broadly however, several advancements are on the horizon for cell and gene therapies in 2023.

Food and Drug Administration 59

Food and Drug Administration Engineering Patients Education 59

pharmaphorum

DECEMBER 23, 2022

There’s been huge progress in treatments for blood cancer in recent years – but drawbacks of expensive CAR-T cell and injected antibody therapies have led drug developers to look at novel oral therapies as patient-friendly alternatives. Oral therapy data presented at ASH. CellCentric, a U.K-based

Side effects 52

Side effects Patients Medical Medicine 52

pharmaphorum

AUGUST 18, 2022

bluebird bio’s Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment for patients with beta thalassaemia who require regular blood transfusions. million for chelation therapy to remove excess iron from the body. million, which compares to the $1.7

FDA 52

FDA Insurance Patients Medical 52

pharmaphorum

JULY 25, 2022

The EMA’s human medicines committee has recommended approval of Johnson & Johnson’s Tecvayli as a fourth-line therapy for multiple myeloma , joining a growing group of BCMA-targeted therapies for the blood cancer.

Medicine 52

Medicine FDA Recruitment Patients 52

European Pharmaceutical Review

AUGUST 24, 2022

1 While the first successes underscored the potential of this class of targeted therapies, they also revealed bottle?necks 2 The role of the antibody is to deliver the payload to a specific site such as a cancer cell. 5 Thus, the antibody is able to protect the payload from unwanted interactions with circulating proteins and cells.

Engineering 86

Engineering Side effects Marketing Patients 86

Pharmaceutical Technology

JUNE 29, 2022

This year has already been eventful when it comes to the development of therapies for rare diseases. Additionally, pricing and access for rare disease therapies continue to be scrutinized closely. Most, if not all, of these therapies used the FDA’s Orphan Drug Designation to aid their development plans.

Food and Drug Administration 98

Food and Drug Administration FDA Marketing Pharmaceutical 98

Pharmaceutical Technology

FEBRUARY 10, 2023

On November 22, 2022, the FDA approved CSL Behring’s Hemgenix (etranacogene dezaparvovec), the first gene therapy treatment for hemophilia B, with a staggering manufacturer price of $3.5 This is not the first treatment to come with a high price tag.

Marketing 105

Marketing Medicine Leads Safety 105

PM360

APRIL 22, 2024

In the rapidly evolving landscape of healthcare, where breakthrough therapies and treatments emerge at an unprecedented pace, conventional boundaries are rapidly shifting. Post-launch, the role of Medical Affairs groups continues to evolve, focusing on driving patient outcomes and optimizing the use of therapies in real-world settings.

Medical 52

Medical Healthcare Healthcare Education 52

Pharmaceutical Technology

MAY 15, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely.

Pharmaceutical 52

Pharmaceutical Leads Biopharma Medicine 52

pharmaphorum

NOVEMBER 16, 2022

The advancements made in drug development over the last decade have seen the arrival of a number of treatments that offer one-time cures and more effective therapies for conditions in smaller patient populations. However, alongside this progress, Ben Hargreaves finds that there is also a conundrum over how to price these therapies.

Medicine 117

Medicine Marketing Healthcare Healthcare 117

Pharmaceutical Technology

JUNE 21, 2023

CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. per dose, it is the most expensive single-use gene therapy in the US.

Patients 98

Patients Food and Drug Administration Engineering Sales 98

pharmaphorum

AUGUST 26, 2022

BioMarin Pharma has its landmark first approval for Roctavian – the first for a haemophilia gene therapy – and will now have to see if that can convert that into a viable business in Europe. BioMarin has said Roctavian will be available at a ‘net’ price of around €1.5 million spread over five years.

FDA 106

FDA Marketing Competition Patients 106

European Pharmaceutical Review

MAY 11, 2023

Novartis shared long-term data for its SMA gene therapy in March 2023, which indicated significant long-term motor-milestone achievements in spinal muscular atrophy (SMA). Without this protein, the motor neuron cells shrink and eventually die. This causes debilitating and potentially fatal muscle weakness.

Food and Drug Administration 73

Food and Drug Administration Government Healthcare Healthcare 73

PM360

APRIL 7, 2023

The shift toward longer, more adequate timeframes to establish proof of concept can be as long as three to four years, 1 given the complex requirements for developing and launching specialty drugs and cell and gene therapies (CGTs) for individuals with complex conditions or rare or orphan diseases.

Manufacturing 52

Manufacturing Marketing Patients Safety 52

European Pharmaceutical Review

DECEMBER 27, 2023

Chimeric antigen receptor (CAR) T cells are the first example of a “living drug”. 1 Following in vitro production from T cells collected from a patient’s leukapheresis procedure, the CAR T cells are infused back into the patient’s blood, where they proliferate and expand. This is variable and to some extent unpredictable.

Manufacturing 86

Manufacturing Food and Drug Administration Patients Pharma 86