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metreleptin

- PMLiVE

bluebird bio shares therapy updates for 2022

The company provided information for its first two gene therapies and its lovo-cel BLA submission for sickle cell disease

Extraordinary lives: advancing change in rare diseases

Rare Disease Day is all about raising awareness and generating change for the 300 million people worldwide living with a rare disease. On this day across the globe, the rare...

Lucid Group Communications Limited

- PMLiVE

FDA issues final guidances for clinical cancer trials

The FDA is working to address inequities, target the right treatments to the right patients and speed progress against the most deadly and rare cancers

- PMLiVE

Janssen and Legend Biotech’s Carvykti receives FDA approval for patients with relapsed or refractory multiple myeloma

In the study, 98% of patients with relapsed or refractory multiple myeloma responded to a one-off treatment of the drug

- PMLiVE

Rare Disease Day – looking to the future with hope

On this year’s Rare Disease Day, Amanda Henkel, VP, Strategy, Nucleus X Consulting (part of Nucleus Global), reflects on recent advances in the rare disease setting.

Nucleus Global

- PMLiVE

Enhertu trial results show significant improvement for breast cancer patients

The treatment, jointly developed by AstraZenca and Daiichi Sankyo, was trialled in the pivotal DESTINY-Breast04 study

Biomarin

FDA keeps BioMarin’s gene therapy for phenylketonuria on hold

The decision follows data suggesting BioMarin’s experimental gene therapies may have the potential to progress cancers

- PMLiVE

AZ’s Saphnelo receives EU approval for systemic lupus erythematosus

There are an estimated 250,000 people living with SLE in Europe and this is the first new drug to gain EU approval in over ten years

- PMLiVE

Eli Lilly’s Omicron drug treatment gets emergency use authorisation from FDA

The drug can be used to treat adults with mild-to-moderate COVID-19, and children aged 12 or older, in specific emergency cases in non-hospital settings

- PMLiVE

FDA accepts Priority Review for Dupixent in children with moderate-to-severe atopic dermatitis

If approved, Dupixent would be the first biologic treatment available in the US for children aged six months to five years with the skin condition

- PMLiVE

FDA issues approval for Sanofi’s Enjaymo treatment for patients with CAD

The treatment is the first-of-its-kind for patients living with cold agglutinin disease, a rare blood disorder

- PMLiVE

NICE recommends AZ’s Forxiga for kidney disease

The recommendation is based on results from a phase 3 trial

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