Pharmafile Logo
T40 2022 Cover
Diversity, Equity and Inclusion
Communiqué 53 Cover Image
- PMLiVE

Isis Pharmacueticals

- PMLiVE

AstraZeneca’s rare disease unit to acquire Pfizer’s early-stage gene therapies for $1bn

The transaction could potentially see staff associated with the portfolio move to Alexion

- PMLiVE

Roche’s spinal muscular atrophy therapy recommended by CHMP for newborns

Evrysdi is already approved in the EU to treat SMA patients aged two months or older

- PMLiVE

Roche partners with Alnylam on hypertension therapy in deal worth up to $2.8bn

Zilebesiran is in phase 2 development to treat hypertension in patients with high-unmet needs

- PMLiVE

Roche’s Ocrevus shows promise as a subcutaneous injection in phase 3 MS trial

The therapy is already approved for certain MS patients as a twice-yearly intravenous infusion

- PMLiVE

Roche’s fixed-duration lymphoma treatment approved by European Commission

Approximately 36,000 people are diagnosed with DLBCL each year in Europe

- PMLiVE

LifeArc launches rare disease programme with £100m investment

An initial £40m will go towards the creation of up to five rare disease research centres

- PMLiVE

Horizon shares positive results for Uplizna in neuromyelitis optica spectrum disorder

The rare and debilitating disease affects approximately one to ten per 100,000 people

- PMLiVE

NIHR and MRC establish UK Rare Disease Research Platform with £14m investment

The platform brings together expertise from across the UK rare disease research system

- PMLiVE

Roche’s Evrysdi shows continued improvement in children with spinal muscular atrophy

The progressive neuromuscular disease affects approximately one in every 10,000 babies

- PMLiVE

FDA approves UCB’s Rystiggo to treat generalised myasthenia gravis

The rare autoimmune disease has a global prevalence of 100 to 350 cases per every one million people

- PMLiVE

Roche’s fixed-duration lymphoma treatment granted FDA accelerated approval

Globally around 160,000 people are diagnosed with non-Hodgkin's lymphoma each year

- PMLiVE

FDA approves Blueprint Medicines’ Ayvakit for indolent systemic mastocytosis

Ayvakit is now the first and only approved medicine designed to treat the underlying cause of the disease

Subscribe to our email news alerts

Latest jobs from #PharmaRole

Latest content

Latest intelligence

Quick links