complement 3 glomerulopathy
Alexion and Aide Health launch app for people with rare diseases
More than 3.5 million people in the UK live with a rare condition
Shionogi to acquire IntraBio in deal worth $2bn
The deal will expand Shionogi’s growing portfolio with IntraBio’s rare diseases capabilities
Roche’s fenebrutinib receives FDA filing acceptance for relapsing and primary progressive MS
If approved, this treatment would offer a new option for the nearly one million Americans living with MS
Ogsiveo recommended by NICE to treat rare soft tissue tumours
Desmoid tumours are rare soft tissue tumours that can grow aggressively into nearby tissues
Novartis’ Cosentyx gets positive CHMP opinion for polymyalgia rheumatica
If approved, this would be the first IL-17A inhibitor licensed in Europe for adults with polymyalgia rheumatica
FDA approves Priovant’s Lisraya for dermatomyositis
This is the first oral drug treatment for this rare disease in adults
AstraZenca’s Etcamah combination therapy given FDA Accelerated Approval for breast cancer
The approval was based on phase 3 trial results showing a 56% risk reduction in disease progression
Pfizer and BioNTech’s XFG-adapted COVID-19 vaccine approved by US FDA
The 2026-2027 vaccine targets the XFG variant to more closely match current variants
Novartis announces trial results for remibrutinib in relapsing multiple sclerosis
MS affects nearly three million people worldwide and has three main types
LEO Pharma acquires rights for dersimelagon from Tanabe Pharma
The company will pay $435m in up front and near-term milestone payments
FDA grants Fast Track status for peanut allergy immunotherapy
The treatment uses peptides designed to retrain the immune system to tolerate peanuts
Novartis’ Pluvicto approved by FDA for metastatic hormone-sensitive prostate cancer
More than 186,000 men are diagnosed each year with mHSPC globally