eosinophilic oesophagitis
Sanofi announces phase 3 study data for infantile-onset Pompe disease
Pompe disease is a rare, progressive neuromuscular disease, with infantile onset being the most aggressive form
Sanofi’s Cenrifki approved in Europe for secondary progressive MS without relapses
SPMS is a debilitating stage of MS where patients experience increasing levels of disability
Regeneron’s New Drug Application for cemdisiran accepted for review by FDA and EMA
Globally, around 150 to 200 out of every million people have myasthenia gravis
Sanofi appoints Paulo Fontoura as Global Head of R&D
Fontoura previously held senior leadership roles at Xaira Therapeutics and Roche
Sanofi’s Sarclisa approved by MHRA as UK’s first anti-cancer treatment given via on-body injector
It is the first anticancer treatment in the UK and globally to be administered this way and a first in multiple myeloma
CSL’s Privigen approved as measles treatment by European Commission
Cases of measles have been rising in Europe since 2023
Regeneron agrees research collaboration with Parabilis Medicines
Regeneron will pay Parabilis $125m upfront, with up to $2.2bn in potential milestone payments and royalties
Sanofi’s Tzield gets expanded FDA approval for early-stage type 1 diabetes
The treatment can be used to delay the onset of clinical type 1 diabetes
PureTech’s deupirfenidone gets FDA and EC Orphan Drug Designations for idiopathic pulmonary fibrosis
Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or fewer than five in 10,000 individuals in the European Union
Sanofi announces positive results for venglustat in type 3 Gaucher disease
There are currently no approved treatments for the neurological symptoms of this disease
Sanofi’s Rezurock receives positive CHMP opinion for chronic graft-versus-host disease
Up to 50% of allogeneic haematopoietic stem cell transplant patients develop chronic GVHD
Sanofi and Regeneron’s Dupixent receives positive NICE final draft guidance for COPD
Around 1.4 million people in the UK are diagnosed with COPD, with two million thought to be undiagnosed