herapeutic Goods Administration
LEO Pharma acquires rights for dersimelagon from Tanabe Pharma
The company will pay $435m in up front and near-term milestone payments
EMA recommends granting marketing authorisation approval for Nezglyal
The treatment is for cerebral adrenoleukodystrophy, a rare neurodegenerative disease
AstraZeneca’s Trixeo recommended by CHMP for approval in EU for asthma
Asthma affects over 360 million people worldwide, including over 40 million people in Europe
Almirall’s paediatric registration of Ebglyss for atopic dermatitis gets EMA acceptance
Atopic dermatitis affects a significant number of children and adolescents and often peaks in childhood
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
EMA celebrates diversity and inclusion in science and healthcare
The EU body underlines its commitment to diverse perspectives in medicines regulation
LEO Pharma to acquire Replay gene therapy platform
The acquisition will advance innovation for patients with rare skin diseases
Shionogi starts phase 2 clinical trial for Pompe Disease
The rare disease affects around one in every 22,000 people worldwide
Astellas’ application to EMA validated for treatment of muscle-invasive bladder cancer
Bladder cancer affects over 224,000 people annually in Europe, with over 30% of cases being muscle-invasive bladder cancer
EMA’s PRIME scheme launches three major new features
The scheme supports the development of treatments targeting unmet medical needs in the EU
PharmaQuest returns: EPA perspectives, through a rare disease lens
By Lottie Sequerra and Emily Morton-West
UCB’s Kygevvi receives positive CHMP opinion for thymidine kinase 2 deficiency
TK2d is an extremely rare disease characterised by severe and progressive muscle weakness