cerebral adrenoleukodystrophy
Bristol Myers Squibb’s New Drug Application for mezigdomide accepted by FDA
Mezigdomide improves progression-free survival in patients with relapsed or refractory multiple myeloma
Teva submits FDA application for paediatric Tourette syndrome therapy
Ecopipam could become the first new FDA-approved treatment for paediatric Tourette syndrome in over ten years
Roche’s Enspryng given FDA Priority Review as thyroid eye disease treatment
This is the first and only at-home subcutaneous treatment option for this disease
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
Roche’s Tecentriq gets FDA Priority Review for specific stage 3 colon cancer
Over one million people are diagnosed globally each year and around 30% of stage 3 patients relapse within five years
Lilly’s Ebglyss approved by FDA as maintenance dose for atopic dermatitis
This new option for patients allows for as few as six maintenance injections per year
FDA gives Rare Paediatric Disease designation to Satellite Bio for urea cycle disorders
The mortality rates of UCDs exceeds 25%, with no curative treatment available for newborns
BMS’ Opdivo gets expanded FDA and EU approval for Hodgkin lymphoma
This type of cancer is the most common one diagnosed in teenagers
US FDA launches ultra-rare diseases framework
The draft guidance specifically discusses genome editing and RNA-based therapies
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
Diabeloop’s DBLG2 receives FDA and CE clearance for automated insulin delivery without meal input
While a lack of meal announcements can lead to lower glycaemic control, it reduces the mental burden for those with type 1 diabetes
Johnson & Johnson’s Darzalex Faspro regimen receives FDA approval for multiple myeloma
Multiple myeloma has an average five-year survival rate of 59.8% and is currently incurable