Rare Disease

News

CHMP backs Amicus' therapy for Pompe disease

Amicus Therapeutics could be just weeks away from getting EU approval for its new Pompe disease therapy Pombiliti after getting a positive opinion on the drug from the EMA's human medicines

Views & Analysis

How eligible patient numbers affect price of rare disease dr...

Having recently assessed the relationship between drug price and prevalence of non-oncology rare diseases, CRA’s Life Sciences Practice team explores in greater depth the situation for thes

Views & Analysis

300 million people worldwide rare disease

Taking the right forecasting approach for rare diseases

There are approximately 7,000 different types of rare disease and researchers estimate that there are more than 300 million people worldwide living with such a condition, according to the N

Views & Analysis

connecting to the global patient community

How one pharma “family business” places patients first

When people think about pharma, they often think about “big” pharma: large, faceless corporations.

News

Young lonely man in a wheelchair on a balcony looking at the nature in the spring

Santhera seeks speedy FDA review of Duchenne drug vamorolone

Santhera has completed a rolling application for its Duchenne muscular dystrophy (DMD) therapy vamorolone in the US, setting up a possible approval and launch in the latter half of 2023.

News

abstract shapes, bone structure, 3d render, bone

Ipsen faces yet another delay for rare disease drug in US

Less than a week before Ipsen's palovarotene was due to go in front of an FDA advisory committee, the meeting has been called off following a request by the regulator for more data.