$1-Billion-Dollar deals – changing tides for rare disease treatments?

It’s estimated that only 5% of known rare diseases have an approved treatment, and that at the current rate of approvals, it would take around 100 years to develop a single treatment for every rare disease.  

Typically, it’s been difficult to develop commercially viable treatments for rare diseases due to the limited patient populations and lack of tailored business models. But in the last few years things have changed.  

According to Evaluate Pharma, Orphan drugs are now “the fastest growing segment of the pharma market and dominate FDA approvals”. In fact, the market for rare diseases is “expected to expand at a CAGR of 12.5% from 2022-2030”.  

This is underscored by several $1 billion dollar plus acquisitions in the last year, of businesses with recent or pending approvals for rare disease treatments.  

• Ipsen acquires Albireo for $952m

Albireo had two recently approved products in rare liver disorders. Read more 

• Chiesi Farmaceutici acquires Amryt Pharma for $1.25bn  

Amryt had three approved products including for, lipodystrophy, hypercholesterolemia and acromegaly. 

Read more 

• Amgen acquires Horizon Therapeutics for $28.7bn

Horizon had three products for rare inflammatory diseases 

Read more 

• AstraZeneca acquires Amolyt Pharma for $800m + milestone payments

Amolyt had three assets for rare endocrine diseases, one in phase 3 trials

Read more 

So, with 95% of rare diseases still needing a treatment, and with the economics now starting to become sustainable, there’s hope for those 400m rare disease patients worldwide.  

This gives me confidence, that our approach at Ambrose is exactly what is needed right now. Our aim is to bring treatments for rare diseases to patients quickly while also providing a financially attractive outcome for investors. At Ambrose, we’re aiming for a double bottom line – providing sound economics and getting life-changing treatments into the hands of patients.  

With thousands of rare conditions still without any treatments, and 400m rare disease patients world-wide, there is plenty of space for experienced teams to step up and make a difference.