A big step forward for those with amyloidosis!
The European Medicines Agency (EMA) granted Pfizer a positive recommendation for the approval of its treatment for amyloidosis, a rare disorder that occurs in patients with cardiomyopathy.
What is Amyloidosis?
Amyloidosis occurs when abnormal proteins called amyloids build up and form deposits. The deposits can collect in organs such as the kidney and heart. This can cause the organs to become stiff and unable to work the way they should. There are six main types of amyloidosis:
- Primary (AL) – with no known cause
- Secondary (AA) – caused by another disease, including some types of cancer
- Familial (ATTR & non-ATTR) – passed down through genes
- Dialysis-related (DRA)
- Senile systemic (SSA or Wild-Type)
Symptoms can vary, depending upon which organs are affected. Treatment depends on the type of amyloidosis you have. The goal is to help with symptoms and limit the production of proteins. If another disease is the cause, it needs to be treated.
First of its Kind
Pfizer’s drug in question, Vyndaqel, would be the first approved treatment for amyloidosis in the European Union and it couldn’t come fast enough: many patients only live 2-3 years following diagnosis.
What Happens Now?
The EMA’s recommendation will now be reviewed by the European Commission and a final decision is expected in the coming months.