Birtamimab

Unassigned

New Medicines

Amyloid light-chain (AL) amyloidosis in patients with organ dysfunction - first-line in addition to plasma cell dyscrasia treatments

Information

New molecular entity
Prothena
Prothena

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

A humanised monoclonal antibody that acts by specifically targeting the amyloid that gets accumulated in the AL form of amyloidosis.
Amyloidosis is a rare disease with an incidence of 1-5 cases per 100,000 people per year. Light chain amyloidosis (AL) is a monoclonal plasma cell disorder very similar to multiple myeloma producing a fibril of monoclonal immunoglobulin (Ig). Organs involved include the heart, kidney, peripheral nerve, gastrointestinal tract, respiratory tract and nearly any other organ. AL was formerly known as primary amyloidosis [3]. 0.7 in 10,000 people in the EU are affected, equivalent to ~35,000 [4].
Amyloid light-chain (AL) amyloidosis in patients with organ dysfunction - first-line in addition to plasma cell dyscrasia treatments
Bladder instillation