dm+d

Unassigned

New Medicines

Paroxysmal nocturnal haemoglobinuria (PNH) - add-on therapy to C5 inhibitor in adults

Information

New molecular entity
Alexion AstraZeneca Rare Disease
Alexion AstraZeneca Rare Disease

Development and Regulatory status

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

Category

Complement factor D inhibitor
UK prevalence is estimated at approximately 1 per 62,500. PNH is a chronic disease with haemolytic crises that may be triggered by several factors such as infection, vaccination, surgery or certain antibiotics. Bone marrow failure may occur prior, along, or as a late complication of the disease (40-50% of cases) [2].
Paroxysmal nocturnal haemoglobinuria (PNH) - add-on therapy to C5 inhibitor in adults
Oral

Further information

Yes

Evidence based evaluations