dm+d

Unassigned

New Medicines

Primary and secondary immune thrombocytopenia

Information

New molecular entity
Sanofi
Principia

Development and Regulatory status

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

Category

Brutons tyrosine kinase (BTK) reversible inhibitor
Annual incidence is between 2 and 3 per 100,000, and prevalence around 9 - 10 per 100,000. There is a slight bias to females (1.3:1) and age peaks below 18 and over 60 [2,3]
Primary and secondary immune thrombocytopenia
Oral

Pemphigus vulgaris (PV) and pemphigus foliaceus (PF)

Information

New molecular entity
Sanofi
Principia

Development and Regulatory status

Discontinued
Discontinued
Discontinued
Yes
Yes
Sep 21Development discontinued based on the PIII PEGASUS trial results [12].

Category

Brutons tyrosine kinase (BTK) inhibitor (BTK is involved in signalling pathways for most white cells)
Pemphigus vulgaris (PV) is by far the most common variant of pemphigus though this variant is rare. UK incidence is estimated to be 0.68 per 100,000 person years [2].
Pemphigus vulgaris (PV) and pemphigus foliaceus (PF)
Oral

Trial or other data

Sep 21Company announces PIII PEGASUS trial of rilzabrutinib for pemphigus failed to hit the primary endpoint (complete remission from weeks 29-37 with minimal doses of corticosteroids). The data shows there was no significant difference between rilzabrutinib and placebo. [8]