Pharmaceutical company Sanofi's novel oral BTK inhibitor, rilzabrutinib, has been granted orphan drug designation (ODD) by the US Food and Drug Administration (FDA).
The therapeutic is currently being investigated in two rare diseases; warm autoimmune haemolytic anaemia (wAIHA) and IgG4-related disease (IgG4-RD).
Both these diseases have significant unmet medical needs — with neither having any currently approved medicines capable of treating them.
The FDA grants orphan drug designation to therapies that address rare medical conditions affecting less than 200,000 people in the US.
Rilzabrutinib is currently under regulatory review in the US, the EU and China for its use in immune thrombocytopenia, with the FDA's decision expected in late August of this year.
It has also received ODD for this condition in the EU and Japan, with a Phase IIb trial finding it had a clinically meaningful effect on response rate, disease markers and glucocorticoid sparing.
“Orphan drug designation for these two rare, immune-mediated conditions validates our ongoing commitment to pursuing potential first- and best-in-class medicines for diseases that affect small populations but persist with unmet medical need," commented Karin Knobe, Global Head of Development, Rare Diseases at Sanofi.
"Our continued exploration of rilzabrutinib across multiple indications speaks to our belief in its potential for multi-immune modulation, as well as our belief in supporting treatment options, no matter how rare a condition.”