Date: 2018-09-10
Type of information: Granting of the orphan status in the US
Product name: SYNT001 - orilanolimab
Compound: orilanolimab
Therapeutic area: Autoimmune diseases - Rare diseases
Action mechanism:
- monoclonal antibody. SYNT001 is an investigational humanized IgG4 monoclonal antibody optimized to inhibit FcRn binding to IgG at both neutral and acidic pH. Studies have shown that SYNT001 rapidly facilitates clearance of IgG and IgG circulating immune complexes (CICs), with the potential to block innate immune responses induced by IgG and CIC, as well as inhibit T cell and B cell activation in response to CIC. Additionally, studies suggest that SYNT001 accomplishes its effects on IgG without destroying immune cells or impacting other types of immunoglobulin. SYNT001 has the potential to exert a rapid therapeutic effect in a wide range of IgG-mediated autoimmune diseases.
Company: Syntimmune (USA - MA)
Disease: pemphigus
Latest news:
- • On September. 10, 2018, the FDA has granted Orphan Drug Designation to SYNT001, Syntimmune’s lead investigational candidate, for the treatment of pemphigus.
- Positive preliminary results from a Phase 1b proof-of-concept trial of SYNT001 in pemphigus, a rare and severe autoimmune disease that leads to painful blistering, were reported at the International Investigative Dermatology meeting earlier this year. The preliminary results showed that SYNT001 appeared to be well-tolerated and induced a rapid reduction in IgG and circulating immune complex levels, as well as clinical improvement as measured by Pemphigus Disease Area Index (PDAI) score, with clinical effect persisting beyond the treatment period.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2018-09-10
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
