Swedish oncology-focused development company Vivesto AB (STO:VIVE) said on Tuesday that it has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for Cantrixil as a treatment for acute myeloid leukemia (AML), marking a regulatory milestone in the drug candidate's development.
This designation provides access to potential development incentives, including tax credits for qualifying clinical trials and exemptions from certain regulatory fees. Cantrixil could also qualify for seven years of US market exclusivity if it receives marketing approval and meets applicable requirements.
Cantrixil is currently in preclinical development for AML, with previous studies demonstrating anti-cancer activity both as a standalone treatment and in combination with established cancer therapies. A Phase I study of Cantrixil has also previously been conducted.
Vivesto said the designation, together with the preclinical findings and previous clinical results, strengthens the basis for advancing Cantrixil toward a Phase I study in AML. The company emphasised that orphan drug status does not constitute marketing approval or establish the drug candidate's safety or efficacy in AML.
Acumen Pharmaceuticals phase 2 Alzheimer's trial misses primary endpoint
Immunome secures EMA validation of varegacestat application for desmoid tumors
FDA approves Sun Pharma's ILUMYA for psoriatic arthritis
Alkermes initiates phase 2 study of ALKS 7290 for major depressive disorder
Phrontline Biopharma reports preliminary safety and antitumour activity for TJ101
HR HealthCare establishes charitable arm
Sobi presents phase 3 gout study results for pozdeutinurad
TME Pharma signs exclusive negotiation agreement with Japan's TMS for NOX-A12 rights
Formation Bio establishes USD600m debut fund for drug development
Ascletis reports positive US Phase II topline results for ASC30 in diabetes
MetaVia announces presentation of DA-1726 Phase 1 data at Precision Liver Symposium
Genmab reports 50% reduction in risk of death in Phase 3 head and neck cancer trial
United Therapeutics receives FDA clearance to advance inhaled PAH therapy into clinical trials
US WorldMeds acquires Orbus Therapeutics
DifGen Pharmaceuticals completes European Decentralised Procedure for Tafamidis 61 mg Capsules