PF-114 has been granted Orphan drug status in the US
Orphan indication: Treatment of chronic myeloid leukaemia (CML)
PF-114 mesylate is a novel oral tyrosine kinase inhibitor (TKI) that blocks native and mutated Bcr-Abl isoforms, including the gatekeeper mutant T315I, which is uniformly resistant to all approved first- and second generation TKIs.
Currently PF-114 undergoes Phase 1/2 in Russia:
Study to Evaluate Tolerability, Safety, Pharmacokinetics and Preliminary Efficacy of PF-114 for Oral Administration in Adults with Ph+ Chronic Myeloid Leukemia, which is Resistant to the 2nd Generation Bcr-Abl Inhibitors or has T315I Mutation in the BCR-ABL Gene.
https://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm
PF-114 mesylate is a novel oral tyrosine kinase inhibitor (TKI) that blocks native and mutated Bcr-Abl isoforms, including the gatekeeper mutant T315I, which is uniformly resistant to all approved first- and second generation TKIs.
Currently PF-114 undergoes Phase 1/2 in Russia:
Study to Evaluate Tolerability, Safety, Pharmacokinetics and Preliminary Efficacy of PF-114 for Oral Administration in Adults with Ph+ Chronic Myeloid Leukemia, which is Resistant to the 2nd Generation Bcr-Abl Inhibitors or has T315I Mutation in the BCR-ABL Gene.
https://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm
