Polycythemia vera is a chronic myeloproliferative neoplasm characterized by clonal overproduction of red blood cells and increased risk of thrombosis, often associated with JAK2 mutations.
January and March 2026 drug pipeline updates included Phase 3 results, Fast Track designations and Priority Review decisions across multiple disease areas. Mavacamten, teplizumab-mzwv, anifrolumab, AXS-05, gedatolisib and AFTX-201 were among the programs with notable milestones.
The myelofibrosis therapeutic pipeline now includes over 40 candidates from more than 35 companies, with recent developments including Orphan Drug Designation for CK0804 and multiple Phase III trial initiations. Key clinical milestones include completed enrollment in Karyopharm's SENTRY trial and upcoming Phase I data from Incyte's combination therapy studies.
Protagonist Therapeutics reported fourth quarter and full year 2025 financial results, with two NDAs submitted and multiple Phase 3 trials advancing. The company holds $646 million in cash and expects regulatory decisions in 2026.
The FDA has accepted New Drug Applications for rusfertide in polycythemia vera with Priority Review and tirabrutinib for relapsed/refractory primary CNS lymphoma under Accelerated Approval, with PDUFA dates set for Q3 2026 and December 2026 respectively.
The FDA has accepted the New Drug Application for rusfertide, a first-in-class hepcidin mimetic peptide for polycythemia vera, and granted Priority Review with a PDUFA goal date in the third quarter of 2026.
Disc Medicine received a complete response letter from the FDA for bitopertin as a treatment for erythropoietic protoporphyria. The FDA acknowledged the drug lowers PPIX levels but requires evidence from the ongoing Phase 3 APOLLO study before approval.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07518940 |
ENERGIA: Personalized Exercise Program for Fatigue in Patients With Myeloproliferative Neoplasms and Chronic Myeloid Leukemia |
NOT_YET_RECRUITING | NA |
| NCT07429266 |
A Study of Sapablursen Evaluating the Safety and Efficacy in Participants With Polycythemia Vera (PV) |
RECRUITING | PHASE3 |
| NCT07362225 |
MPN PROGRESSion Registry: Observational Study Tracking Symptoms, Treatments, and Disease Progression in People With Myeloproliferative Neoplasms (MPNs) |
RECRUITING | |
| NCT07203768 |
A ELN-Multicenter Study on Phenotypic Evolution and Clinical Outcomes |
RECRUITING | |
| NCT07012109 |
Study of BEBT-507 Injection in Subjects With Polycythemia Vera (PV) |
NOT_YET_RECRUITING | PHASE1 |
| NCT06923670 |
Prevalence Of Germline Gene Mutations In Patients With Myeloproliferative Neoplasms With Family History |
NOT_YET_RECRUITING | NA |
| NCT06785870 |
EVAluation of Erythrocytosis PRospEctive Cohort STudy |
NOT_YET_RECRUITING | |
| NCT06752941 |
LOW-PV Continuation |
RECRUITING | |
| NCT06752746 |
A Study of Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of 9MW3011 in Patients With Polycythemia Vera |
RECRUITING | PHASE1 |
| NCT06743035 |
Ropeginterferon Alfa-2b in Patients With Polycythemia Vera (PV) Without Symptomatic Splenomegaly |
RECRUITING |
