Asset profile
Mitapivat · Pyrukynd
Unknown / undisclosed
Evidence outlook
Approved: Pyruvate Kinase Deficiency
Regulatory approval supersedes design-level evidence grading; the trials behind approval are preserved below as the drug's evidence history.
Also in development:Sickle Cell Disease · Phase 3Transfusion-Dependent Beta Thalassemia
How the evidence outlook works ↗
Featured trials
The studies most instrumental to this drug's clinical progression, individually graded on design and result. Pick an indication; Research unlocks the trials, endpoints, key results and grades.
Competitive landscape
17 competitors in this universe: 0 direct rivals (same mechanism and indication), 0 same-class, 17 same-indication.
| Drug | Stage | Modality | Mechanism | Evidence signal | Reported efficacygraded featured trials |
|---|---|---|---|---|---|
| Mitapivat (Pyrukynd): this drug | Approved | — | — | ||
| Crizanlizumab-Tmca (Adakveo) | Approved | Monoclonal antibody | P-selectin | ||
| Exagamglogene Autotemcel (Casgevy) | Approved | Gene therapy / editing | — | ||
| Hydroxyurea (Droxia) | Approved | — | — | ||
| Hydroxyurea (Siklos) | Approved | — | — | ||
| Hydroxyurea (Xromi) | Approved | — | — | ||
| L-Glutamine (Endari) | Approved | — | Amino Acid | ||
| Lovotibeglogene Autotemcel (Lyfgenia) | Approved | Gene therapy / editing | — | ||
| Voxelotor (Oxbryta) | Approved | — | — | ||
| Senicapoc | Phase 3 | — | — | ||
| Decitabine / Tetrahydrouridine (NDec) | Phase 2 | — | DNMT1 (DNA methyltransferase 1) | ||
| HBI-002 | Phase 2 | Small molecule | Carbon monoxide (CO)-based gas therapeutic; oral liquid CO formulation | ||
| EDIT-301 | Phase 1/2 | Cell therapy | — | ||
| ITU512 | Phase 1/2 | — | — | ||
| Nula-Cel | Phase 1/2 | Cell therapy | — | ||
| DISC-3405 | Phase 1 | — | — |
…and 2 more. Research shows the complete landscape.
Unlock the full comparison
Research adds each competitor's evidence grade and sourced trial results, aligned per race, so you can see who actually has the data.
Upcoming events
(5 total)
Estimated primary-completion dates from ClinicalTrials.gov. These are registry estimates, never confirmed readout dates; they can move as trials progress.
2028 (1)
February 2028 (1)
What to watch
2029 (4)
March 2029 (1)
What to watch
June 2029 (1)
What to watch
All registered studies
Every ClinicalTrials.gov study of this drug's own program, grouped by indication and phase. Research unlocks the studies themselves: titles, statuses, dates and featured-trial cross-links.