AtlasOther conditionsHemoglobinopathies and hemolytic anemias

Thalassemias

4Curable or preventable in mostAI-generated
Burden-over-time charts — how many people this disease affects and whether that number is rising or falling — are coming to this page. The treatability read, milestones, and live trial pipeline below are current.

Milestones

dot size = significance
199020002010202020251999 — Deferiprone approved (FDA)2005 — Deferasirox approved (FDA)2019 — Luspatercept approved (FDA)2019 — Betibeglogene autotemcel approved (FDA)2023 — Exagamglogene autotemcel approved (FDA)
1999: Drug approval — Deferiprone approved (FDA). 2005: Drug approval — Deferasirox approved (FDA). 2019: Drug approval — Luspatercept approved (FDA). 2019: Drug approval — Betibeglogene autotemcel approved (FDA). 2023: Drug approval — Exagamglogene autotemcel approved (FDA)

What's being tested now

2010: 1 trials started2014: 2 trials started2015: 1 trials started2017: 1 trials started2018: 3 trials started2019: 4 trials started2020: 4 trials started2021: 4 trials started2022: 11 trials started2023: 9 trials started2024: 26 trials started2025: 11 trials started2026: 13 trials started20102020202626

Trials started per year, stacked by phase (darker = later phase). Source: ClinicalTrials.gov via Clin2, which tracks studies recruiting in recent years — early years undercount.

See all 86 active Alpha-Thalassemia trials on Clin2 →

Related

Burden estimates: IHME Global Burden of Disease Study. Drug indications: ChEMBL (CC BY-SA). Trial data: ClinicalTrials.gov via Clin2. Velocity, acceleration, and turning points are computed by Clin2 — how this page is made. Treatability describes what medicine can do, not what is available everywhere. This page describes populations, not people, and is not medical advice.