OriginalSourceMedpagetodayLeanUnratedCredibilityUnratedPublished22:32 UTCSourceMedpagetodayLeanUnratedCredibilityUnratedPublished22:32 UTC
“Late-Stage Sickle Cell Disease Study Splits on Main Goals”
Mitapivat raised hemoglobin but did not reduce pain crises in sickle cell trial
Key points
- In the RISE UP phase III trial, 41% of mitapivat patients had a hemoglobin response vs 3% on placebo.
- However, annualized sickle cell pain crises did not differ significantly (2.6 vs 3.1; P=0.1213).
- Mitapivat showed improvements in hemolysis markers and reduced transfusion burden among responders.
- The trial enrolled 207 patients across 69 sites in 15 countries, with a 2:1 randomization to mitapivat or placebo.
- FDA review is ongoing, with a potential February next-year decision on sickle cell disease approval.
How it got here
Medpagetoday publishes the article.
concise reads it.