Roche has reported the initial analysis data from the Phase III HAVEN 7 clinical trial where its bispecific factor IXa-directed and factor X-directed antibody Hemlibra (emicizumab) offered benefit in infants with severe haemophilia A without factor VIII inhibitors.
These infants were previously untreated or minimally treated.
The single-arm, descriptive trial is designed to evaluate the safety, efficacy, pharmacodynamics and pharmacokinetics of subcutaneously administered Hemlibra in infants.
Findings showed that the treatment provided meaningful bleed control in babies aged up to 12 months and was also found to be well tolerated.
In the trial, 54.5% of trial subjects did not have any bleeding cases that needed treatment following Hemlibra dosing at a median follow-up period of 101.9 weeks.
Furthermore, no treated or untreated bleeds were reported in 16.4% of the participants at this period.
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No cases of spontaneous bleeds that needed treatment were observed with all treated bleeds caused by trauma.
New treatment-associated safety signals were not reported in the trial with no cases of treatment-related serious adverse events, intracranial haemorrhages or deaths observed.
Data from the trial was in line with positive interim analysis findings and those from prior Phase III HAVEN studies.
Hemlibra is created to combine factor IXa and factor X proteins linked to a natural coagulation cascade. This approach aids in reinstating the blood clotting process in haemophilia A patients.
Created by Chugai Pharmaceutical, the therapy is being jointly developed by Chugai, Roche and Genentech across the world.
Roche Global product development head and chief medical officer Levi Garraway said: “The results of HAVEN 7 provide additional confidence in the efficacy and safety profile of Hemlibra for babies with severe haemophilia A, and add to its extensive clinical and real-world evidence across all ages.
“Conducted in collaboration with the haemophilia A community, this trial reflects our ongoing commitment to listen and respond to the needs of those impacted by this condition, in hopes of advancing treatment standards even further.”
The latest development comes after the company reported positive data from the Phase III INAVO120 trial of inavolisib plus palbociclib (Ibrance) and fulvestrant in locally advanced or metastatic breast cancer patients.