Phase 3 Gene Therapy Study on Hemophilia B Launched by Pfizer
July 23, 2018
Source: MedEdge
1,212
Pfizer has initiated its new work on a phase 3 study of an investigational gene therapy designed to treat hemophilia B.
Along with Spark Therapeutics, Pfizer launched a phase 3 lead-in study at several centers to assess the efficacy and safety of present factor IX prophylaxis replacement therapy in the regular care provisions.
The efficacy data that will be generated from the lead-in study will become the within-subject control group for patients who sign up in the subsequent part of the phase 3 study. This study will then assess fidanacogene elaparvovec (formerly SPK-9001) for the treatment of hemophilia B.
As per the data from an ongoing phase 1/2 trial of fidanacogene elaparvovec released by Pfizer and Spark Therapeutics in May 2018, all 15 participants with hemophilia B could discontinue regular infusion of factor IX concentrates without grave unfavorable incidents. Fidanacogene elaparvovec, a vector, contains a bioengineered adeno-associated virus capsid and a human coagulation factor IX gene which is highly active.
By Ddu
Copyright©2024 Ddu. All rights reserved.
Read more on
- 【EXPERT Q&A】Is it mandatory to conduct clinical trials for drug registration in India? August 9, 2023
- PFIZER AND BIONTECH ANNOUNCE VACCINE CANDIDATE AGAINST COVID-19 ACHIEVED SUCCESS IN FIRST INTERIM ANALYSIS FROM PHASE 3 STUDY November 10, 2020
- Pfizer Drug Lowers Mortality Risk by 30 Percent in a Rare Cardiac Condition August 29, 2018
- Experimental Ebola Vaccines used During Ebola Outbreak in Congo August 14, 2018
- Hemophilia A Gene Therapy by Spark Therapeutics Needs Phase 3 Trial August 9, 2018
your submission has already been received.
OK
Subscribe
Please enter a valid Email address！
Submit
The most relevant industry news & insight will be sent to you every two weeks.
