Pfizer launches phase III of gene therapy for haemophilia B

Pfizer has launched a late-stage trial to assess the efficacy and safety of fidanacogene elaparvovec, a research gene therapy, to treat haemophilia B.
third phase of the import study began after Spark's Type B haemophilia program was transferred to the pharmaceutical giant.
the trial will assess the effectiveness and safety of current factor IX preventive replacement therapy in routine care for type B haemophilia. The resulting data will be used in the in-patient control group of patients in the next Phase III study, and the fidanacogene elaparvovec used to treat the condition will be evaluated.
part of this critical Phase III study will include patients who have been in the study for at least six months, Pfizer said.
"With the introduction of research and the active recruitment of patients, we are excited to begin our phase III program to evaluate the treatment of type B haemophilia at fidanacogene elaparvovec," said Brenda Cooperstone, Senior Vice President and Chief Development Officer, Global Product Development Rare Diseases, Pfizer.
" current data show that this potential one-off treatment is highly promising. We look forward to the opportunity to continue spark therapy for people with type B haemophilia to make progress. "
haemophilia is a rare hereditary haemorrhagic disease that takes a long time to clot due to a lack of one of the many coagulation factors. People with haemophilia are at risk of excessive and repeated bleeding after moderate injuries, which can be life-threatening. (This web article)