Giroctocogene fitelparvovec is being developed as part of an agreement between Sangamo and Pfizer for the global development of gene therapies for hemophilia A. Credit: Coolcaesar.

Pfizer and Sangamo Therapeutics have reported positive follow-up data from the Phase I/II Alta study of giroctocogene fitelparvovec (PF-07055480) to treat patients with severe hemophilia A.

The investigational gene therapy Giroctocogene fitelparvovec consists of a recombinant adeno-associated virus serotype 6 vector (AAV6) encoding the complementary deoxyribonucleic acid for B domain deleted human FVIII.

Go deeper with GlobalData

Premium Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

The open-label, dose-ranging, multi-centre Alta clinical trial has been designed to assess the safety and tolerability of the therapy in severe hemophilia A patients.

Across four dose cohorts, the mean age of the 11 patients assessed is 30 years and all are male.

All five patients with severe hemophilia A were given the 3e13 vg/kg dose and they showed sustained factor VIII (FVIII) activity levels, with a median of 64.2% via chromogenic assay.

No patients experienced bleeding events or required FVIII infusions during the trial.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

Pfizer Rare Disease Research Unit senior vice-president and chief scientific officer Seng Cheng said: “We are excited that these data affirm previous findings from this Phase I/II study, and that all five patients have sustained levels of factor VIII activity with no bleeding events or use of factor replacement therapy.

“The Phase III lead in study is ongoing, and we look forward to dosing patients with this investigational gene therapy in the pivotal Phase III trial later this year.”

Giroctocogene fitelparvovec received orphan drug, fast track, and regenerative medicine advanced therapy (RMAT) designations from the US Food and Drug Administration and Orphan Medicinal Product designation from the European Medicines Agency.

The therapy is being developed as part of a collaboration agreement between Sangamo and Pfizer for the global development and commercialisation of gene therapies for hemophilia A.

Sangamo chief medical officer Bettina Cockroft said: “These follow-up data indicate that treatment with giroctocogene fitelparvovec resulted in sustained factor levels up to 14 months following treatment and suggests the potential of this investigational gene therapy to alleviate the treatment burden of current hemophilia disease management.”