Pfizer has begun a pivotal Phase lll trial to evaluate the efficacy and safety of fidanacogene elaparvovec therapy for the treatment of haemophilia B.

The open-label, multi-centre, lead-in study aims to enrol around 110 adult male patients.

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

The trial’s interventional portion is expected to enrol patients who have completed at least six months in the lead-in portion of the study.

Primary objectives of the trial are annualised bleeding rate (ABR), incidence of serious adverse events, and events of special interest (ESI).

The trial’s secondary objectives include AIR, dose and total factor consumption, and number of bleeding events, including spontaneous and/or traumatic.

It is the result of a licence agreement reached between Pfizer and Spark Therapeutics, developer of fidanacogene elaparvovec, in May this year.

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.
“The current data suggests immense promise for the use of this potential one-time treatment option.”

The agreement has allowed Pfizer to assume sole responsibility for all the subsequent pivotal studies, regulatory activities, manufacturing and global commercialisation of any products resulting from fidanacogene elaparvovec.

Pfizer Global Product Development Rare Disease senior vice-president and chief development officer Brenda Cooperstone said: “With the lead-in study now open and actively recruiting patients, we are excited to begin our Phase lll programme evaluating fidanacogene elaparvovec for the treatment of haemophilia B.

“The current data suggests immense promise for the use of this potential one-time treatment option.”

Almost exclusively found in males, haemophilia is a rare genetic bleeding disorder that results in the blood to take a long time to clot because of a deficiency in one of several blood clotting factors.