Be Biopharma’s engineered B Cell medicine aims to transform haemophilia B treatment with FDA orphan drug status. Credit: Shidlovski / Shutterstock.

The US Food and Drug Administration (FDA) has granted orphan drug designation to Be Biopharma’s (Be Bio) BE-101 for the treatment of haemophilia B.

A new engineered B Cell medicine, BE-101 is designed to deliver the human FIX gene into primary human B cells, enabling the expression of active FIX to manage haemophilia B.

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BE-101 aids in maintaining therapeutic FIX activity levels with a single infusion, offering the possibility of reduced dosing frequency compared to current FIX replacement therapies.

This could significantly alleviate the current treatment burden and potentially decrease annual bleeding rates and FIX usage.

The company is preparing to launch a Phase I/II BeCoMe-9 study of BE-101 in adults with severe or moderately severe haemophilia B in the second half of 2024.

Orphan drug status is awarded to drugs or biologics that target rare ailments affecting fewer than 200,000 individuals in the US.

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It confers benefits such as seven years of exclusive marketing rights post-approval, exemption from FDA user fees and tax credits for clinical trial costs.

Established in October 2020, Be Bio has received backing from investors including Alta Partners, ARCH Venture Partners, Atlas Venture, Seattle Children’s Research Institute and Takeda Ventures.

Be Bio CEO Joanne Smith-Farrell stated: “Despite recent advances, patients with haemophilia B still suffer from bleeding events, joint damage and chronic pain.

“BE-101 has the potential to be the first and only Factor IX replacement therapy that is extremely durable, re-dosable and titratable, representing an opportunity to dramatically improve the treatment paradigm in haemophilia B. The orphan drug designation reinforces the potential of this therapy and underscores the need for improved therapeutic options for these patients.”