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Elixirgen Therapeutics Receives FDA Rare Pediatric Disease Designation for EXG-34217 in Dyskeratosis Congenita

• Elixirgen Therapeutics received FDA Rare Pediatric Disease Designation for EXG-34217, a treatment for dyskeratosis congenita and related telomere biology disorders (DC/TBD). • EXG-34217 consists of autologous CD34+ hematopoietic stem cells treated with EXG-001, encoding human ZSCAN4, using a Sendai virus vector. • The FDA's designation acknowledges the significant unmet need for DC/TBD therapies and the potential of EXG-34217 to address this rare condition. • Elixirgen is currently enrolling patients in a Phase 1/2 trial at Cincinnati Children’s Hospital Medical Center to evaluate EXG-34217.

Elixirgen Therapeutics, Inc. has been granted Rare Pediatric Disease Designation (RPDD) by the U.S. Food and Drug Administration (FDA) for EXG-34217, a therapy targeting dyskeratosis congenita and related telomere biology disorders (DC/TBD). The company is currently enrolling patients in its Phase 1/2 trial evaluating EXG-34217 in patients with telomere biology disorders at Cincinnati Children’s Hospital Medical Center.

EXG-34217: A Novel Approach to DC/TBD Treatment

EXG-34217 is composed of autologous CD34+ hematopoietic stem cells (HSCs) modified ex vivo with EXG-001. EXG-001 utilizes a non-integrating, non-transmissible, temperature-sensitive Sendai virus vector to introduce human ZSCAN4 into the cells. The ongoing Phase 1/2 trial (NCT04211714) aims to assess the safety and efficacy of this innovative approach.

Significance of Rare Pediatric Disease Designation

The FDA grants RPDD to therapies aimed at serious or life-threatening diseases affecting fewer than 200,000 people in the U.S., primarily those under 18 years of age. This designation can lead to a Priority Review Voucher (PRV) upon approval of the marketing application for EXG-34217, potentially accelerating the review of future applications.

Executive Perspective

"We’re pleased with the FDA’s decision to grant EXG-34217 RPDD for the treatment of DC/TBD, a rare group of diseases that result in abnormally short telomeres that can lead to bone marrow failure and other serious health conditions,” said Aki Ko, chief executive officer of Elixirgen Therapeutics. “The RPDD is a recognition of the significant unmet need in this space and the potential of EXG-34217, and we look forward to working with the FDA as we continue to advance this program."

About Dyskeratosis Congenita

Dyskeratosis congenita and related telomere biology disorders are a group of rare genetic diseases characterized by abnormally short telomeres, leading to bone marrow failure and other severe health complications. These disorders present a significant unmet medical need, making the development of effective therapies like EXG-34217 crucial.
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Reference News

[1]
Elixirgen Therapeutics Receives Rare Pediatric Disease Designation for EXG-34217 in ... - BioSpace
biospace.com · Sep 26, 2024

Elixirgen Therapeutics receives Rare Pediatric Disease Designation for EXG-34217, treating dyskeratosis congenita and te...

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