Potential Gene Therapy for Heart Disease in FA Gets FDA Support

The U.S. Food and Drug Administration (FDA) has given rare pediatric disease and orphan drug designations to LX2006, Lexeo Therapeutics‘ investigational gene therapy for people with Friedreich’s ataxia (FA).

The designations each provide benefits meant to ease the therapy’s path through development.

Rare pediatric disease status is provided to medications designed to treat life-threatening illnesses affecting fewer than 200,000 people aged 18 years and younger in the United States. It qualifies the therapy for an accelerated review — typically six months, rather than a year — and the developer is given a voucher that can be exchanged for an accelerated review of a different treatment.

Orphan drug designation is reserved for investigational therapies targeting disorders affecting fewer than 200,000 people in the United States. It allows a developer to possibly qualify for tax credits, certain application fee waivers, and grants seven years of marketing exclusivity upon regulatory approval.

“Being granted both Rare Pediatric Disease and Orphan Drug designation shows the tremendous urgency for new, impactful therapeutic approaches such as LX2006 for people diagnosed with Friedreich’s ataxia,” R. Nolan Townsend, CEO of Lexeo Therapeutics, said in a press release.

LX2006 is a gene therapy intended to treat heart conditions associated with FA, which affect nearly all patients at some point in their lives and are a major cause of death.

The loss of frataxin in patients caused by a mutation in the FXN gene prevents cells from meeting their energy demands. As heart cells have some of the highest energy demands in the body, frataxin deficiency hits them particularly hard.

The gene therapy uses a modified and harmless virus, called an adeno-associated virus (AAV), to deliver a functional copy of the FXN gene to cells in the heart. It is designed as a potential one-time treatment, and is delivered via an intravenous (into-the-vein) injection.

Lexeo recently obtained the rights to LX2006 from its maker, Adverum Biotechnologies, and is completing preclinical studies to support a request to advance into clinical trials. The company plans to initiate a Phase 1/2 clinical trial for FA patients with disease-related cardiomyopathy later this year.

“It is critical that we advance new disease-modifying therapies with the potential to transform the lives of FA patients, and we look forward to continuing our collaboration with the FDA as we advance LX2006 through clinical development,” Townsend said.