Potential Treatment for Friedreich’s Ataxia Earns Orphan Drug and Rare Pediatric Disease Designations

According to a story from GlobeNewswire, the gene therapy company LEXEO Therapeutics has recently announced that its investigational gene therapy LX2006 has been granted both Orphan Drug designation and Rare Pediatric Disease designation from the US Food and Drug Administration (FDA). This therapy is in development as a treatment for Friedreich’s ataxia, a rare genetic disease.

About Friedreich’s Ataxia

Friedreich’s ataxia (FRDA) is a neuromuscular genetic disorder which is characterized by abnormal gait due to the degeneration of nerve tissue in the spinal cord. The disorder is linked to a genetic mutation of the FXN gene. The degeneration of the nerves causes the spinal cord to become thinner and neurons begin to lose some of their myelin sheath, a fatty insulating layer that protects these cells and is necessary for normal function. Symptoms of Friedreich’s ataxia include muscle weakness affecting the limbs, heart disorders, poor coordination, diabetes, high foot arches, vision and hearing problems, scoliosis, and slurred speech. Symptoms begin to appear at 7-10 years and the disorder progresses slowly overall. Management strategies for the disorder may include ACE inhibitors, surgery, speech and rehabilitation therapy, and orthoses. Life expectancy is around 50 years. Heart problems are the leading cause of death. To learn more about Friedreich’s ataxia, click here.

About Rare Pediatric Disease Designation

Rare Pediatric Disease designation is reserved for therapies that have the potential to treat severe, life-threatening illnesses that mostly affect people who are no more than eighteen years old and are considered rare, meaning that they impact less than 200,000 people in the US. The developing company may be eligible for a priority review voucher if the drug gains approval. This voucher can be used on a future application or can be sold to another company.

About Orphan Drug Designation

Orphan Drug designation is typically reserved for drugs that are meant to treat a disease that is considered rare. To qualify, this drug must have the potential to fulfill a currently unmet medical need or offer potential advantages in safety or efficacy over currently available treatments. Orphan Drug designation confers several benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a seven-year period of market exclusivity if the drug gains approval.

About LX2006

LX2006 is classified as an IV administered adeno-associated virus (AAV) mediated gene therapy that encodes for the frataxin (FXN) gene. LEXEO plans to start a phase 1/2 clinical trial later this year.

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