The FDA grants designations, such as the Rare Pediatric Disease and Orphan Drug designations, to aid in the drug development process. Recently, LEXEO Therapeutics has received these two designations for their treatment, LX2006. This adeno-associated virus (AAV)-mediated gene therapy is currently in the clinical trial stage and being evaluated as a treatment for Friedreich’s ataxia (FRDA).
About the Designations
Rare Pediatric Disease Designation
This designation is reserved for therapies in development for the treatment of rare and severe or life-threatening diseases that mainly impact patients under the age of 18. Rare is defined as affecting less than 200,000 people in the United States. Companies strive for this designation as it can come with certain benefits, such as a priority review voucher. For example, if LEXEO is also granted a biologics license application, they will qualify for a voucher that can then be traded, applied, or sold.
Orphan Drug Designation
Like the Rare Pediatric Disease designation, this designation is designated for therapies for diseases impacting fewer than 200,000 people within the country. It also comes with incentives, such as tax credits. You can learn more about these designations – and others – that impact the development of rare disease drugs here.
LEXEO is very excited to receive these designations, as it demonstrates the need and urgency for viable Friedreich’s ataxia treatments. They hope that future development goes well and are planning for a Phase I/II trial of LEX2006 for cardiomyopathy associated with FRDA. This trial is set to begin later this year.
About Friedreich’s Ataxia
Friedreich ataxia is a rare, hereditary disease that is characterized by movement and neurological symptoms. Affected individuals typically notice the effects around age 15, where they will experience difficulty moving and slurred speech. Symptoms will continue and may expand to problems with vision and hearing, cardiac arrhythmia, chest pain, fatigue, shortness of breath, foot abnormalities, scoliosis, and diabetes mellitus. A mutated FXN gene is responsible for this condition. This gene normally produces frataxin, which is necessary for proper mitochondrial function. When it is mutated, this process is interrupted and the mitochondria can no longer perform their functions. It is inherited in an autosomal recessive pattern. Treatment is symptomatic.
You can find the source article here.