According to a story from pharmpro.com, the pharmaceutical company Abeona Therapeutics announced that it has been given Rare Pediatric Disease Designation for its ABO-202 program by the U.S. Food and Drug Administration (FDA). This program is in development for treating CLN1 disease, which is a form of Batten disease that begins very early in life when the patient is still an infant.
Batten disease is a genetic, fatal nervous system disease that normally begins in childhood. Symptoms include problems with eyesight, seizures, learning regression, loss of coordination, and behavioral changes. As the disease progresses, patients lose their sight, speech, and motor abilities. They also suffer mental impairment and worsening of prior symptoms. Genetic mutations are responsible for causing Batten disease, and different variants are categorized by genes are affected (CLN1 is the gene involved in CLN1 disease, for example). People with the CLN1 variant die around age five, which is much earlier in comparison to other forms of Batten disease. At this juncture, there are no treatments available that can stop the progression of the condition, though some may delay the worsening of symptoms. To learn more about Batten disease, click here.
This is not the first time that ABO-202 has been given a special designation, and has previously received orphan drug status. However, Rare Pediatric Disease Designation is generally issued less frequently. Drug candidates that receive this status most show potential to treat a condition that has no currently approved therapies. The designation also means that the FDA can give the company a prior review voucher if the treatment gains approval for the rare disease in question. The company is then free to use the voucher for any of its drug candidates in order to receive priority review. A drug with priority review with be evaluated by the FDA within six months as opposed to the typical standard of ten months.
