Neurogene Announces EMA Grants Orphan Drug Designation for Aspartylglucosaminuria Gene Therapy

Neurogene Inc., a company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, announces that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its adeno-associated virus vector (AAV) with engineered transgene encoding the human AGA gene for patients diagnosed with aspartylglucosaminuria. Aspartylglucosaminuria (AGU) is an inherited rare, progressively debilitating neurodegenerative lysosomal storage disorder. Currently there are no approved treatments to slow or cure AGU in patients living with this disease.

“Due to the lack of approved treatment options and the severity of symptoms, the prognosis and quality of life for patients and families affected by AGU can often be very poor,” said Minna Laine, M.D., Ph.D., Child Neurologist, Helsinki University Hospital, Helsinki, Finland. “The EMA granting Orphan Drug Designation for a potential gene therapy for AGU represents promise while underscoring the urgent, unmet medical need for patients around the world diagnosed with this condition.”

The AGU Orphan Drug Designation represents Neurogene’s third ODD for a rare neurological disease. Earlier this year the U.S. Food and Drug Administration (FDA) granted Neurogene ODD for gene therapies addressing CLN5 and CLN7, two subtypes of Batten disease which are also neurodegenerative and have no available treatment options to slow or halt the disease.

“Gaining this important regulatory milestone designation means we are one step closer to moving the first AGU treatment option into the clinic,” said Rachel McMinn, Ph.D., Neurogene’s Founder and Chief Executive Officer. “Neurogene is collaborating with regulatory authorities, families and patients to make a safe and effective gene therapy for AGU available as quickly as possible.”