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EMA Orphan Drug Designation for CLN5 Latte Disease

Therapy follows FDA orphan drug designation

The designation demonstrates continued progress in

Creation of the Batten Disease franchise

NEW YORK – (BUSINESS WIRE) – Neurogene Inc., a company founded to bring life-changing genetic drugs to patients and families affected by rare neurological diseases, today announced that the European Medicines Agency (EMA) has granted orphan drug designation (ODD) to its adeno-associated virus (AAV) encoding a codon-optimized human CLN5 transgene (hCLN5) for patients diagnosed with CLN5, a subtype of Batten disease .

“The signs and symptoms of CLN5 disease include vision loss, cognitive and motor impairment, and seizures. The disease often progresses rapidly within a few years of symptom onset, leading to premature death. With no treatment options available, the result is devastating for children and their families, ”said Angela Schulz, MD, Ph.D., Children’s Hospital, NCL Specialty Clinic, International Center for Lysosomal Storage Disorders , University Medical Center Hamburg-Eppendorf.

“This regulatory designation is an important step in addressing the urgent and unmet needs of families and patients living with CLN5, and follows the orphan drug designation previously granted in 2020 by the US Food and Drug Administration (FDA). United for this same gene therapy candidate, ”said Rachel McMinn, Ph.D., Founder and CEO of Neurogene. “Neurogene is working with Batten disease experts, regulatory authorities and caregivers to provide safe and effective gene therapy for this disease as quickly as possible. ”

About the orphan drug designation

The EMA’s Orphan Designation is designed to encourage the development of new treatments for life-threatening or chronic debilitating conditions that are rare (affecting no more than

five in 10,000 people in the European Union) and currently have no approved treatment options available. Drugs that meet the EMA’s orphan designation criteria are eligible for several incentives, including 10 years of market exclusivity, protocol support, and potentially reduced fees for regulatory activities. Applications for orphan designation are examined by the EMA Committee for Orphan Medicinal Products (COMP), using the committee’s network of experts.

About CLN5, a subtype of Batten disease

Batten’s disease belongs to the lysosomal storage disorder (LSD) family. LSDs are progressive, multisystem, inherited metabolic disorders associated with premature death. Batten disease, also known as neuronal ceroid lipofuscinosis (NCL), is a family of rare neurodegenerative diseases caused by pathogenic changes in one of a series of genes that cause toxic deposits to build up in several organ systems. , including the brain, eyes, skin and others. CLN5 is a rare, late childhood subtype, with pediatric onset and rapidly progressive, caused by a variant of the CLN5 gene. It is characterized by loss of vision, seizures and a gradual decline in intellectual and motor skills from childhood. The diagnosis of the disease is confirmed by genetic tests. Neurogene has developed an educational website,, to support patients, families and caregivers looking for more information, resources and genetic testing.

About genetic testing

Neurogene is committed to reducing the barriers to obtaining a genetic diagnosis for patients and has partnered with Invitae to co-sponsor two genetic testing programs in the United States and Canada. Healthcare providers can order the Detect Lysosomal Overload Disease panel free of charge for patients with suspected lysosomal overload disease. In addition, the Invitae Epilepsy panel is available free of charge to any child under the age of eight who has had an unprovoked seizure. The Detect Lysosomal Overload Disease panel is available free of charge for patients with suspected lysosomal overload disease. CLN5 and other genetic variants of Batten disease are available on both genetic testing panels. Visit for more details.

About Neurogene Inc.

Neurogene Inc. is focused on the development of genetic drugs that change the lives of patients and their families affected by rare and devastating neurological diseases. We work in partnership with leading academic researchers, patient advocacy organizations and caregivers to provide patients with therapies that address the underlying genetic cause of a wide range of neurological diseases for which no options are available. effective treatment currently exists. Our main programs use adeno-associated virus (AAV) gene therapy technology to deliver a normal gene to patients with a dysfunctional gene. Neurogene is also developing new gene therapy technologies to advance treatments for complex neurological diseases that conventional gene therapy cannot successfully treat. For more information, visit


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