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Orphan Drug Designation for lead compound CAB1803 for treatment of pantothenate kinase-associated neurodegeneration (PKAN)


European Medicines Agency (EMA) granted orphan medicinal product designation to its lead compound CAB1803 for Pantothenate-kinase-associated neurodegeneration (PKAN)
PKAN is an inherited disease, caused by a mutation in the gene responsible for producing an enzyme called pantothenate kinase-2, resulting in gradual brain damage and often leading to the very premature death of affected patients.

EMA grants Orphan Drug designation to medicines intended to treat, prevent or diagnose life-threatening and debilitating diseases, with a prevalence no greater than five in 10,000 in the European Union (EU). At the time of designation, PKAN affected less than 0.1 in 10,000 people in the EU. EMA Orphan designation by EMA is granted based on a detailed review of available current scientific evidence and leads to a number of incentives, including scientific, administrative and procedural assistance by EMA as well as reduced fees and extended market exclusivity.

Patients with the PKAN cannot produce a functioning pantothenate kinase-2 enzyme, resulting in degenerative brain damage. Symptoms of PKAN usually appear in patients during childhood, and include severe dystonia (painful muscle spasms) and Parkinson-like effects such as rigidity and slow movement, as well as loss of sight and impaired swallowing. PKAN is a debilitating and life-threatening disease due to complications of severe muscle spasms that can affect swallowing and lead to pneumonia (infection of the lungs) and malnutrition, frequently resulting in the death of patients as early as in their second decade of life. There is currently no treatment that could prevent or delay the course of this disease.

“Our proprietary compound CAB1803 has the potential to be an important therapy for patients with the rare disease PKAN. This EMA status represents an important milestone for us, reinforcing our commitment to further develop this compound and provide this treatment option to patients as soon as possible” said dr. Enej Kuscer, CEO of Acies Bio. 



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