AudioCure Pharma’s AC102 receives EMA orphan drug designation for the treatment of sudden sensorineural hearing loss (SSNHL)

• The EMA’s Committee for Orphan Medicinal Products has granted orphan drug designation to AC102, concluding that AudioCure’s lead compound may be of significant benefit for patients suffering with the rare and chronically debilitating disease of sudden sensorineural hearing loss (SSNHL).
• Comprehensive preclinical in vivo data provide strong evidence of efficacy in a model of acute hearing loss.

BERLIN--(BUSINESS WIRE)--The German pharmaceutical company AudioCure Pharma GmbH announced today that their first in class molecule AC102 has received orphan drug designation by the European Medicines Agency (EMA) for the treatment of sudden sensorineural hearing loss (SSNHL). The EMA exclusively grants orphan drug designation to medicinal products intended to treat a severely debilitating condition of low prevalence when there is sufficient evidence that the drug has the potential to bring significant benefit to affected patients.

Dr. Reimar Schlingensiepen, CEO of AudioCure commented: “The orphan drug designation is a major milestone in the development program of AC102, enabling a more efficient and cost-effective regulatory pathway, and is therefore an important step in pursuing our goal of developing treatments for hearing disorders with significant unmet medical needs. Preparations for our Phase I clinical trial with AC102 are well underway and we look forward to future interactions with the EMA in our effort to develop our lead compound to the market.”

SSNHL is a form of hearing loss with a rapid onset. In most cases the cause is unknown. The prevalence of the disorder is considered rare with 4 out of 10,000 people affected. Prof. Dr. Stefan Plontke, Head of Ear, Nose and Throat Clinic at Martin Luther University, Halle, Germany explained: “Hearing loss affects peoples’ quality of life much more than one would expect. Even mild levels of hearing loss can increase the long-term risk of cognitive decline and dementia. Nevertheless, there are no approved drugs on the market for acute hearing loss. As a medical doctor I am in urgent need of a treatment with a proven efficacy and safety profile that I can offer to my patients.”

AC102´s mode of action is unique in addressing, in parallel, three of the pathological processes that lead to hearing loss: it protects cells and restores functionality of the inner ear not only by preventing cell death of outer hair cells but also by enabling the repair processes of inner hair cells and reducing inflammation.

For further information, please visit https://www.audiocure.com/newsroom/news-in-brief/