Herantis Pharma secures European orphan designation of CDNF to treat ALS

"This positive opinion underlines two things", explains Dr. Henri Huttunen, Chief Scientific Officer of Herantis.

"First, there is a dire need for novel treatments for ALS, which is a truly devastating disease. Second, based on scientific data we have justified the assumption that CDNF will be of significant benefit to ALS patients. While years of hard work remains, this is an important milestone for us developing a novel treatment for ALS."

ALS is caused by the progressive loss of neurons that control voluntary muscles. This leads to stiffness, weakness, difficulty in speaking, swallowing, and eventually breathing. Most patients die from respiratory failure. The underlying cause of the disease can be genetic but in most cases remains unknown. No efficacious treatments are known.

"Even though an orphan indication, ALS is a major societal challenge", reminds Pekka Simula, Herantis’ CEO.

"140,000 new cases are diagnosed every year and the financial burden of motoneuron diseases in Europe is about €8 billion every year. 140,000 people a year are basically given a slow death sentence until we develop effective treatments. We will do our best to make CDNF available in the future as a new treatment option for ALS patients."

Orphan drug designation can be provided in the European Union to drugs intended to treat diseases or disorders affecting no more than five persons per 10,000. In addition at least early preclinical data are required, which suggest that the drug could provide patients with benefits that improve over currently available therapies.

Orphan drug designation does not exempt the company from clinical studies for establishing the safety and efficacy of the drug. The designation however provides benefits and incentives such as fee waivers, scientific advice, and an extended marketing exclusivity period against competition.