Herantis Pharma announced that it has received a positive opinion from the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) on its application for orphan drug designation for CDNF as a potential treatment for amyotrophic lateral sclerosis (ALS).
A positive opinion is the first step toward orphan drug designation which, in Europe, is issued by the European Commission.
“This positive opinion underlines two things,” Dr. Henri Huttunen, chief scientific officer at Herantis, said in a press release. “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.”
CDNF is short for Cerebral Dopamine Neurotrophic Factor and is a protein naturally occurring in the brain. Herantis has patented the externally produced factor, which has neuroprotective and growth promoting properties. The drug was developed based on research by Professor Mart Saarma from the Institute of Biotechnology at the University of Helsinki.
The drug has shown good results in animal models of Parkinson’s disease, and Herantis is currently preparing a first clinical study of CDNF in Parkinson’s patients.
CDNF’s development for ALS is still in preclinical stages. The Committee’s opinion was a result of positive data from early ALS studies showing that the drug can both increase survival and reduce symptoms in animal models of the disease.
“Even though an orphan indication, ALS is a major societal challenge,” said Pekka Simula, Herantis’ CEO. “140,000 new cases are diagnosed every year and the financial burden of motor neuron 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 is reserved for drugs intended to treat rare diseases, defined in Europe as affecting less than 5 in every 10,000 people. The designation does not allow less rigid clinical testing, but it does make the drug development process swifter by providing benefits, fee waivers, and scientific advice from the European Medicines Agency.