FDA Acts to Speed Development of Herantis Pharma’s Potential Treatment for ALS

Herantis Pharma announced that a pipeline therapy known as cerebral dopamine neurotrophic factor (CDNF) has been granted Orphan Drug Status by the U.S. Food and Drug Administration (FDA) to speed its development and testing as a potential treatment for amyotrophic lateral sclerosis (ALS) and Parkinson’s disease.

Cerebral dopamine neurotrophic factor is a neuroprotective and neurotrophic protein that has shown potential for the regeneration and protection of dopamine-generating neurons, cells involved in Parkinson’s pathology. It was developed based on research at the Institute of Biotechnology at the University of Helsinki, lead by professor Mart Saarma, and Herantis holds a patent on the factor.

The drug has been shown to be effective in preclinical models of Parkinson’s disease (PD), and several studies, including chronic toxicology studies, have shown the safety of CDNF administration. Early research in an ALS disease model indicated that the drug significantly increased survival and reduced disease symptoms.

Herantis is now preparing for clinical testing of CDNF as a Parkinson’s treatment, and has a preclinical program underway for the treatment of ALS.

“This positive decision by the FDA greatly encourages us in the planning of a clinical development program of CDNF for the treatment of ALS in addition to the treatment of Parkinson’s disease,” Pekka Simula, the company’s CEO, said in a press release.

The FDA grants orphan drug status to drug candidates aiming to treat rare diseases or conditions that affect less than 200,000 individuals in the U.S., or for those affecting more than 200,000 people but where the costs of developing and marketing a treatment are not expected to be recovered. For orphan drug status to be granted, a company must establish a medically plausible basis for the in-development drug to be effective.

This designation will allow the company to obtain several benefits and development advantages for this drug and specific indication, such as tax credits for qualified clinical testing, exemptions from certain costs under the Prescription Drug User Fee Act (PDUFA), and seven-year marketing exclusivity should the drug be approved. CDNF received a similar designation from the European equivalent of the FDA, the European Medicines Agency, earlier this year.

“We are very pleased with the world’s leading medicines agencies reaching independently the same conclusion on the strength of the preclinical evidence,” said Dr. Henri Huttunen, chief scientific officer of Herantis.