PledPharma has filed an application for an Orphan Drug Designation (ODD) to the US FDA for the its drug candidate which should reduce liver injury by paracetamol overdose, Aladote.
The current standard of care for paracetamol poisoning (NAC) is effective if the patient seeks medical care within 8 hours of ingestion. Patients arriving later to the hospital, and for those with high paracetamol levels, there is a need for a more effective treatment. PledPharma noted that Aladote has shown good efficacy in relevant preclinical models, even in the time-window when N-acetylcysteine treatment is no longer is effective. Aladote is a first-in-class drug candidate developed to prevent the development of acute liver injury caused by paracetamol overdose.
Aladote is considered by PledPharma to fulfil the requirements for Orphan drug status in the US, i.e. less than 200,000 patients per year are affected from paracetamol overdose and the scientific rational as well as the clinical results from the recently completed proof-of-principle study indicate that Aladote has a potential in reducing liver injury for these patients, PledPharma said, expecting the FDA is expected to respond to the application in the first half of 2019.
Nicklas Westerholm, CEO, Pled Pharma AB, said: “The reason for an application is the benefits of an orphan drug status. If granted, we are likely to acquire in total less development costs and a shorter development lead time. Furthermore, we receive additional dedicated FDA support during the drug development and get seven years of market exclusivity following a market approval.”
Paracetamol is the most used drug in the world for the treatment of fever and pain, but also one of the most overdosed drugs – intentional or unintentional. Paracetamol overdose is also one of the most common method in intentional suicide attempts.