According to a story from pm360online.com, the company PledPharma recently announced that its drug candidate Aladote has completed its first safety evaluation with a positive result. Aladote is in development for the treatment of paracetamol poisoning. More study is ongoing that will test the drug’s safety when used in combination with N-acetylcysteine, which is currently the most common standard of care for paracetamol poisoning.
About Paracetamol Poisoning
Paracetamol poisoning, also known as acetaminophen poisoning, is the result of excessive use of the pain medication paracetamol. Paracetamol is widely used for the treatment of mild to moderate pain and fever; the substance can be found in many over the counter medications, such as cough syrup and Tylenol. Though generally considered safe, a dose of ten grams or more in a 24 hour period is likely to cause harm, and the recommended daily dosage is no more than four grams. Paracetamol poisoning can be lethal, and there are cases of people intentionally using at in suicide attempts. Symptoms include sweating, pale skin, nausea, vomiting, abdominal pain, kidney failure, and liver damage/failure. If patients survive the, kidney and liver function can normalize in a couple of weeks. Treatment includes gastric decontamination, liver transplant, and N-acetylcysteine. To learn more about paracetamol poisoning, click here.
Aladote offers a significant advantage over N-acetylcysteine. The primary disadvantage of N-acetylcysteine is that it only effective in stopping liver failure if it is introduced within eight hours after the patient took paracetamol. This is a problem, as some patients may not develop major symptoms until much later. After this period, the events that initiate failure of the liver have already started. However, the data suggests that Aladote can still prevent liver failure beyond the eight hour window.
Orphan Drug Designation
Upon the completion of the study and the release of its data, PledPharma intends to apply for Orphan Drug Designation for Aladote in the US. Orphan Drug Designation would provide benefits to the development cycle of the drug. It is reserved for investigational drugs that are intended to treat rare diseases, which is defined as any disease that affects 200,000 people or less in the country. To qualify, the therapy must either address a currently unmet medical need or display significant advantages over currently available treatments.