Stockholm, Sweden, July 13, 2022. Egetis Therapeutics AB (publ) (Nasdaq Stockholm: EGTX) today announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending Aladote (calmangafodipir) for designation as orphan medicinal product for the Prevention of Acute Liver Failure, a life-threatening condition.
Aladote is a first-in-class drug candidate developed for the treatment of paracetamol overdose in combination with N-acetylcysteine (NAC) to prevent acute liver failure. A proof-of-principle study has been successfully completed and the design of the upcoming pivotal Phase IIb/III study with the purpose of applying for market approval in the US and Europe for Aladote has been finalized after interactions with the US Food and Drug Administration (FDA), EMA and the UK Medicines and Healthcare products Regulatory agency (MHRA). Egetis expects to start this pivotal study for Aladote in 2022.
The positive opinion adopted by the EMA on the application for orphan designation for the Prevention of Acute Liver Failure follows the Orphan Drug Designation for the Treatment of Acetaminophen Overdose granted by the US FDA in 2019.
Nicklas Westerholm, CEO, Egetis Therapeutics, commented: “We are delighted to receive a positive opinion for orphan designation from EMA for Aladote for the prevention of acute liver failure. There is a substantial medical need for additional treatment options for the approximately 25% of patients presenting to hospital after eight hours of paracetamol poisoning. These patients are at increased risk of Acute Liver Failure. We look forward to initiating our pivotal Phase IIb/III study with Aladote later in 2022.”
N-acetylcysteine (NAC) is the current standard of care antidote for paracetamol poisoning. It is most effective if given within eight hours of the overdose. Patients arriving later to the hospital, and for those with a severe overdose, there is a need for more efficacious treatment options. The scientific rationale as well as clinical results from a completed proof-of-principle study indicate that Aladote in combination with NAC has the potential to reduce liver damage in the specified patient population.
In the EU, orphan drug status is given to products that treat, prevent, or diagnose a disease which is life-threatening or chronically debilitating and affects less than 5 in 10,000 people across the EU. Sponsors who obtain orphan designation in the EU benefit from protocol assistance, a type of scientific advice specific for designated orphan medicines, waivers or reductions of certain fees as well as a ten-year market exclusivity once the medicine is on the market. The European Commission is responsible for granting the orphan designation based on the positive opinion from EMA. For more information about orphan designation in the EU, please see www.ema.europa.eu/en/human-regulatory/overview/orphan-designation-overview
