AlzeCure Pharma has announced that the European Medicines Agency has granted orphan drug status to ACD440, its clinical‑stage pain treatment being developed for erythromelalgia.
The company, which focuses on diseases affecting the nervous system including Alzheimer’s disease and pain, said the designation strengthens the prospects for advancing ACD440 as a potential therapy for patients with this rare and debilitating condition.
Märta Segerdal, CMO at AlzeCure Pharma, said: “This positive news from the EMA regarding orphan drug status for ACD440, combined with the positive interaction and message we have received from the FDA, gives us increased opportunities to offer an effective treatment to these very severely affected patients.”
ACD440 is the lead pain candidate within the company’s Painless platform and has already completed a positive phase IIa trial in chronic peripheral neuropathic pain.
The compound received orphan drug designation from the US Food and Drug Administration in 2025, alongside supportive guidance for continued development in a phase II/III trial aimed at regulatory approval.
Erythromelalgia affects just over 13 in 100,000 people and is characterised by intense burning pain, severe redness and heat in affected areas, most commonly the extremities.
There is currently no approved treatment, leaving patients with significant unmet medical need.
ACD440 is a first‑in‑class TRPV1 antagonist being developed as a topical gel for chronic peripheral neuropathic pain.
The approach aims to maintain low systemic exposure while achieving high local concentrations for sustained analgesic effect.
Martin Jönsson, CEO of AlzeCure Pharma, said: “The fact that we have now been granted orphan drug status in the EU also, strengthens our competitive advantages and the conditions for out-licensing this important and promising project.”
He added: “Orphan drug status entails a number of very important advantages, with the possibility of obtaining a faster path to approval through processes, such as accelerated or conditional approval, and prioritised review. In addition, a stronger and extended market exclusivity is provided during a 10 year period, which strengthens our competitive advantages. Also, the price of orphan drugs in general is very high, e.g. the USA with a median price of around SEK 2 million for an annual treatment.”
