AlzeCure Pharma AB (publ) (FN STO: ALZCUR), a biotech company that develops candidate drugs for diseases affecting the nervous system, focusing on Alzheimer's disease and pain, today announced that the European Medicines Agency (EMA) has granted the company's application for orphan drug status for the clinical pain medication ACD440 as treatment in erythromelalgia.
“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,” said Märta Segerdal, CMO at AlzeCure Pharma.
ACD440, the company's lead pain drug candidate within the Painless platform, has previously completed a positive Phase IIa clinical trial in patients with chronic peripheral neuropathic pain. The compound received orphan drug designation from the US Food and Drug Administration (FDA) in 2025. In addition to the grant of orphan drug designation, the company also received positive guidance from the FDA supporting the continued development of the compound in a Phase II/III trial in peripheral neuropathic pain for regulatory approval.
Erythromelalgia is a rare chronic disease that affects an average of just over 13 out of 100,000 people and is characterized by intense burning pain and severe redness of the skin. The disease most often occurs in the extremities such as the feet, hands, ears and nose, but can also occur in other parts of the body. The painful part of the body often swells and the skin becomes very hot. There is currently no approved treatment available for patients suffering from erythromelalgia, so the medical need is very great.
ACD440 is a first-in-class TRPV1 antagonist in clinical development as a novel topical local treatment for chronic peripheral neuropathic pain. The drug candidate, which was incorporated via a strategic in-licensing, originated in Big Pharma and is based on a strong scientific foundation, which was awarded a Nobel Prize in 2021. The substance is being developed as a gel for topical use, which keeps systemic exposure very low while maintaining high local concentrations of the substance to achieve maximum analgesic effect and over a long period of time.
”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. 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 prioritized 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*,” said Martin Jönsson, CEO of AlzeCure Pharma.
*) Althobaiti H et al, Disentangling the Cost of Orphan Drugs Marketed in the United States, Healthcare (Basel), 2023 Feb 13;11(4):558; https://pmc.ncbi.nlm.nih.gov/articles/PMC9957503/
