15/07/2025
AlzeCure’s pain project ACD440 granted orphan drug status in the US by the FDA
“We are of course very happy about this good news. The granting of orphan drug status for ACD440, combined with the positive interaction and response we have received from the FDA regarding our preIND application for a pivotal phase II/III study, gives us increased opportunities to offer these very severely affected patients an effective treatment,” 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 chronic patients with peripheral neuropathic pain.
Ahead of the next stage of clinical development, AlzeCure applied for a preIND meeting with the FDA regarding the development of ACD440 as a drug for the treatment of the rare disease erythromelalgia.
In addition to the granting of orphan drug status, the company also recently received positive guidance from the FDA supporting the continued development of the compound for a Phase II/III study 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.
”Orphan drug status provides a number of very important benefits, with the possibility of a faster path to approval through processes such as accelerated or conditional approval, as well as priority review. In addition, stronger and extended market exclusivity is provided, which strengthens our competitive advantages and the conditions for out-licensing this important and promising project. In addition, the price of orphan drugs in the US is very high, with a median price of around SEK 2 million for an annual treatment*,” said Martin Jönsson, CEO of AlzeCure Pharma.
https://www.alzecurepharma.se/en/alzecures-pain-project-acd440-granted-orphan-drug-status-in-the-us-by-the-fda/
