STOCKHOLM, SE / ACCESS Newswire / July 15, 2025 / AlzeCure Pharma (STO:ALZCUR) (FRA:AC6) AlzeCure Pharma AB (publ) (FN STO:ALZCUR), a pharmaceutical company that develops candidate drugs for diseases affecting the nervous system, focusing on Alzheimer's disease and pain, today announced that the US Food and Drug Administration (FDA) has granted the company's application for orphan drug status for the clinical pain candidate drug ACD440.
"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.
* 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/
For more information, please contact
Martin Jönsson, CEO
Tel: +46 707 86 94 43
martin.jonsson@alzecurepharma.com
About AlzeCure Pharma AB (publ)
AlzeCure ® is a Swedish pharmaceutical company that develops new innovative drug therapies for the treatment of severe diseases and conditions that affect the central nervous system, such as Alzheimer's disease and pain - indications for which currently available treatment is very limited. The company is listed on Nasdaq First North Premier Growth Market and is developing several parallel drug candidates based on three research platforms: NeuroRestore ® , Alzstatin ® and Painless.
NeuroRestore consists of two symptomatic drug candidates where the unique mechanism of action allows for multiple indications, including Alzheimer's disease, as well as cognitive disorders associated with traumatic brain injury, sleep apnea and Parkinson's disease and is being prepared for phase 2. The Alzstatin platform focuses on developing disease-modifying and preventive drug candidates for early treatment of Alzheimer's disease. Painless is the company's research platform in the field of pain and contains two projects: ACD440, which is a drug candidate in the clinical development phase for the treatment of neuropathic pain with positive phase 2 results, and TrkA-NAM, which targets severe pain in conditions such as osteoarthritis. AlzeCure aims to pursue its own projects through preclinical research and development through an early clinical phase, and is continually working on business development to find suitable outlicensing solutions with other pharmaceutical companies.
FNCA Sweden AB is the company's Certified Adviser. For more information, please visit www.alzecurepharma.se .
About erythromelalgia
Erythromelalgia is a rare condition that affects just over 13 in 100,000 people and causes burning pain, redness, warmth and swelling, most often in the feet or hands. The symptoms are worsened by heat and relieved by cold. The disease can be primary (inherited) or secondary to other conditions, as part of autoimmune diseases, as a side effect of certain medications, or an effect of certain blood disorders (e.g. polycythemia vera or essential thrombocythemia). The cause is believed to be a combination of disturbances in the nervous system's pain signaling and the function of the thinnest blood vessels. Treatment focuses on relieving symptoms, often with cooling, pain medications and sometimes drugs that affect blood flow or nerve signals.
Erythromelalgia is a very painful condition characterized by burning pain, which occurs in sudden attacks triggered by heat, either in the environment or locally. These attacks can last for several hours, and recur from a few times a week to several times a day. Patients often describe the pain as if the skin is "burning".
Cooling the painful area is the most effective relief, while despite the frequent trials of many drugs, there is no established effective treatment. Frequent or prolonged cooling as a treatment often leads to severe frostbite and infection, which can be life-threatening.
The disease greatly affects quality of life. Walking, standing or just being in warm environments or wearing shoes that get hot can be unbearable. Many have difficulty managing their jobs, experience sleep problems and suffer from isolation.
About orphan drugs
Orphan drugs are used to treat, prevent or diagnose a rare disease - that is, a disease that affects no more than 5 in 10,000 people in the EU. In the US, the limit is a maximum of 200,000 people with the disease.
To encourage the development of such drugs, authorities offer special benefits, e.g.:
Prolonged market exclusivity
Scientific advice from pharmaceutical authorities
Reduced fees for approval applications
Orphan drugs are important because they make it possible to treat patients with rare and often serious diseases who might otherwise not have received treatment. Furthermore, the price of orphan drugs is often significantly higher than that of other medicines.
This information is information that AlzeCure Pharma is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact persons set out above, at 2025-07-15 15:37 CEST.
Image Attachments:
Martin Jönsson O Märta Segerdahl AlzeCure Pharma
Attachments
AlzeCure's pain project ACD440 granted orphan drug status in the US by the FDA
SOURCE: AlzeCure Pharma
View the original press release on ACCESS Newswire:
https://www.accessnewswire.com/newsroom/en/healthcare-and-pharmaceutical/alzecures-pain-project-acd440-granted-orphan-drug-status-in-the-us-by-1048862
