MP-004 awarded Orphan Drug designation by the FDA

Miramoon Pharma, a biotechnology company focused on developing innovative therapies for rare diseases, has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to MP-004, a novel compound for the treatment of retinitis pigmentosa (RP).

RP is a group of inherited eye disorders that cause progressive vision loss and blindness. It affects about 1 in 4,000 people worldwide and has no approved treatment for most patients. Miramoon’s MP-004 compound is a small molecule that modulates the activity of leaky RyR calcium receptors. By restoring the channel function, MP-004 aims to prevent or slow down the degeneration of photoreceptors, the cells that capture light and enable vision.

Orphan Drug Designation is a special status granted by the FDA to drugs that are intended to treat rare diseases or conditions that affect less than 200,000 people in the U.S. The designation provides certain benefits to the sponsor, such as tax credits, market exclusivity, and waiver of fees.

“We are thrilled to receive Orphan Drug Designation for MP-004, which reflects the unmet medical need and the potential of our compound to make a difference for patients with retinitis,” said Dr. Adolfo López de Munain, CMO and founder of Miramoon Pharma. “We look forward to advancing MP-004 into clinical trials and bringing this innovative therapy closer to the market.”

MP-004 is the first of many lead compounds that Miramoon Pharma has identified using its proprietary platform that targets dysfunctional RyR channels. The company believes that this novel approach can address a wide range of indications, including cardiac, neurodegenerative and muscle disorders, where RyR dysregulation plays a key role. Miramoon Pharma is actively seeking licensing opportunities and partnerships to expand its pipeline and accelerate the development of its candidates.