Klotho Neurosciences Secures FDA Orphan Drug Designation for Promising ALS Treatment
Boston, MA – July 10, 2025 – Klotho Neurosciences, Inc. today announced a significant milestone in its quest to combat amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. The company has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for its investigational drug, KLTO-202. This designation recognizes KLTO-202 as a potential therapeutic agent for a rare disease and provides certain incentives to accelerate its development and review.
ALS is a devastating neurodegenerative disease that affects nerve cells in the brain and spinal cord, leading to progressive muscle weakness and paralysis. Currently, there is no cure for ALS, and available treatments primarily focus on managing symptoms and slowing disease progression. The urgent need for new and effective therapies drives intense research and development efforts within the scientific and pharmaceutical communities.
The FDA’s Orphan Drug Act provides incentives to encourage the development of drugs and biologics for rare diseases and conditions affecting fewer than 200,000 people in the United States. These incentives can include market exclusivity for seven years upon approval, tax credits for qualified clinical research expenses, and assistance in designing clinical trials. This designation is a testament to the potential of KLTO-202 to address a critical unmet medical need in the ALS patient population.
Klotho Neurosciences is at the forefront of exploring the therapeutic potential of the Klotho protein. The Klotho protein is a hormone that plays a vital role in cellular aging and has been implicated in various biological processes, including neuroprotection and cognitive function. Preclinical studies by Klotho Neurosciences suggest that KLTO-202, which is designed to modulate the activity of the Klotho protein, may offer a novel approach to treating ALS by potentially protecting motor neurons from degeneration and promoting their survival.
“We are incredibly encouraged by the FDA’s decision to grant Orphan Drug Designation to KLTO-202,” stated Dr. Anya Sharma, CEO of Klotho Neurosciences. “This designation represents a crucial step forward in our mission to develop a groundbreaking treatment for individuals living with ALS. We are committed to advancing the development of KLTO-202 through rigorous clinical trials and bringing hope to the ALS community.”
The company plans to initiate further clinical studies to evaluate the safety and efficacy of KLTO-202 in patients with ALS. The Orphan Drug Designation is expected to facilitate these efforts by providing regulatory support and potentially streamlining the path to market approval, should the drug demonstrate clear benefits in human trials.
The ALS community, comprised of patients, caregivers, and researchers, will be closely watching the progress of KLTO-202. This development offers a beacon of optimism, underscoring the dedication of companies like Klotho Neurosciences in the ongoing fight against this challenging disease. The company’s commitment to advancing innovative science for rare diseases like ALS is a welcome and vital contribution to global health.
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PR Newswire Policy Public Interest published ‘Klotho Neurosciences, Inc. Granted FDA Orphan Drug Designation for KLTO-202 for Treatment of Amyotrophic Lateral Sclerosis (“ALS” or “Lou Gehrig’s Disease”)’ at 2025-07-10 09:00. Please write a detailed article about this news in a polite tone with relevant information. Please reply in English with the article only.