GNT Pharma's 'Chrisdesalazine' Designated as FDA Orphan Drug for Lou Gehrig's Disease

GNT Pharma is developing 'Chrisdesalazine' as a treatment for degenerative brain diseases. (Photo by GNT Pharma)

[Asia Economy Reporter Chunhee Lee] Genetipharm announced on the 1st that 'Chrisdesalazine,' being developed as a treatment for degenerative brain diseases, has been designated as an Orphan Drug Designation (ODD) for the development stage treatment of amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease) by the U.S. Food and Drug Administration (FDA).

ALS is the most common motor neuron disease in adults, characterized by the progressive degeneration and death of motor neurons in the brain and spinal cord, typically occurring at an average age of 58 to 60. Once onset occurs, generalized muscle paralysis appears, causing impairments in voluntary movements such as speaking, eating, moving, and breathing. Most patients die from respiratory failure within an average of 3 to 5 years after onset. The incidence rate of ALS is 0.6 to 3.8 per 100,000 people, and the prevalence rate is 4.1 to 8.4 per 100,000 people, with the current estimated number of patients worldwide exceeding 400,000.

Genetipharm was selected for the '2022 Pharmaceutical Industry Full-Cycle Global Expansion Support Project' led by the Korea Health Industry Development Institute and has been preparing for the entry of Chrisdesalazine into the U.S. and European markets. With Chrisdesalazine now designated as an FDA orphan drug for ALS treatment, it will receive various benefits in the U.S. during the development stage, including tax incentives, priority review, post-marketing exclusivity, and extension of marketing authorization.

Currently approved ALS treatments by the FDA include 'Rilutek' (active ingredient riluzole), a glutamate toxicity inhibitor that extends life by 3 to 6 months, and 'Radicava' (active ingredient edaravone), an antioxidant drug that slows the progression of daily living disabilities. As of 2019, sales of ALS treatments in the U.S. amounted to $197 million (approximately 281.3 billion KRW), with Radicava accounting for the highest sales at $162 million (approximately 231.3 billion KRW).

Byungjoo Kwak, CEO of GNT Pharma (Photo by GNT Pharma)

Chrisdesalazine was developed as a multi-target drug that simultaneously removes reactive oxygen species and inflammation, which are causes of onset and progression of degenerative brain diseases. In animal models, it showed superior effects in slowing the rate of motor function deterioration and extending life compared to comparator drugs including riluzole.

In Phase 1 clinical trials, oral single and multiple doses of Chrisdesalazine were administered to 75 healthy adults, including elderly subjects, to evaluate safety and pharmacokinetic characteristics, confirming excellent safety and tolerability. Among them, the total amount of Chrisdesalazine absorbed into the blood (systemic exposure) after oral administration of 100 mg Chrisdesalazine in adults including the elderly was twice as high as the systemic exposure in ALS and Alzheimer's disease mouse models. Accordingly, the target dose of Chrisdesalazine in Phase 2 clinical trials for ALS and Alzheimer's disease is expected to be 100 mg once daily.

Byungjoo Kwak, CEO of Genetipharm (adjunct professor at Yonsei University Department of Life Sciences), said, “Chrisdesalazine demonstrated superior efficacy in animal models compared to ALS treatments Rilutek and Radicava, and excellent safety was verified in humans at the target dose. We plan to officially start the ALS clinical trials of Chrisdesalazine, designated as an FDA development-stage ODD, next year.”