GNT Pharma said on Monday that Crisdesalalzine (ingredient: aspirin, sulfasalazine), an investigational treatment for amyotrophic lateral sclerosis (ALS) obtained orphan drug designation (ODD) from the Korean and European regulators.

The double designation follows its winning ODD from the FDA last year to treat ALS, also known as Lou Gehrig's disease.

Along with clinical trial design advice, reduction of screening costs, tax credits, priority screening, and exclusive sales rights, GNT Pharma will also receive support for the drug development of Crisdesalalzine from the EMA and Korea’s Ministry of Food and Drug Safety.

Crisdesalalzine is a synthetic new drug that GNT Pharma is developing as a treatment for degenerative brain diseases such as Alzheimer’s disease, ALS, and Parkinson's disease with the support of the Ministry of Science and ICT.

ALS is a degenerative brain disease characterized by the gradual degeneration and death of cerebrospinal motor neurons. It is a disease that causes muscle weakness and disorders affecting patients' eating, talking, and breathing over time and most patients die of respiratory failure within three to five years of onset.

Particularly, Crisdesalazine is a double-target brain cell protection drug that inhibits the production of the inflammatory factor prostaglandin E2 (PGE2) by inhibiting the inflammatory protein mPGES-1, and captures and removes free radicals.

In animal models, Crisdesalazine proved superior and safer than comparative ALS drugs in preventing the death of spinal motor neurons, improving disability, and prolonging life, the company said. In phase 1 clinical trials, Crisdesalazine also proved safe to administer orally at 600 mg, six times higher than the expected effective dose.

"After comprehensively evaluating Crisdesalazine’s mechanism of action in clinical and non-clinical trials, we have now obtained ODD in the U.S., Korea, and Europe, and will continue to conduct clinical trials so that it can be developed as an innovative ALS treatment,” said GNT Pharma CEO Gwag Byoung-joo.