GNT Pharma announced on the 27th that 'Chrisdesalazine,' which is being developed as a treatment for degenerative brain diseases, has been designated as an orphan drug for amyotrophic lateral sclerosis (ALS) by the Korea Ministry of Food and Drug Safety and the European Medicines Agency (EMA).
When designated as an orphan drug, benefits such as consultation on clinical trial protocol design, reduction of review fees, tax credits, priority review, and exclusive marketing rights are provided. With such support, the cost of developing new drugs for rare diseases is significantly reduced, and the success rate from Phase 1 clinical trials to drug approval reaches 17%, more than twice that of general new drugs. The global orphan drug market is growing at an average annual rate of 12%. As of 2018, the average annual patient burden cost for rare disease drugs in the United States was approximately $150,854 (about 200 million KRW), which is 4.48 times higher than that of general specialty drugs.
Degenerative brain diseases are intractable diseases that occur as nerve cells gradually die along with aging, caused by the accumulation of toxic factors such as inflammation and reactive oxygen species in the brain. Representative degenerative brain diseases include dementia, ALS, and Parkinson's disease.
Chrisdesalazine is a dual-target neuroprotective drug that blocks the action of the inflammation-inducing protein mPGES-1, thereby inhibiting the production of the inflammatory factor prostaglandin E2, and removes reactive oxygen species through a strong free radical scavenging effect. GNT Pharma explained that in ALS animal models, it was found to be superior and safer compared to comparator drugs by preventing the death of spinal motor neurons while improving disabilities and extending lifespan. In a completed Phase 1 clinical trial involving 72 healthy adults including the elderly, oral administration of 600 mg, six times the expected effective dose, was safe.
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a degenerative brain disease characterized by the gradual degeneration and death of motor neurons in the brain and spinal cord. It causes muscle weakness and, over time, leads to difficulties in eating, speaking, and breathing. Most patients die from respiratory failure within 3 to 5 years after onset.
Byung-Joo Kwak, CEO of GNT Pharma (also an adjunct professor at Yonsei University Department of Life Sciences), said, "We will proceed without delay with the optimal clinical trials to verify the efficacy of Chrisdesalazine so that it can be developed as an innovative treatment for ALS."
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