Benzinga | Jan 24, 2022 08:06AM EST

Codexis Announces FDA Orphan Drug And Rare Pediatric Disease Designations For CDX 6512 For Treatment of Homocystinuria

Codexis, Inc. (NASDAQ:CDXS), a leading enzyme engineering company enabling the promise of synthetic biology, today announced that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation (ODD) for CDX-6512 for the treatment of homocystinuria. CDX-6512 is a gastrointestinal-stable methionine-gamma-lyase, as a potential orally-administered enzyme therapy for homocystinuria (HCU). The FDA also granted the company rare pediatric disease (RPD) designation for CDX-6512. CDX-6512 is currently in pre-IND development and is the most advanced wholly owned program in the Company's biotherapeutics pipeline.



"The orphan drug and rare pediatric disease designations for CDX-6512 further build upon the momentum our engineered enzymes have generated as potential first-in-class oral therapeutics for inborn errors of metabolism," said John Nicols, President and CEO of Codexis. "These designations represent an important step forward in the development of CDX-6512 as a potential treatment of homocystinuria and we look forward to continuing its advancement toward the clinic."

CDX-6512 is a gastrointestinal-stable enzyme specifically engineered to be highly resistant to both the acidic conditions of the stomach and to proteases of the upper intestines, enabling it to effectively degrade methionine that is liberated from protein digestion. Elevated levels of this amino acid and its metabolite homocysteine, leads to the various clinical manifestations of HCU. The Company previously presented pre-clinical data highlighting the CDX-6512 program at the 14th International Congress of Inborn Errors of Metabolism (ICIEM) in November 2021.

ODD is granted by the FDA to drugs and biologics intended for the treatment, diagnosis or prevention of diseases or disorders that affect fewer than 200,000 people in the United States. The designation provides incentives for sponsors to develop products, which may include tax credits toward the cost of clinical trials and prescription drug user fee waivers. The ODD could also entitle Codexis to a seven-year period of marketing exclusivity in the United States for CDX-6512 should the company receive FDA approval for the treatment of homocystinuria for this product candidate.

RPD designation is granted by the FDA to drugs intended to treat serious or life-threatening diseases that primarily affect individuals aged from birth to 18 years and fewer than 200,000 persons in the United States.