Codexis Inc. (NASDAQ: CDXS) has announced that the FDA has granted its CDX-6512 drug candidate an orphan drug designation in homocystinuria treatment.
CDX-6512 has the potential of treating homocystinuria
As a gastrointestinal-stable methionine-gamma-lyase, CDX-6512 can be used as a homocystinuria enzyme treatment (HCU). The FDA has also designated CDX-6512 as a rare pediatric disorder (RPD). CDX-6512 is the most developed wholly-owned product in Codexis’ biotherapeutics pipeline, now in the pre-IND study.
CEO and president of Codexis John Nicols said, “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.”
Nicols explained that the designations are a vital element in the process of developing CDX-6512 as a possible homocystinuria treatment, and the company is looking to advance it to clinical trials.
CDX-6512 is a gastrointestinal-stable enzyme developed to be extremely resistant to both upper-intestinal proteases and stomach acid, allowing it to successfully break down methionine generated during protein digestion. As a result, the different clinical symptoms of HCU are caused by elevated amounts of the amino acid as well as its metabolite homocysteine. Codexis presented pre-clinical findings emphasizing the CDX-6512 program at the 14th International Congress of Inborn Errors of Metabolism (ICIEM) in November 2021.
ODD to grant Codexis marketing exclusivity in the US
The FDA grants ODD to biologics and medicines that are designed to treat, diagnose, or prevent illnesses or conditions that impact less than 200,000 individuals in the US. Sponsors may be eligible for tax credits covering the cost of clinical studies and waivers of prescription drug user fees as a result of the designation. In addition, should Codexis gain FDA approval for homocystinuria treatment for this pharmaceutical candidate, the ODD might grant the business a seven-year commercial exclusivity in the United States for CDX-6512.
The FDA gives RPD designation to drugs that are designed to treat severe or life-threatening illnesses that mainly affect children up to 18 years old and affect less than 200,000 individuals in the US.