In a recent news release, enzyme engineering company Codexis, Inc. shared that its product CDX-6512 received both Orphan Drug and Rare Pediatric Disease designations from the FDA. Both designations were granted for the treatment of homocystinuria, a rare inherited condition.
CDX-6512
CDX-6512 is designed as an enzyme therapy for those with homocystinuria. People with homocystinuria are unable to break down methionine, a type of amino acid. As a result, methionine and homocysteine (a metabolite of methionine) accumulate in the body. The heightened levels result in homocystinuria. Altogether, CDX-6512 works as an enzyme therapy which is not only able to resist stomach acids for increased efficacy, but which breaks down methionine. Codexis believes that this could relieve the symptoms and burden of homocystinuria.
The therapy received both Orphan Drug and Rare Pediatric Disease designations. Orphan Drug designation is granted to drugs or biologics intending to treat, prevent, or diagnosis rare diseases or conditions. For the purpose of this status, “rare” conditions are those affecting under 200,000 Americans. As a result of receiving this designation, the drug developer (in this case, Codexis) also receives fee waivers, tax credits, increased regulatory assistance, and 7 years of market exclusivity upon approval. Rare Pediatric Disease designation, on the other hand, is granted to drugs or biologics intending to treat rare, serious, or life-threatening illnesses affecting under 200,000 pediatric patients (those 18 or under).
About Homocystinuria
Homocystinuria is a rare inborn error of metabolism that is inherited in an autosomal recessive pattern. This means that patients must inherit one defective gene from each parent to develop this condition. As described above, those with this condition are unable to metabolize methionine, causing elevated methionine and homocysteine levels. Homocystinuria affects people in Qatar, Germany, Norway, and Ireland at a higher rate than others on a global scale. Symptoms associated with homocystinuria include:
- High foot arches
- A tall, thin build with long limbs
- Failure to thrive
- Chest deformities
- Increased risk of blood clots
- Osteoporosis
- Nearsightedness
- Psychiatric disorders
- Intellectual disabilities
- Seizures
Learn more about homocystinuria.
