Sickle Cell Disease
Sickle cell disease is a rare blood disorder. It is progressive, debilitating, and sadly can be life-threatening. The genetic defect leads patients’ red blood cells to be shaped abnormally. Named after the shape they take, these inflexible cells cause harm to patients because they aren’t able to carry as much oxygen in the hemoglobin. This leads to inflammation, pain, bacterial infections, anemia, and strokes.
Rare Pediatric Disease Designation
Aruvant has just announced wonderful news for sickle cell disease patients. The FDA has granted a new designation to ARU-1801, specifically for the condition. ARU-1801 is their lead drug candidate. It is a gene therapy which uses a modified fetal hemoglobin.
Rare Pediatric Disease Designation allows the company to obtain a voucher for priority review, if they receive approval for their Biologics License Application.
This designation has shown that the FDA understands there is a high unmet need for this condition. Rare Pediatric Disease Designation is only provided to therapies which the FDA believes have a strong chance of supporting rare, serious, or life threatening disease affecting individuals who are under the age of 18 who have a rare condition. Rare diseases specifically are defined as those which affect fewer than 200,000 people in the entire country.
Aruvant is so excited about this investigative therapy because it doesn’t require high intensity myeloablative conditioning regimens. These are used in other investigative therapies for the disease. Unfortunately, these regimens have many risks for complications and typically lead to long hospital stays for patients.
Instead of this approach, ARU-1801 uses a Reduced Intensity Conditioning approach. This approach is associated with much fewer risks. For example, there is a much lower chance that a patient will become infertile. Additionally, they have shorter hospital stays.
You can read more about this therapy here.