Experimental Treatment for Recurrent Respiratory Papillomatosis Earns Orphan Drug Designation

According to a story from PR Newswire, the biotechnology company INOVIO has just announced that the company’s investigational product candidate INO-3107 has earned Orphan Drug designation from the US Food and Drug Administration (FDA). This experimental DNA medicine is being developed as a treatment for recurrent respiratory papillomatosis, a rare disease. The drug is currently being tested in a phase 1/2 clinical trial. INOVIO focuses on developing DNA based medicines to treat cancer and infectious diseases.

About Recurrent Respiratory Papillomatosis (RRP)

Recurrent respiratory papillomatosis, also known as laryngeal papillomatosis, is a rare disease in which tumors form in the larynx, or voice box. These tumors, called papillomas, occur because of an infection caused by the human papillomavirus (HPV). While these tumors are not cancerous, they can recur easily. It is more likely to occur in immunocompromised people and alongside other viral infections. There are juvenile and adult forms of recurrent respiratory papillomatosis. Symptoms include a change in vocal quality, hoarseness, shortness of breath, narrowing airway, coughing, pneumonia, problems swallowing, and acute respiratory distress syndrome. Tumors can spread to the lungs in severe cases. There is no cure for the disease and repeated treatment is usually necessary. The primary intervention is surgery to take out the tumors, but there is a risk of complications. Other options include voice therapy, antiviral drugs, and HPV vaccines. To learn more about recurrent respiratory papillomatosis, click here.

About Orphan Drug Designation

Orphan Drug designation is usually reserved for therapies that are intended to treat diseases that are considered rare. This is defined as any disease that affects less than 200,000 people in the US. To qualify for this designation, an medication must either have the potential to fulfill a critical unmet medical need or display advantages in safety or efficacy over currently available therapies. Orphan Drug designation confers several benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a period of market exclusivity lasting seven years if the drug is ultimately approved by the FDA.

About the Trial

The ongoing trial for INO-3107 is currently enrolling a total of 63 patients. These patients are expected to have disease linked with either HPV 6 or HPV 11, the most common genotypes of HPV linked with recurrent respiratory papillomatosis. In addition, these patients must require surgeries at least twice a year to remove tumors for three years in a row. Adult patients will undergo surgery to remove tumors and then will be treated with four doses of the drug, once every third week.

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