In May 2023, Pharmaceutical Technology reported that INO-3107, a DNA medicine vaccine candidate developed by biotechnology company Inovio Pharmaceuticals (“Inovio”), received Orphan Drug designation from the European Commission. INO-3107 was developed for people living with recurrent respiratory papillomatosis (RRP). It targets human papillomavirus 6 and 11, which have both been associated with this rare condition.
This status is granted to drugs or biologics intended to treat, diagnose, or prevent a rare condition; in the European Union, a “rare” condition is one affecting no more than 5 in every 10,000 people. Additionally, Orphan Drug status comes with a variety of incentives such as protocol assistance and ten years of market exclusivity upon drug approval. Learn more about Orphan Drug designation.
About Recurrent Respiratory Papillomatosis (RRP)
Human papillomavirus (HPV) is more widespread than we think. An estimated 90% of sexually active men, and 80% of sexually active women, will develop HPV in their lifetime. In many cases, the immune system helps the body clear HPV within a few years. However, in a smaller subset of people, HPV can turn into cancer or other conditions such as RRP. Risk factors for RRP include being born to a young mother (under 20 years old), being the first-born child, or being vaginally delivered after prolonged labor.
RRP causes wart-like growths to form in the respiratory tract, most often in the larynx and vocal cords. While these growths are typically non-cancerous, they can turn cancerous in rare cases. There is no cure for RRP, but it can be managed. Surgery to remove the growths, and medicine to slow the growths, are often used in tandem. Juvenile cases (often in children aged 12 or younger) are often more aggressive than RRP in adults.
Symptoms of RRP may include:
- Hoarseness
- Labored, noisy, or difficult breathing which begins when breathing in but progresses to both inhaling and exhaling
- Chronic cough
- Dysphagia (difficulty swallowing)
- Failure to thrive
- Difficulty speaking
- Choking episodes
- A weak cry (in infants)
- Squamous cell carcinoma (rare complication)
- Recurrent pneumonia (complication)
- Chronic lung disease (complication)
