In the European Union, Orphan drug designation is granted by the European Commission (EC) to drugs or biologics that are intended to diagnose, prevent, or treat rare conditions. The definition of rare differs globally. For the European Union, rare refers to conditions that affect no more than five in every 10,000 people. This status comes with benefits such as 10 years of market exclusivity upon drug approval, as well as protocol assistance. Recently, biopharmaceutical company Precigen, Inc. announced via news release that its investigational therapy PRGN-2012 for recurrent respiratory papillomatosis (RRP) was granted Orphan Drug designation.
So what is PRGN-2012? This investigational vaccine, developed using Precigen’s proprietary AdenoVerse™ platform, uses a gorilla adenoviral vector to deliver human papillomavirus (HPV) 6 and HPV 11 antigens. It has the potential to offer a more targeted and less invasive treatment. At the moment, treatment typically consists of medication to slow respiratory growths, as well as surgery to remove the growths.
Currently, researchers are evaluating PRGN-2012 in adults with recurrent respiratory papillomatosis in a Phase 1/2 clinical trial.
This treatment has also received Orphan Drug, Breakthrough Therapy, and Accelerated Approval designations from the U.S. FDA.
Get the Facts: Recurrent Respiratory Papillomatosis (RRP)
An estimated 80% of sexually active women and 90% of sexually active men will contract human papillomavirus (HPV) within their lifetimes. Usually, HPV does not cause issues. Most people clear the virus from their body within years. But HPV can cause health issues, such as cancer or recurrent respiratory papillomatosis, in some individuals.
Recurrent respiratory papillomatosis is a rare respiratory tract neoplastic disease caused by HPV 6 and/or 11. RRP causes the formation of wart-like growths, most often on the vocal cords or larynx. When this disease occurs in children, it tends to be more aggressive.
Is it cancerous?
Typically, no. The growths are usually benign. However, RRP transforms into invasive squamous cell carcinoma (SCC) in about 1-3% of cases.
Risk Factors
Your risk of developing RRP increases if you were:
- Born to a young mother under 20 years old
- Vaginally delivered after prolonged labor
- Are the first-born child
Symptoms of RRP
There are a number of symptoms associated with RRP. These can include a chronic cough, difficulty speaking or swallowing, failure to thrive, stridor (noisy or difficult breathing), a hoarse voice, a weak cry, choking, frequent or recurrent pneumonia, and chronic lung disease.
