Orphan Drug designation is a status granted by the FDA to drugs or biologics intended to treat patients with rare conditions. In this case, the FDA defines “rare” as affecting 200,000 or less Americans. Once a drug is granted Orphan Drug status, the drug developer receives a variety of benefits, from tax credits and fee waivers to up to 7 years market exclusivity upon approval. According to Asbestos.com, the FDA recently granted Orphan Drug designation to ONCOFID-P, a potential treatment option for patients with malignant pleural mesothelioma (MPM).
ONCOFID-P
Unfortunately, there are not many treatment options for patients with MPM. In 2020, the FDA approved the combination treatment of Opdivo and YERVOY – the first new treatment option in over 15 years. Still, if ONCOFID-P showed promise in treating patients, it could fulfill an unmet need.
So what is ONCOFID-P? This treatment blends Paclitaxel, a form of chemotherapy, with hyaluronic acid. Ultimately, the hyaluronic acid helps the treatment bind to CD44-expressing cancer cells. Normally, CD44 plays a role in cell growth and proliferation. Thus, by targeting CD44-expressing cells, the treatment is designed to reduce tumor size and stop cancer progression. Because of its intrapleural administration, it should also be relatively safe and well-tolerated.
In preclinical studies, ONCOFID-P has shown to reduce tumor size and promote an anti tumor microenvironment. Moving forward, the drug will be explored in clinical trials. In the past, ONCOFID-P has also been explored as a treatment option for patients with invasive bladder cancer.
Malignant Pleural Mesothelioma (MPM)
Malignant pleural mesothelioma (MPM), although rare, is the most common form of mesothelioma, making up about 75% of diagnoses. Altogether, MPM is an aggressive cancer, which begins in the pleura (lining) of the lungs. This cancer is caused by asbestos exposure and inhaling asbestos fibers, which get caught in the lungs, causing irritation, inflammation, and ultimately, MPM development. In many cases, however, symptoms will not appear until over 20 years following diagnoses. Prognosis is typically bad but can differ based on when the tumor is discovered. Symptoms include:
- Shortness of breath
- Difficulty breathing and swallowing
- Night sweats
- Fatigue
- Fluid in the lungs
- Lumps on the chest
- Fever
- Unintended weight loss
- Chest, rib, and lower back pain
- Pain while breathing
- Swelling of the arms and/or face
- Coughing up blood
