Plakous Therapeutics released a press release to announce that Protego-PD™, their therapy for infants with necrotizing enterocolitis (NEC), received both the Orphan Drug and Rare Pediatric Disease designations from the FDA. There are no current FDA-approved treatments for NEC, making Protego-PD a potentially promising option.
Protego-PD was developed by Plakous Therapeutics, a biotherapeutic company who describes itself as:
pioneering in situ restorative and regenerative healing with cytokines and growth factors remaining in the human placenta after the delivery.
So, it makes sense that Protego-PD is an orally-administered placental therapy. Learn more about the value of Protego-PD here.
The FDA granted both Orphan Drug designation and Rare Pediatric Disease designation to Protego-PD. So what does that mean?
- Rare Pediatric Disease designation offers drug developers priority review vouchers, which allows them to get a quicker review of their marketing applications.
- Orphan Drug designation is given to a drug or biologic that addresses an unmet need in the rare disease community. Orphan drugs assist patients with rare diseases (under than 200,000 people affected). This designation comes with benefits such as priority review, 7 years of marketing exclusivity, and tax credits.
Necrotizing enterocolitis (NEC) is a condition which causes intestinal inflammation, primarily in the colon. When the intestines swell, holes can form, allowing bacteria to enter and infect the abdomen. Generally, NEC impacts premature babies; in fact, around 10% of premature infants will develop necrotizing enterocolitis. For very low birth weight babies (VLBWB) under 3 pounds, the percentage rises to 90%. However, the condition is much rarer in full-term infants. Necrotizing enterocolitis is fatal for about 30% of VLBWB.
Symptoms generally appear within 3-12 days after birth. These include:
- A bloated or swollen stomach
- Bloody stool
- Apnea (pauses in breathing)
- Abdominal discoloration
- Gastrointestinal issues, like diarrhea or constipation
- Trouble feeding
- Frequent vomiting
- Low blood pressure
- Slowed heart rate
Learn more about necrotizing enterocolitis here.