PFIC therapy IN016 earns FDA’s rare pediatric disease designation
Treatment aims to address root cause of rare genetic liver disease
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to IN016, Innorna’s experimental therapy that aims to address the root cause of progressive familial intrahepatic cholestasis (PFIC).
The FDA designation is given to investigational therapies intended to treat serious or life-threatening conditions affecting fewer than 200,000 people in the U.S., and specifically those younger than 18.
It is intended to encourage pharmaceutical companies to develop treatments for rare pediatric diseases that might not otherwise prove profitable. Should the FDA eventually approve IN016, Innorna may qualify for a priority review voucher for a subsequent application for a different product. Priority review status shortens the review process from a standard 10 months to about six months.
“The [rare pediatric disease] designation represents a significant step in developing … IN016 as [a] potential [treatment] for … PFIC,” Linxian Li, PhD, Innorna’s founder and CEO, said in a company press release.
Rare pediatric disease designation will help speed IN016’s development
PFIC is a group of rare genetic liver diseases characterized by impaired production and/or secretion of bile, a digestive fluid, by liver cells. It is classified into different types based on the gene that is mutated.
The condition typically causes cholestasis, or slowed bile flow from the liver to the intestines, in early childhood. For patients, this leads to bile accumulation in the liver, and leakage to the bloodstream, and ultimately, symptoms such as itching and jaundice, or a yellowing of the skin and the whites of the eyes.
Over time, the disease may evolve into irreversible liver scarring and liver failure.
While medications are available that may help reduce bile accumulation and/or relieve symptoms, “more effective treatment is in great need for PFIC,” Innorna stated in the release.
We are excited to continue [IN016’s] advancement toward the clinic.
IN016 is designed to address the condition’s root cause. It relies on the company’s proprietary lipid nanoparticle (LNP) technology, which uses tiny fatty vesicles to deliver specific molecules of messenger RNA (mRNA) to specific cells. mRNA is the intermediate molecule derived from DNA that guides protein production.
By delivering to cells mRNA molecules specific to proteins that are defective in PFIC patients, the therapy is expected to restore levels of these proteins. In so doing, IN016 would thus normalize bile production and/or flow, and ease disease symptoms.
According to the company, its LNP technology was developed to overcome many challenges associated with the delivery of mRNA therapies for rare diseases, including tissue-specific delivery, as well as to improve delivery safety and efficiency.
Innorna noted that the company is working to quickly bring its novel mRNA therapy to PFIC patients — efforts it says the rare pediatric disease designation will “greatly facilitate.”
“We are excited to continue [IN016’s] advancement toward the clinic,” Li said.
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