PFIC treatment IN016 gets FDA orphan drug status
Therapy designed to deliver mRNA molecules to liver
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to IN016, an experimental therapy Innorna is developing for progressive familial intrahepatic cholestasis (PFIC).
The designation is meant to incentivize companies to invest in treatments for rare conditions for which there might not otherwise be an economic incentive to do so. Among other perks, orphan drug designation guarantees that if IN016 is ultimately approved, Innorna will be entitled to seven years of market exclusivity.
The FDA in July granted the therapy rare pediatric disease designation, which is similarly designed to incentivize investments in therapies for rare diseases but with a specific focus on children.
The two designations will “greatly facilitate IN016 clinical development/approval process, therefore bring this potential therapy to PFIC patients quickly,” Innorna said in a company press release.
PFIC concerns a group of rare genetic disorders in which the liver’s ability to make and/or excrete bile is impaired. Normally, the liver makes bile and ships it out to the intestines to aid in digestion. But people with PFIC typically develop cholestasis, in which the flow of bile is slowed or stalled. As a result, bile builds up in the liver and leaks out into the bloodstream, which can cause liver damage and drive itching and other disease symptoms.
Nanoparticles bring mRNA to liver cells
IN016 aims to address the root causes of PFIC using lipid nanoparticles, or tiny vesicles made of fatty molecules, to deliver genetic material into liver cells. This genetic material, called messenger RNA (mRNA), is an intermediary molecule derived from DNA that guides protein production.
IN016 is designed to deliver to liver cells specific mRNA molecules that allow people with PFIC to produce whichever bile-regulating protein is defective. The ultimate goal is to facilitate normal bile production and excretion to lessen liver damage and ease disease symptoms.
The idea of delivering mRNA molecules into cells to allow production of defective proteins is not new, but it is technically challenging, as it’s difficult to efficiently get mRNA into just the right type of cells.
Innorna has been working on lipid nanoparticles to overcome these challenges. The company has a library of thousands of specific nanoparticles tailored to help with different approaches.
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