FDA Grants Orphan Drug Designation to RCT1100 For Primary Ciliary Dyskinesia


RCT1100 is designed to deliver DNAI1 mRNA directly to affected airways through an inhaled nebulizer and fills an urgent unmet need in the treatment of those with primary ciliary dyskinesia.

The FDA has granted RCT1100, a novel investigational inhaled mRNA therapy, orphan drug designation for the treatment of primary ciliary dyskinesia (PCD), according to a news release by ReCode Therapeutics.1

3D rendering of human cilia.

Image credit: Sebastian Kaulitzki | stock.adobe.com

PCD is a progressive and rare genetic disease that is characterized by the dysfunction of cilia, which are hairlike structures that line the respiratory tract. If ciliary function becomes defective, there is an inability to clear mucus from airways, leading to viruses and bacteria becoming trapped and causing chronic infections and compromised respiratory function. A small percentage of patients even require a lung transplant due to the disease.1

Currently, there are no disease-modifying treatments or a cure that exist for PCD, making the development of a treatment a critical unmet need. It is estimated to affect at least 1 in 7500 people across multiple ethnicities, with around 45,000 patients in the United States. There is a lack of widespread screening, which means about 80% of people with PCD are thought to be undiagnosed, according to the news release.1

This specific treatment was developed to treat PCD that is caused by pathogenic mutations in the DNAI1 gene. According to ReCode Therapeutics, mutations in this gene account for about 7% of PCD cases.1

RCT1100 was developed to deliver DNAI1 mRNA to target airway cells. This mRNA is translated into the DNAI1 protein inside of cells, which by targeting the underlying cause of the disease can establish proper ciliary function in the airways.1

In preclinical studies, ReCode’s DNAI1 mRNA was well-tolerated and performed well in patients with PCD. The mRNA was able to be delivered directly to the lungs as an inhaled aerosol, and there was no significant exposure to other tissues. Importantly, a robust delivery of DNAI1 mRNA to the lung and expression of the protein in target cells was observed.2

Additionally, in a preclinical design, optimized DNAI1 mRNA reduced immune reactivity and was well-tolerated in patients.2

“PCD is a rare genetic lung disorder that currently has no FDA-approved treatments; therefore, receiving this designation is an important recognition for the PCD patient community," Shehnaaz Suliman, MD, MBA, MPhil, CEO of ReCode Therapeutics said in the news release.1

The FDA’s orphan drug designation program is designed to incentivize the creation of drugs and biologics for rare conditions and diseases. Some treatments for rare diseases have ben discontinued, or “orphaned,” because there was not enough financial motivation to continue production.3

This designation gives incentive to relevant stakeholders, including product developers and researchers, to support the development of safe drugs for rare diseases such as PCD.3

ReCode is actively enrolling patients in part B of a phase 1, open-label study that is designed to assess the tolerability and safety of a single ascending dose of inhaled RCT1100 administered with a nebulizer in patients with PCD that was caused by pathogenic mutations in the DNAI1 gene.1

"We are actively enrolling PCD patients in our single-dose study across the US and UK and look forward to sharing an update when enrollment is completed,” Suliman said.1

1. ReCode Therapeutics. ReCode Therapeutics Receives U.S. FDA Orphan Drug Designation for RCT1100 for the Treatment of Primary Ciliary Dyskinesia. News release. Published in BusinessWire June 27, 2024. Accessed June 28, 2024. https://www.businesswire.com/news/home/20240627289896/en
2. ReCode Therapeutics. ReCode Therapeutics Presents Preclinical Data from Inhaled mRNA Therapeutic Program in Primary Ciliary Dyskinesia (PCD) in Three Posters at the ATS 2022 International Conference. News release. Published on May 15, 2022. Accessed June 28, 2024. https://recodetx.com/recode-therapeutics-presents-preclinical-data-from-inhaled-mrna-therapeutic-program-in-primary-ciliary-dyskinesia-pcd-in-three-posters-at-the-ats-2022-international-conference/
3. FDA. Rare Diseases at FDA. Updated on December 13, 2022. Accessed June 28, 2024. https://www.fda.gov/patients/rare-diseases-fda#:~:text=An%20orphan%20drug%20is%20a,drug%20development%20for%20rare%20diseases.

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