Inhaled CF therapy RCT2100 granted FDA’s orphan drug status
In early clinical testing for patients not eligible for CFTR modulator therapies
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to RCT2100, an experimental treatment for cystic fibrosis (CF) that’s currently in early clinical testing for patients who do not respond to or do not tolerate CFTR modulator therapies.
The designation is meant to give extra incentives to companies that are investing in treatments for rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S. Among the perks is guaranteed seven years of market exclusivity if a therapy is ultimately approved.
“We are grateful to the cystic fibrosis community and physicians for the continued support of the RCT2100 clinical program,” Shehnaaz Suliman, MD, CEO of Recode Therapeutics, which is developing RCT2100, said in a company press release.
CF is caused by mutations in the CFTR gene, which provides instructions to make the CFTR protein. This protein plays a crucial role in regulating mucus production by controlling the movement of salt and water across cell membranes. In CF, the protein is dysfunctional or absent, resulting in thick, sticky mucus that builds up in organs and drives most disease symptoms.
CFTR modulators work only in patients with certain mutations
In recent years, a new class of treatments called CFTR modulators has revolutionized care for some people with CF. Modulator therapies can bind to mutated CFTR protein, boosting the protein’s activity to help restore normal mucus production. However, modulators work only in people with CF caused by certain mutations, and like any medicine, they can cause side effects that some patients are not able to tolerate.
When genes are read to make protein, the genetic code is copied from a cell’s DNA into a temporary molecule called messenger RNA (mRNA), which is then used as a template for protein production. RCT2100 is designed to deliver, via inhalation, a healthy version of CFTR mRNA to lung cells, allowing cells to produce healthy CFTR protein. Notably, this approach is expected to work in all CF patients irrespective of their underlying mutation.
“While there have been tremendous advancements in the development of novel CF therapies in the past two decades, we are focused on the subset of CF patients who are not eligible for current treatments and who have been waiting for a treatment,” Suliman said.
Recode is currently running a Phase 1b clinical trial (NCT06237335) testing RCT2100. The first part of the study tested the therapy’s safety in healthy volunteers, and the second part of the study is evaluating the treatment in people with CF who cannot take CFTR modulators. The trial is currently recruiting adults ages 18 to 55 at sites in the U.S., U.K., France, and the Netherlands.
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