Renexxion goes to FDA with cystic fibrosis drug

cystic fibrosis

Renexxion Ireland Limited has opened an investigational new drug (IND) application with the U.S Food and Drug Administration (FDA) for naronapride for the treatment of GI motility disorders in patients with cystic fibrosis (CF).

This is a first step towards conducting clinical trials of the drug candidate in CF patients. Naronapride, post approval, could be a potential best-in-class pan-GI prokinetic for the treatment of GI dysmotility in CF.  

GI dysmotility in CF has a complex neuroendocrine pathophysiology causing significant morbidity. It is listed as a top research priority by patients. GI dysmotility affects more than half of the patients with CF, and is characterized by gastroparesis, abdominal pain, and severe refractory constipation. 

Although there have been advances in the treatment of CF, there is still an unmet need for the treatment of GI symptoms. Naronapride is a novel small molecule, pan-GI prokinetic with positive results from phase II clinical trials in upper and lower GI indications. It has demonstrated a comparable safety profile to placebo, making naronapride a potentially safe and effective treatment for GI dysmotility in CF.

“We are very excited about the potential of naronapride to improve the quality of lives of individuals with CF. Following discussions with the CF Foundation, CF Key Opinion Leaders and our European partner; we have identified a high unmet need for the treatment of GI dysmotility in CF. We have received constructive input from the FDA on both our clinical development plan and phase II clinical trial design. Receiving IND clearance represents a major milestone in moving forward with our clinical development plan in parallel with our partner in the EU in gastroparesis,” said Peter Milner, chairman and CEO.

Need for effective treatments

“The global cystic fibrosis patient community remains in need of effective treatments for gastrointestinal motility complications like constipation and gastroparesis.  Despite advances in treatments for the pulmonary complications of cystic fibrosis, no pro-motility drugs have replaced cisapride since its withdrawal from the market,” said Zachary Sellers, assistant professor of pediatrics-gastroenterology at Stanford University School of Medicine. 

Dhiren Patel, associate professor of pediatrics at the St. Louis University School of Medicine said: “We welcome clinical trials of naronapride in cystic fibrosis patients with gastrointestinal motility disorders.  If naronapride is proven to be effective in treating these patients, it would significantly improve their quality of life.”

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