The status was granted by the EMA’s Committee for Orphan Medicinal Products (COMP), which reviews experimental treatments developed for rare diseases.
Thick mucus accumulates in the lungs of cystic fibrosis (CF) patients, making it difficult to breathe and promoting the growth of bacteria and subsequent lung infections, leading to further damage. Bacteria tend to attach to the lungs and form biofilms, which are thick layers of bacteria that become impenetrable to antibiotics.
SNSP113 (formerly called SYGN113) is a complex modified sugar designed to address the underlying causes of pulmonary complications in patients with CF.
The inhaled therapy targets the bacterial infection, and aids in mucus clearance. It disperses bacterial biofilms by interfering with the way bacteria stick together, and is able to kill antibiotic-resistant bacteria. SNSP113 also makes the mucus less sticky and helps in its clearance, opening up the airways. This multiple mode of action lowers inflammation, and reduces pulmonary damage and tissue scarring.
With orphan designations, the EMA encourages development of therapeutics like SNSP113 that address chronic or life-threatening rare diseases. As an orphan medicine, SNSP113 will receive several incentives from EMA during its development, such as reduced regulatory fees, potential funding for clinical trials, and scientific advice in the form of protocol assistance.
“We believe that SNSP113 has the potential to be the first broad-spectrum therapy to improve pulmonary function in patients with CF, and look forward to working with the EMA as we progress the program,” Robert Gallotto, president and CEO of Synspira Therapeutics, said in a press release.
If approved as a CF treatment in the EU, SNSP113 will be eligible for 10 years of market exclusivity, which can be extended for two more years if the therapy shows effectiveness in children. Synspira also will have access to centralized marketing authorization — the ability to apply for a single authorization that will permit its marketing throughout the EU.
SNSP113 was granted orphan drug status for CF treatment by the U.S. Food and Drug Administration (FDA) in October 2018.
In 2019, the company is planning to start a clinical trial evaluating SNSP113 in CF patients. The study will be partly funded by a $3 million development award granted by the Cystic Fibrosis Foundation for clinical advancement with SNSP113.
“With orphan designation in both the U.S. and EU, we have achieved an important milestone in our global regulatory strategy and are committed to advancing SNSP113 as a novel approach to treating this debilitating disease,” Gallotto said.