Pharmaxis announced that Bronchitol (mannitol) has been approved in Russia for the treatment of both pediatric and adult patients with cystic fibrosis (CF), the largest market to date for the medication with this indication.
Bronchitol is also approved to treat pediatric and adult CF patients (older than age 6) in Australia, and is an approved treatment for adult CF patients in the European Union and Israel. The U.S. Food and Drug Administration has not approved the medication.
Bronchitol is a dry powder mannitol, inhaled twice daily using a small handheld device. Bronchitol works by rehydrating the airways or lung surface, and promoting a productive cough. Clinical trials have shown that Bronchitol helps to increase mucus clearance, and improve the lung function and the quality of life of people with CF. The disease that affects at least 7,400 people in Russia, according to the Russian Cystic Fibrosis Registry.
It is the first drug to be processed under new regulations intended to improve patients access to medications, part of new orphan drug legislation announced by the Russian Ministry of Health in January. Bronchitol was designated an orphan drug by regulators in February, and its approval in that country opens it to patients age 6 or older.
“This is an important milestone for the company and a noteworthy achievement for Australian innovation. Bronchitol will be manufactured and exported to Russia from our purpose‐built factory in Sydney. It will be used to treat children aged 6 and above and adults throughout Russia who are suffering from the debilitating symptoms of cystic fibrosis,” said Gary Phillips, Pharmaxis CEO, in a press release. “I am also proud of the work we have done in having Bronchitol become the first drug to be granted marketing approval under the new Russian legislation. We were instrumental in demonstrating the need for the new legislation and this will ultimately mean better access to a range of medicines for Russian patients in need.”
Russia’s Ministry of Health Orphan Committee will now review Bronchitol’s application for reimbursement under its Seven Nosologies Program, which supports patient access to expensive medications for orphan — or rare — diseases such as CF. Currently, the country has 40 CF centers for children and three for adult patients, as well as small centers sited at pulmonology departments of pediatric hospitals.
“The staff of the Russian and Moscow centres of cystic fibrosis are very pleased that Bronchitol has state registration in Russia and that our patients will have access to a new innovative product,” said Nikolay Kapranov, president of the Russian Association of CF patients. “We believe the vast majority of our CF patients will be able to have free access to this modern product once it is included in the 7 Nosologies Program.”
Pharmaxis is currently conducting a 26-week, randomized, double-blind, and parallel-group clinical trial, (NCT02134353), evaluating Bronchitol administered twice-daily in adult CF patients. The study will assess improvements in lung function, pulmonary exacerbations, and the drug’s overall safety. The company expects results by mid-2017.