Arch Biopartners recently announced that it filed an application for orphan drug designation to the European Medicines Authority (EMA) for its medicine AB569, developed as a treatment for the bacterium Pseudomonas aeruginosa (P. aeruginosa) that causes pulmonary infections in patients with cystic fibrosis (CF).
The designation would provide Ontario, Canada-based Arch Biopartners with several incentives, including protocol assistance, guidance from the EMA on the types of clinical studies, access to a centralized authorization procedure, lower regulatory fees, and protection for 10 years from market competition, according to a company press release.
CF is a genetic disorder caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is responsible for the regulation of mucus production, sweat, and digestive fluids. Patients with lung CF often suffer from a build-up of mucus in the airways that makes them more predisposed to lung infections.
Respiratory infections with P. aeruginosa, a common bacterium found in soil, water, skin, and other man-made environments, happens in more than 40 percent of CF patients between the ages of 6 and 10. This value increases to 60 percent by the age of 17, and reaches around 75 percent of all CF patients ages 25-34.
A number of antibiotics are currently available on the market to treat infections with P. aeruginosa including aminoglycosides, quinolones, and carbapenems. But P. aeruginosa has an extreme potential for developing resistance to antibiotics and other phagocyte-mediated killing. This results in the decline of lung function in CF patients, and eventually a poor prognosis.
Arch Biopartners’s AB569 is a novel therapy for pulmonary infections with mucoid and non-mucoid P. aeruginosa bacteria that are resistant to traditional antibiotics. In the U.S., AB569 received orphan drug designation by the FDA in Nov. 2015 for its combined active ingredients in an inhaled solution.
In Europe, the main prevalence of CF is around 0.74 cases per 10,000 individuals among 27 European Union countries. This is below the described limit for an orphan or rare disease, so AB569 may be qualified for an orphan drug designation.
The EMA is currently examining the application, and the Committee for Orphan Medicinal Products (COMP) will most likely start the official review in about 30 days. The outcomes of this review are expected to be transferred to the European Commission by the end of May. After that, depending on the recommendations, the EC will decide whether to grant an orphan drug designation for AB569.
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