The US Food and Drug Administration has approved a new drug for the management of cystic fibrosis in patients with Pseudomonas aeruginosa (Pa) bacteria in the lungs, which is the leading cause of loss of lung function in CF patients, according to the drug's manufacturer, Novartis.
TOBI Podhaler (tobramycin inhalation powder) is the first and only dry powder, non-nebulized CF formula on the market, and is delivered in 112mg doses (28 mg per capsule), twice daily.
The drug was developed using Novartis PulmoSphere technology, which creates hollow, porous particles of tobramycin that allows a pocket-sized inhaler to deliver a dry powder rather than as a nebulized solution, according to Novartis.
The drug does not need to be stored in a refrigerator and, unlike nebulized Pa treatments, does not require a power source to operate the delivery device. The disposable Podhaler device can be wiped clean with a dry cloth after each use and is replaced weekly. The system shortened administration time for patients by approximately 70% compared to TOBI, saving about 13 hours per treatment cycle, according to the company.
"This is good news for the CF community," Robert J. Beall, PhD, the President and CEO of the Cystic Fibrosis Foundation, said in a press release. "Managing daily CF treatments is a challenge for people with CF. TOBI Podhaler helps relieve that burden by shortening the time it takes to administer the medicine and making it easy for people with CF to take their treatment with them wherever they need to go."
The Phase III clinical study involved 674 CF patients aged six years and older with Pa in their lungs; 425 patients received at least one dose of TOBI Podhaler. Two trials evaluated the efficacy of TOBI Podhaler vs. placebo, while a third trial assessed the safety of TOBI Podhaler vs. TOBI. All studies evaluated TOBI Podhaler at the approved twice-daily dosage of 112 mg in cycles of 28 days on, immediately followed by 28 days off treatment. Results of the efficacy studies showed that TOBI Podhaler improved lung function compared to placebo, according to Novartis.
Novartis emphasized that the product only received approval at 112mg twice daily, for CF patients age 6 and over, with Pseudomonas aeruginosa (Pa) bacteria in their lungs.It is not known if TOBI Podhaler is safe and effective in patients under six years of age, in those with lung function outside of a certain range, or in those whose lungs contain bacteria called Burkholderia cepacia, the company said.
TOBI Podhaler is anticipated to be available in the US in the second quarter of 2013.
More information on TOBI Podhaler and its safety information is available on the Novartis website.