Novartis Pharmaceuticals Corporation has received the US Food and Drug Administration (FDA) approval for TOBI Podhaler (tobramycin inhalation powder) 28 mg per capsule for the management of cystic fibrosis (CF) patients with Pseudomonas aeruginosa (Pa) bacteria in the lungs. Pa is the leading cause of loss of lung function in CF patients.

Its 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.

TOBI Podhaler is a new, non-nebulized formulation and delivery system of tobramycin, the same active ingredient as in TOBI (tobramycin inhalation solution, USP) 300 mg/5 mL, which has been on the market for approximately 15 years. TOBI Podhaler is the first and only FDA-approved dry powder inhaled antibacterial for Pa in the US. It delivers tobramycin into the patient’s lungs via a pocket-sized dry powder inhaler and offers better portability than TOBI, which is administered using a nebulizer. In a phase III study, TOBI Podhaler shortened administration time for patients by approximately 70 per cent compared to TOBI, saving about 13 hours per treatment cycle. This does not include the time saved on setting up and maintaining the nebulizer and compressor.

TOBI Podhaler 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. While the nebulizer used to administer TOBI must be disinfected in boiling water for 10 minutes every other treatment day, the disposable Podhaler device must only be wiped clean with a dry cloth after each use and is then replaced weekly.

“This is good news for the CF community,” said Robert J Beall, Ph.D., president and CEO, Cystic Fibrosis Foundation. “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 new TOBI Podhaler dry powder formulation was developed using proprietary Novartis PulmoSphere technology. This technology enables the creation of hollow porous particles of tobramycin that can be delivered as a dry powder rather than as a nebulized solution.

“TOBI Podhaler is an example of how Novartis is utilizing innovative technologies to better meet the needs of patients. By eliminating the need for a nebulizer to deliver tobramycin and providing a small, lightweight design, TOBI Podhaler reduces administration time and improves portability for patients on-the-go,” said André Wyss, president of Novartis Pharmaceuticals Corporation. “It also underscores our long-term commitment to the cystic fibrosis community."

TOBI Podhaler was studied in a phase III clinical programme involving 674 CF patients aged six years and older with Pa in their lungs, of whom 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 dose of 112 mg twice daily (the contents of four 28 mg capsules per dose) 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.

The safety of TOBI Podhaler was evaluated in 425 patients who received at least one dose of TOBI Podhaler, including 273 who were exposed across three cycles. In the two placebo-controlled efficacy trials, adverse reactions reported more commonly with TOBI Podhaler compared to placebo included pharyngolaryngeal pain (sore throat), dysphonia (voice alteration) and dysgeusia (taste disturbance) in one study and cough and hypoacusis (decreased hearing) in the other study. In the safety study, the most commonly reported adverse reactions (as defined as >10 per cent in either treatment arm) with TOBI Podhaler were cough, lung disorder (pulmonary or CF exacerbations), productive cough, dyspnea (shortness of breath), pyrexia (fever), oropharyngeal pain (sore throat), dysphonia (voice alteration), hemoptysis (coughing up blood) and headache.

CF is a life-shortening genetic disease that primarily impacts the lungs and digestive system, making it hard to breathe and to digest food. Pa is the leading cause of loss of lung function in people with CF. The treatment burden for CF is very high. CF therapy requires multiple medications to be used every day with a combined average treatment time of 108 minutes per day.

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