FDA approves time-saving antibacterial drug for cystic fibrosis patients

FDA approved tobramycin inhalation powder (TOBI Podhaler, Novartis) for the management of cystic fibrosis patients with Pseudomonas aeruginosa), a bacterium that causes lung infections.

TOBI Podhaler is the first and only FDA-approved inhaled dry powder antibacterial for P aeruginosa in the United States. It does not require a nebulizer, involves minimal setup and cleanup, and does not need to be disinfected. TOBI Podhaler is designed for patients on-the-go. It delivers tobramycin into the patient’s lungs via a pocket-sized dry powder inhaler, and does not need refrigeration or a power source to deliver the medicine.

Cystic fibrosis is a genetic disease that affects about 30,000 pediatric and adult patients in the United States. Cystic fibrosis causes the body to produce thick, sticky mucus that builds up in the lungs and blocks airways. The buildup of mucus makes it easy for bacteria like P aeruginosa to grow and cause a chronic lung infection that, over time, can severely damage the lungs. Many patients with cystic fibrosis are treated with antibiotics using a nebulizer machine.

TOBI Podhaler, a plastic, hand-held inhaler device, contains a dry powder formulation of tobramycin, an antibiotic used to treat P aeruginosa infection. The powder is inhaled twice daily using the Podhaler device for 28 days. Patients should then stop TOBI Podhaler therapy for 28 days before resuming again. 

“We expect broad access for CF patients through public and private insurance, similar to that of TOBI [tobramycin inhalation solution, USP] 300 mg/5 mL,” Tina Tuttle, Novartis Pharmaceuticals spokesperson, told Formulary.

TOBI Podhaler’s effectiveness was established in a study of 95 pediatric and adult patients with cystic fibrosis. All patients were between 6 and 21 years (mean age of 13.3 years), had a forced expiratory volume in 1 second (FEV₁) within the range of 25% to 80% of predicted normal values, and were infected with P aeruginosa. They had not received inhaled antipseudomonal antibiotics within the previous 4 months. Patients were randomly assigned to receive TOBI Podhaler or a placebo for the first 28 days of the study. All patients then received treatment with TOBI Podhaler for the remainder of the 24-week study. 

The study was designed to determine the improvement in lung function by measuring the change in FEV₁. Patients treated with TOBI Podhaler experienced a statistically significant increase of 12.54% in FEV₁ compared to 0.09% in patients treated with placebo. Additional data supporting safety and effectiveness were available from other studies enrolling 487 patients.

“CF therapy requires multiple medications to be used every day with a combined average treatment time of 108 minutes per day,” Tuttle said. “In a phase 3 study, TOBI Podhaler 28 mg per capsule, shortened administration time for patients by approximately 70% compared to TOBI 300 mg/5 mL, saving about 13 hours per treatment cycle. This does not include the time saved on setting up and maintaining the nebulizer and compressor. Overall TOBI Podhaler has a safety profile similar to that of TOBI, with the exception of cough, dysphonia [voice alteration], and dysgeusia [taste disturbance] which are more common with TOBI Podhaler.”

P aeruginosa bacteria are widespread in the environment, according to Tuttle. “It is usually harmless to the average person, but a source of serious health concern to a person with cystic fibrosis,” she said. “Repeated lung infections from P aeruginosa are the leading cause of declining lung function in people with cystic fibrosis. On average, people with cystic fibrosis and P aeruginosa can experience a 2% annual decline in lung function. Up to 80% of adults between the ages of 25 and 34 with cystic fibrosis have P aeruginosa.

Common side effects reported in patients treated with TOBI Podhaler during the clinical studies included cough, productive cough that produces phlegm or mucus; hemoptysis; lung disorder; shortness of breath; fever; mouth and throat pain; dysphonia; and headache.