Regulatory Focus™ > News Articles > New CF Therapy Approved, Once Again Based on Help From Patient Group

New CF Therapy Approved, Once Again Based on Help From Patient Group

Posted 25 March 2013 | By Alexander Gaffney, RAC

For the second time in as many years, the US Food and Drug Administration (FDA) has approved a new product intended to treat patients with cystic fibrosis (CF), a rare disease that affects fewer than 30,000 patients in the US.


The new product, known as the TOBI Podhaler (tobramycin inhalation powder) is a combination product that contains an antibiotic powder, tobramycin, which is used to treat CF patients with Pdeudomonas auruginosa, a type of bacteria that causes lung infections.

The bacteria is particularly disadvantageous in CF patients, and a statement by Novartis, the drug's sponsor and manufacturer, explained that infections caused by Pseudomonas aeruginosa bacteria are the leading cause of loss of lung function in CF patients.

The infections are thought to presently infect approximately 80% of CF patients, who are particularly susceptible to lung infections because of the pathophysiology of the disease, which causes excess growth of mucus in the lungs and airways-a favorable environment for bacterial growth.

While the TOBI Podhaler isn't the first product approved to treat CF patients with Pseudomonas aeruginosa infections, FDA and Novartis said it represents an improvement over existing treatments because it is portable. Other treatments involving TOBI rely on a nebulizer to administer the treatments, which is not as portable and must be regularly disinfected by users.

The TOBI Podhaler, on the other hand, is a pocket-sized inhaler that is replaced once per week, removing the need to disinfect the product. It also does not need to be refrigerated, allowing patients additional flexibility.

An Improvement in Form More than Function

The combination of these two factors, as well as the new formulation of TOBI, allows the drug to be administered in 70% less time than existing treatments, saving patients "about 13 hours per treatment cycle," Novartis claimed.

"Today's approval broadens the available delivery mechanism options for patients with cystic fibrosis who require treatment for P. aeruginosa," said Edward Cox, director of the Office of Antimicrobial Products in the FDA's Center for Drug Evaluation and Research (CDER). "This product is the first dry powder antibacterial drug delivered with a handheld dry powder inhaler."

"This is good news for the CF community," added Robert Beall, president and CEO of the Cystic Fibrosis Foundation, a group deeply involved in developing treatments for CF. "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."

Like prior CF treatments, including Vertex Pharmaceuticals' Kalydeco, Novartis' research on the TOBI Podhaler was supported by the Cystic Fibrosis Foundation's Therapeutics Development Network Coordinating Center (CFFT).

The product's effectiveness was tested in a trial of 95 pediatric and adult patients with CD, all of whom were older than six years of age and infected with P. aeruginosa. FDA's approval statement explained that patients taking the TOBI Podhaler treatment experienced a 12.5% increase in lung function (FEV1) compared to just .09% in a placebo control group.

The product is expected to be available in the second quarter of 2013, Novartis said.

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