Innovative Treatment for Cystic Fibrosis Patients, Tobi Podhaler (Tobramycin Inhalation Powder) Receives Positive Draft Guidance From Nice
Frimley, UK, 24 October 2012 – Today the National Institute for Health and Clinical Excellence (NICE) has issued a preliminary recommendation, known as the Appraisal Consultation Document (ACD), indicating its intention to recommend TOBI® Podhaler® (tobramycin inhalation powder) as a cost-effective therapy1. This innovative drug-delivery device was launched last year for the suppressive treatment of chronic pulmonary infection due to Pseudomonas aeruginosa (Pa) in adults and children aged 6 years and older with cystic fibrosis (CF)2.
Today’s draft guidance recommends tobramycin dry powder as an option for treating chronic pulmonary infection caused by Pa in people with CF, where nebulised tobramycin is considered an appropriate treatment. This is when nebulised colistimethate is contraindicated, not tolerated or has not produced an adequate clinical response1 .The NICE appraisal committee concluded that tobramycin dry powder provides a cost benefit compared to existing nebulised alternatives when combined with the Department of Health approved patient access scheme1. This is the appraisal committee’s preliminary recommendations. It is not NICE’s full guidance and may change after consultation.
Dr Diana Bilton, Consultant Respiratory Physician, Royal Brompton Hospital, London said, “this preliminary recommendation from NICE is really encouraging. Our experience is that patients really benefit from a swap to this antibiotic inhaler in terms of a reduction in treatment burden and being able to get on with life instead of spending time on a nebuliser.”
To manage all aspects of their condition, people with CF can take up to two hours a day to complete all of their treatments3. The NICE Committee recognised that the strict routine and amount of time spent receiving treatment have a significant impact on the daily activities of people with CF and their families1. The Committee felt that reducing the time that people with CF spend receiving treatment would be beneficial in improving the quality of life of people with CF and their families1. The Committee also concluded that in clinical practice people with CF will benefit from the speed and convenience of this dry powder inhaler1.
The Podhaler® is a novel drug delivery system with patented technology that delivers tobramycin inhalation powder via a hand-held, portable inhaler2,3. Trials have shown that those patients using the drug-delivery device completed their treatment in just five to six minutes, twice a day4. This is considerably faster than TOBI® (300mg/5ml tobramycin nebuliser solution), which takes approximately 20 minutes to administer, twice a day; decreasing treatment time by 72% (or by approximately 28 minutes per day) compared with tobramycin nebuliser solution (300mg/5ml)4.
Clinical trial data have also shown that the drug-device combination has similar efficacy to tobramycin nebuliser solution (300mg/5ml)4. It also offers the clinical benefits of improved lung function and reduced hospitalisations, compared with placebo5. In addition it has the advantage of greater convenience and patient satisfaction compared with nebulised tobramycin (300mg/5ml)4.
The majority of adverse reactions reported with tobramycin inhalation powder were mild to moderate, and the incidence of serious adverse events was shown to be similar between tobramycin inhalation powder and tobramycin nebuliser solution (300mg/5ml)4 .The safety of tobramycin inhalation powder has been studied in two separate clinical trials4,5. The most commonly reported adverse events in the main safety study, where tobramycin inhalation powder was compared to tobramycin nebuliser solution, were cough, lung disorder, productive cough, fever, shortness of breath, mouth and throat pain and change in voice2,4 .
The ACD provides initial draft recommendation from NICE, following input from professional and patient care groups. Today’s announcement is the appraisal committee’s preliminary guidance and final guidance on the use of tobramycin inhalation powder is due to be published in early 2013.
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Notes to editors:
TOBI® Podhaler® (tobramycin inhalation powder) was granted a Marketing Authorisation for an orphan medicinal product for human use on 20 July 2011 by the European Commission. The device was launched in the UK in September 2011 and received positive SMC guidance in June 2012.
The positive NICE ACD decision was based on the Novartis submission of data demonstrating non-inferiority to tobramycin inhalation solution (via a nebuliser) measured by relative change in FEV1 % predicted over three treatment cycles in a phase III, open-label, randomised study4 and the submission of a patient access scheme (PAS).
The tobramycin inhalation powder is a novel dry powder, which has been produced using patented PulmoSphere® technology and delivers light, porous tobramycin particles that require little effort to be inhaled deep into the lungs and is the first licensed treatment to be formulated using this technology2,3. It has a recommended dosage of four capsules (4 x 28 mg = 112 mg tobramycin), administered twice daily for 28 days (alternating cycles of 28 days on treatment followed by 28 days off treatment)2. The two doses (of 4 capsules each) should be inhaled as close as possible to 12 hours apart and not less than 6 hours apart2. The plastic inhaler is light-weight, portable and, unlike a nebuliser, has no need for an external power source or batteries, and the tobramycin dry powder capsules do not require refrigeration2,3.
For full Product Characteristics, please visit http://www.medicines.org.uk
CF is one of the UK's most common life-threatening inherited diseases, affecting more than 9,000 people6,7. The majority of CF deaths result from loss of lung function linked to inflammation due to chronic bacterial infection, principally Pseudomonas aeruginosa (Pa)5,7,8. Pa is the most common respiratory infection in CF patients over the age of 7 years old9. A round half of the CF population can expect to live beyond 38 years, although improvements in treatments mean a baby born today with CF could expect to live even longer6.
Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, eye care, cost-saving generic pharmaceuticals, preventive vaccines and diagnostic tools, over-the-counter and animal health products. Novartis is the only global company with leading positions in these areas. In 2011, the Group's continuing operations achieved net sales of USD 58.6 billion, while approximately USD 9.6 billion (USD 9.2 billion excluding impairment and amortization charges) was invested in R&D throughout the Group. Novartis Group companies employ approximately 124,000 full-time-equivalent associates and operate in more than 140 countries around the world. For more information, please visit http://www.novartis.co.uk
1. Colistimethate sodium and tobramycin dry powders for inhalation for treating pseudomonas lung infection in cystic fibrosis. Appraisal Consultation Document, 24 October 2012
2. TOBI Podhaler Summary of Product Characteristics http://www.medicines.org.uk/emc/medicine/24989/spc Accessed October 2012
3. Geller DE, et al. J Aer Med and Pulm Drug Deliv 2011; 24 (4); 175-182
4. Konstan MW, et al. J Cyst Fib 2011; 10: 54-61
5. Konstan MW, et al. Ped Pulm 2011; 46: 230-238
6. Cystic Fibrosis Trust: http://www.cftrust.org.uk/aboutcf/whatiscf/ Accessed October 2012
7. VanDevanter DR, et al. Respiratory Research 2010; 11: 137-144
8. LiPuma JJ, et al. Clin Microbiol Rev 2010; 23 (2): 299-323
9. UK CF Registry Annual Data Report 2010. http://www.cftrust.org.uk/aboutcf/publications/cfregistryreports/ Annual_Data_Report_2010.pdf Accessed October 2012
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Posted: October 2012