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Cystic Fibrosis Care Center


Ataluren Trials

Both the adult and PEDS CF Centers will participate in the Ataluren trials to assess efficacy of this medication in CF patients with Nonsense mutations. Ask your Doctor if you are eligible - we hope to begin enrollment in May 2015.

OneCenter Project

The adult & PEDS teams are still working on the OneCenter research project sponsored by the CFF. We continue to hold evening educational sessions for parents of children in the adolescent age group about the transition process. Ask your Center staff about the dates and opportunities to participate.

CF Rise Educational Initiative

Both CF teams have been trained in the CF Rise which is the CFF endorsed & Gilead sponsored organized approach to education in CF- targeting the adolescent population. Since the adolescent population is known to skip meds, miss medication doses and this makes it easier for them to get sick, lose weight & very importantly, lose lung function, this CF Center agrees that this is an important group to target for better education & understanding of their CF diagnosis, care and medications, and treatments. We are actively rolling out the use of this program. Ask your CF team members, how you may participate in this wonderful educational initiative.

Confusion about payment for Cayston and its nebulizer (Altera)

There is confusion about Medicare coverage for Cayston and the Altera nebulizer. In December, the Centers for Medicare & Medicaid Services (CMS) made the determination that the drug would be covered under Medicare Part D, a decision which excludes the Altera nebulizer from Medicare coverage. The CF Foundation (CFF) met with CMS officials numerous times in the latter half of 2010 to articulate its position that CF patients expect and require timely and affordable access to both Cayston and the Altera nebulizer. CFF continues to work with CMS to determine a pathway which enables Medicare beneficiaries with CF patients to obtain both the medication and the required delivery device, without undue financial burden including high out-of-pocket costs.

FDA approves VERTEX 770 drug : Kalydeco®, (Ivacaftor) in patients with CF and the G551D mutation

Kalydeco™ (generic name, ivacaftor; previously known as VX-770) is a new oral medication for the treatment of cystic fibrosis, approved by the U.S. Food and Drug Administration (FDA) in January 2012. It is the first drug available that targets the underlying cause of CF — a faulty gene and its protein product, CFTR. The FDA approved Kalydeco (kuh-LYE-deh-koh) for people ages 6 and older with the G551D mutation of CF.

Click here for more information.


Pharmaceutical company Pharmaxis today announced the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the granting of a Marketing Authorization for Bronchitol, clearing the way for the product to be used in Europe "for the treatment of cystic fibrosis in adults as an add-on therapy to best standard of care. Pharmaxis expects the European Commission to confirm this opinion and grant the Marketing Authorization for Bronchitol in January 2012. SOURCE: Pharmaxis Ltd, Sydney, Australia Bronchitol has been developed to help clear mucus (a major source of lung infections), improve lung function and reduce exacerbations in patients with cystic fibrosis. Bronchitol is a proprietary formulation of mannitol administered as a dry powder in a convenient hand-held inhaler. Bronchitol hydrates the lungs, helps restore normal lung clearance, and allows patients to clear mucus more effectively. Clinical studies have shown Bronchitol to be safe, effective, and well tolerated in treating patients cystic fibrosis.

Two Potential Drugs for CF Show Disappointing Results

Denufosol, a drug being tested in mild CF lung disease does not appear to be effective, according to the newly-released results of the phase III TIGER-2 trial. Denufosol is a novel channel regulator that increases the movement of the salt chloride while inhibiting the movement of sodium; this would make the very thick CF mucus more hydrated and less sticky. TIGER-2 (Transport of Ions to Generate Epithelial Rehydration) was a phase III study of CF patients with mild lung disease (baseline FEV1 >75% to < 110% predicted). The study was a 48 weeks study that enrolled a total of 466 people with CF. "The analysis of the primary endpoint, key secondary endpoints and select subgroup populations in TIGER-2 indicates an absence of meaningful treatment benefit in this patient population," said Dr. Charles Johnson, Chief Medical Officer, during the teleconference. Dr. Johnson is the former director of the CF Center at Washington University, St. Louis.

An advisory panel of the U.S. Food & Drug Administration (FDA) said it did not have sufficient clinical trial data to recommend approval of liprotamase, a non-porcine pancreatic enzyme therapy for the treatment of pancreatic enzyme insufficiency in CF. The FDA panel was not convinced that liprotamase was any better than current FDA-approved porcine-derived pancreatic enzyme products.
Source: Cystic Fibrosis News Network. January 13, 2011

ARIKACE™ Demonstrates Sustained Benefit

Transave, Inc., today reported interim results from a multi-cycle Phase II open label clinical trial in 49 CF patients on its lead investigational drug, ARIKACE™ (liposomal amikacin for inhalation). The data indicated that ARIKACE™, delivered once daily for 28 consecutive days followed by 56 days off-treatment for four cycles demonstrated statistically significant improvement in lung function that was sustained during the 56 days off study drug. ARIKACE™ was well-tolerated during the four cycles. According to the company, preparations are underway to launch Phase III studies. ARIKACE demonstrated statistically significant reduction in Pseudomonas density which was sustained of the treatment period of four cycles for 12 months.
Source: ARIKACE™ Demonstrates Sustained Benefit in the Treatment of Cystic Fibrosis Patients Who Have PSEUDOMONAS Lung Infections

ARIKACE® trial on hold

The U.S. Food and Drug Administration (FDA) has notified Insmed, the manufacturers of ARIKACE®, that the agency has placed a clinical hold on Insmed's phase 3 clinical trials for ARIKACE® (liposomal amikacin for inhalation) in Cystic Fibrosis (CF) patients with Pseudomonas lung infections and patients with non-tuberculous mycobacterial (NTM) lung disease. A clinical hold is a notification issued by FDA to the sponsor to delay a proposed clinical trial or suspend an ongoing clinical trial. The Company has been informed by FDA that this decision was based on an initial review of the interim results of a long-term rat inhalation carcinogenicity study, recently reported to the agency by Insmed, with ARIKACE. In this study, rats received daily doses of ARIKACE by inhalation for up to two years.

PARI Pharma's Altera Delivers Gilead's Cayston in Head-to-Head Study with Tobramycin Inhalation Solution

Phase III clinical trials of Cayston delivered by the Altera Nebulizer System versus tobramycin inhalation solution in CF patients with Pseudomonas aeruginosa delivered a mean increase in FEV (1) percent predicted from baseline to day 28 of 8.35 percent, meeting statistical superiority for the Cayston/Altera combination over the tobramycin solution. Safety results were similar across both arms of the study of 268 randomized patients, with lower incidence of cough in patients receiving Cayston. Altera delivers Cayston in two to three minutes - "a significant reduction in delivery time compared to other inhalation therapies," according to the president of PARI Pharma GmbH.
Source: PARI Pharma's Altera Delivers Gilead's Cayston in Head-to-Head Study with Tobramycin Inhalation Solution

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