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FDA Approves Symdeko for Treatment of CF Children, Ages 6 to 11, with Certain CFTR Mutations

The U.S. Food and Drug Administration (FDA) has approved Symdeko (tezacaftor/ivacaftor and ivacaftor) tablets for the treatment of children, ages 6 to 11 years, with cystic fibrosis (CF) who have certain mutations that respond to treatment, its developer, Vertex Pharmaceuticals, has announced. In order to be eligible for the treatment, children must carry either two copies of the F508 deletion — the most common genetic mutation associated with CF — or have at least one mutation in the CFTR gene that responds to treatment with Symdeko. Symdeko, known as Symkevi in Europe, contains a combination of tezacaftor (VX-661) and ivacaftor (sold by Vertex under the brand name Kalydeco). The combo ther

‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy

People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the rare-disease community help them decide what policies to adopt? Sanda Courbier, senior manager of the Rare Barometer Program. (Photo courtesy of Eurordis) One way is to survey patients and caregivers about their needs, then share the results with officials so their decisions can be based on facts rather than conjecture. For the last three years, Eurordis-Rare Diseases Europe— a Paris-based alliance of

Eluforsen Found to be Safe for Adults with Cystic Fibrosis Caused by F508del Mutation, Study Reports

The experimental treatment eluforsen is safe for adults with cystic fibrosis(CF) caused by a mutation called F508del, a new study reports, supporting further investigation and development of the treatment. The study, titled “Antisense oligonucleotide eluforsen is safe and improves respiratory symptoms in F508DEL cystic fibrosis,” was published in the Journal of Cystic Fibrosis. CF is caused by mutations in the CFTR gene. F508del is the most common mutation in this gene; in fact, nearly 90% of CF patients have at least one CFTR copy with this mutation, and around half of those patients have two copies with the same mutation (of note, every person has two copies of the gene, one inherited from

Murepavadin Antibiotic Well-tolerated by Animals Infected with Pseudomonas Aeruginosa, Preclinical D

Murepavadin (POL7080), Polyphor‘s lead investigational antibiotic targeting Gram-negative bacteria, behaved as expected and was well-tolerated when administered to mice harboring a lung infection caused by Pseudomonas aeruginosa, data from a preclinical study show. Of note, P. aeruginosa is a bacteria that often infects the lungs of patients with cystic fibrosis (CF). The findings were presented in an oral presentation, titled “Pharmacokinetics and pharmacodynamics of murepavadin (POL7080) in neutropenic lung infection models when evaluated by aerosol administration,” at the 42ndEuropean Cystic Fibrosis Conference, June 5–8, 2019, held in Liverpool, U.K. “Respiratory infections, especially t

EffRx Pharmaceuticals Signs Exclusive License Agreement with Pharmaxis for the Registration and Comm

EffRx Pharmaceuticals SA, a commercial-stage company that commercializes niche and orphan medicines in Switzerland and Europe, today announced it has entered into an exclusive license agreement with Pharmaxis Ltd, a listed pharmaceutical research company in Australia, for the registration and commercialization of Bronchitol® (mannitol) for cystic fibrosis in Switzerland. Under the terms of the agreement, EffRx will take responsibility for registering, obtaining pricing and reimbursement as well as commercializing Bronchitol® in Switzerland. Bronchitol® works by rehydrating the airway/lung surface and promoting a productive cough, hence improving mucus clearance and lung function. The product

Slammed by Side Effects: The Downside of Antibiotics

Everyone with cystic fibrosis knows that feeling. Sometimes, it creeps up on you slowly. Other times, it hits right away, like a truck speeding down the wrong side of the highway. That feeling is, of course, caused by side effects from antibiotics. As I sit here, going into week three on orals, it’s something I find myself contemplating quite a bit. How do you deal with it and still maintain a relatively normal life? There isn’t a magic, easy answer, partly because symptoms can vary wildly. An antibiotic might make one person nauseated, while it makes another exhausted. An unlucky third person will experience both, and something else to boot. My answers may not be broadly applicable, but hop

New study confirms vaping is not safer than smoking- it obstructs mucus clearance

Research published in the American Thoracic Society’s American Journal of Respiratory and Critical Care Medicine confirms that E-cigarette vaping with nicotine can obstruct mucus clearance from airways. The new study draws attention to the fact that when human airway cells are exposed to e-cigarette vapor with nicotine, mucus or phlegm moves slowly across the surface. Team of researchers from the University of Miami, University of Kansas and Mt. Sinai Medical Center in Miami Beach have recognized this phenomenon as “mucociliary dysfunction.” The team also compared the results with vivo sheep, whose airways, being similar to humans react in the same way when exposed to e-cigarette vapor. Muco

Trial of Nebulized CF Candidate Therapy Completes Single-ascending Dosing

The RESTORE-CF clinical trial investigating Translate Bio’s candidate therapy MRT5005 has completed the single-ascending dose regimen in patients with cystic fibrosis (CF), the company announced. The ongoing Phase 1/2 trial (NCT03375047) is testing the safety and efficacy of single and multiple escalating doses of nebulized MRT5005, compared with each other and a placebo, in 32 adults with CF who have class I or II genetic mutations in the CFTR gene (the gene that is defective in CF patients). The trial, being conducted in collaboration with the Cystic Fibrosis Foundation Therapeutics Development Network, is now moving into the multiple ascending dose phase. “We are excited to share that we

Specific Peptide May Lower Antibiotic Resistance of P. aeruginosa Bacteria, Study Reports

Expression of a peptide called RpoN* in antibiotic-resistant Pseudomonas aeruginosa isolates from cystic fibrosis (CF) patients made the bacteria more susceptible to antibiotics and reduced virulence in a roundworm infection model, a study has found. The study, “Blocking RpoN reduces virulence of Pseudomonas aeruginosaisolated from cystic fibrosis patients and increases antibiotic sensitivity in a laboratory strain,” was published in the journal Nature Scientific Reports. Pseudomonas aeruginosa is an opportunistic bacterial strain, and the most common pathogen found in CF patients. The ability of P. aeruginosa to produce biofilm (layers of bacteria sticking to surfaces and other bacteria) ca

Life-saving designer ‘NINJA virus’ hailed as medical breakthrough in curing infection

A VIRUS cocktail designed to seek and destroy bacteria has cleared a 15-year-old lung transplant patient of an antibiotic-resistant infection, in a potential world-breakthrough. The young girl, who has cystic fibrosis — a genetic condition which causes mucus to build up in the lungs — had received a life-saving double lung transplant at Great Ormond Street Hospital in London. Weeks after the surgery, doctors spotted telltale signs of an infection, including bumps poking through her skin and redness around the incision sites. Antibiotics could not halt the spread of the antibiotic-resistant infection which had reached her liver — and so in a world-first, doctors turned to bacteria-slaying vir

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