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FDA Grants Priority Review to Vertex’s 1st Triple Combo for CF

The U.S. Food and Drug Administration (FDA) has granted priority review to Vertex’s new drug application for its triple combination — elexacaftor (VX-445) plus tezacaftor and Kalydeco (ivacaftor) — for cystic fibrosis (CF) patients who cannot use the company’s other disease-modifying treatments or don’t benefit as intended. The FDA is expected to announce its final decision by March 19, 2020, according to the announced Prescription Drug User Fee Act (PDUFA) action date. Now the agency will review the data Vertex has delivered to support the approval of the triple combo therapy, which includes positive results from two clinical trials in the Phase 3 AURORA program — AURORA F/MF and F/F trials

New Intra-Nasal Imaging to Examine Airways in Cystic Fibrosis Patients

A simple, minimally invasive new tool for observing differences in the nasal airways of cystic fibrosis patients in vivo at a cellular level has recently been described in a paper published in Science Translational Medicine. The new method offers high-resolution images of the hair-like structures called cilia that are found throughout nasal airways as well as complete features of the clearance of mucus, which is weakened in people with CF, causing major morbidity. The imaging catheter, which is roughly 2-mm in diameter, uses optical coherence tomography having a resolution of about 1 micrometer—a 50th the size of a human hair—allowing scientists to probe the nasal passages of patients withou

Pharmaxis hopeful of FDA approval for cystic fibrosis inhalation medicine Bronchitol by Q1 2020

Pharmaxis Ltd (ASX:PXS) chief executive officer Gary Phillips updates Proactive Investors on the FDA approval timeframe for the company’s Bronchitol inhalation medicine used to treat adult cystic fibrosis patients. The healthcare-focused company is confident it will meet information requirements for a Q1 2020 approval. Earlier this year, the FDA Pulmonary and Allergy Drug Advisory Committee (PADAC) released a positive vote that provides a clear path to meet this approval, subject to some further information being provided, which is now being undertaken and delivered. The CEO also updated the market on the company’s deal with Boehringer for liver disease medicine, as well as its LOXL2 inhibit

Gene Editing Tool Corrects CF-causing Mutations in Patient-derived Cells, Study Shows

Using the CRISPR gene editing tool, a team of researchers was able to permanently correct two mutations in the CFTR gene — mutations that cause cystic fibrosis (CF) — in CF patient-derived cells. The researchers showed that the corrected CFTR gene was functional and working normally in mini-intestines called organoids, which had been derived from CF patients and grown in the lab. The study, “Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing,” was published in the journal Nature Communications. The gene editing tool CRISPR-Cas9 — a strategy that allows the correction of genomic defects — is considered one of the most promising approaches to correc

Powerful weapon discovered to fight antibiotic resistance in Staphylococcus aureus

Staphylococcus aureus bacteria are a major cause of serious infections that often persist despite antibiotic treatment, but scientists at the UNC School of Medicine have now discovered a way to make these bacteria much more susceptible to some common antibiotics. The scientists, in a study published in Cell Chemical Biology, found that adding molecules called rhamnolipids can make aminoglycoside antibiotics, such as tobramycin, hundreds of times more potent against S. aureus - including the strains that are otherwise very hard to kill. The rhamnolipids effectively loosen up the outer membranes of S. aureus cells so that aminoglycoside molecules can get into them more easily. "There's a great

Lung damage bacteria sniffed out by dogs in cystic fibrosis research

A new study found the animals can detect ultra-low concentrations of pseudomonas, the commonest cause of lung infection in the disease. Dogs can sniff out bacteria that is a major of cause of lung damage in people with cystic fibrosis before it takes hold, according to a study. Research found the animals can detect ultra-low concentrations of pseudomonas, the commonest cause of lung infection in the disease. Bio detection dogs have a very high level of accuracy when asked to identify bacteria associated with serious lung infections, research by Imperial College London and the Cystic Fibrosis Trust suggests. A major cause of lung damage in cystic fibrosis (CF) is infection with bacterial path

Minimally Invasive Imaging Device Shows Changes in Nasal Airways, May Allow Earlier CF Diagnosis

A minimally invasive imaging device that generates high-resolution images of nasal airways, including the cilia and mucus, allows researchers to visualize changes at the cellular level that underlie cystic fibrosis (CF) in patients. The data confirms that mucus dehydration and abnormalities of the airway’s epithelia, as well as loss of cilia — hair-like structures that have diverse motility and sensory functions — are major hallmarks of the disease. The findings of “Intranasal micro-optical coherence tomography imaging for cystic fibrosis studies” were published in the journal Science Translational Medicine. Dehydration of the airway and mucus abnormalities are thought to result in slower mu

Proteostasis Begins Dosing in Phase 2 Trial of 3 CF Investigational Therapies

The first patient has been dosed in a Phase 2 clinical trial testing Proteostasis Therapeutics‘s combinations of three investigational therapies for cystic fibrosis (CF), the company announced. The three treatments under investigation are all designed to fix the defective CFTR protein due to mutations in the CFTR gene, which is the cause of CF. The compounds under investigation are PTI-428, which is an amplifier; PTI-801, which is a corrector; and PTI-808, which is a potentiator. The therapies are being tested in double (PTI-808 and PTI-801), and triple (PTI-808, PTI-801, and PTI-428) combinations. In a previous Phase 1 study (NCT03500263), the triple combination therapy was shown to have an

Pregnancy in CF Women With Poor Lung Function Not Linked to Pulmonary Decline, Study Shows

Poorer lung function in pregnant women with cystic fibrosis (CF) is associated with a lower birth weight for offspring, and a higher likelihood of cesarean section — but not with changes in lung or metabolic parameters, new research suggests. The study, titled “Pregnancy outcome in women with cystic fibrosis and poor pulmonary function,” was published in the Journal of Cystic Fibrosis. Pregnancy can be a risky endeavor for women dealing with a chronic disease, especially for those with more severe symptoms. While some studies have suggested that pregnancy does not cause adverse effects in CF patients, there is speculation that women who have particularly poor lung function may be at an incre

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