Breakthrough therapy for cystic fibrosis approved by FDA
Trikafta is the first approved treatment that is effective for patients 12 years and older with the most common cystic fibrosis mutation.
Trikafta (elexacaftor/ivacaftor/tezacaftor), the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation has been approved by the US Food and Drug Administration (FDA).
The therapy is approved for patients 12 years and older with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is estimated to represent 90 percent of the cystic fibrosis population.
"At the FDA, we're consistently looking for ways to help speed the development of new therapies for complex diseases, while maintaining our high standards of review," said acting FDA Commissioner Ned Sharpless, MD. "Today's landmark approval is a testament to these efforts, making a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options and giving others in the cystic fibrosis community access to additional effective therapy."
Trikafta is a combination of three drugs that target the defective CFTR protein, helping the protein made by the CFTR gene mutation function more effectively. Trikafta is the first approved treatment that is effective for cystic fibrosis patients 12 years and older with at least one F508del mutation.
The efficacy of Trikafta in patients with cystic fibrosis aged 12 years and older was demonstrated in two trials - the first a 24-week, randomised, double-blind, placebo-controlled trial in 403 patients who had an F508del mutation and a mutation on the second allele that results in either no CFTR protein or a CFTR protein that is not responsive to ivacaftor or tezacaftor/ivacaftor alone.
The second was a four-week, randomised, double-blind, active-controlled trial in 107 patients who had two identical F508del mutations.
In each trial, the primary analysis looked at increases in the percent predicted forced expiratory volume in one second, known as ppFEV1, which is an established marker of cystic fibrosis lung disease progression. Trikafta increased the ppFEV1 in both trials.
In the first trial, treatment with Trikafta also resulted in improvements in sweat chloride, number of pulmonary exacerbations (worsening respiratory symptoms and lung function) and body mass index compared to the placebo.
By Mettler-Toledo GmbH
Yesterday, the FDA approved the use of Trikafta, a triple combination therapy, for treatment of the most common cystic fibrosis (CF) mutation. This is the first medication to be approved for use among such a
On Monday, the U.S. Food and Drug Administration today approved a new therapy to treat cystic fibrosis. Trikafta is described as the first triple combination therapy available to treat patients with the most common cystic
Vertex has scored an earlier than expected FDA approval of its cystic fibrosis triple therapy Trikafta, and has priced it at $311,000 for a year of treatment. The combination regimen of VX-445
Boston-based Vertex Pharmaceuticals Inc. has achieved one of the biggest feats to date for cystic fibrosis patients - a treatment that could benefit 90 percent of patients with the rare lung disease. The FDA approved a
The triple combo era in cystic fibrosis is here thanks to a Monday Vertex approval-and it's here earlier than industry watchers expected. A lot earlier. Five months ahead of schedule-and just three months after the company
The US Food and Drug Administration (FDA) has approved the first triple combination therapy to treat patients with the most common cystic fibrosis (CF) mutation. Vertex Pharmaceuticals' Trikafta combines elexacaftor, ivacaftor, and tezacaftor and is approved
By Manojna Maddipatla and Carl O'Donnell (Reuters) - Vertex Pharmaceuticals Inc has priced its three-drug combination for cystic fibrosis (CF) at $311,503 per year, after winning early U.S. approval on Monday. Shares of the drugmaker,
More News in Business
Roku has announced that it has entered into an agreement to acquire Boston-based dataxu, an ad DSP that enables marketers to plan and buy video ad campaigns. dataxu provides marketers with an automated bidding and
A man peeks out his apartment's peephole and discovers a hallway full of lurkers staring at his closed door. Another man looks up from his phone on a street corner to see a crowd of
NEW YORK, Oct. 22, 2019 (GLOBE NEWSWIRE) -- Halper Sadeh LLP, a global investor rights law firm, is investigating whether the sale of Cision Ltd. (NYSE: CISN) to an affiliate of Platinum Equity ("Platinum Equity")
By Patrick Thomas Manufacturing activity across mid-Atlantic states strengthened in October, according to a report from the Federal Reserve Bank of Richmond, as indexes for shipments, the volume of new orders and employment rose. The Federal Reserve
Roku is beefing up its advertising business with the acquisition of Boston-based dataxu, a demand-side platform that will allow marketers to plan, buy and optimize their video ad campaigns that run on Roku's devices