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 —...
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...
FDA Grants Orphan Drug Status to AR-501, Potential CF Treatment for Chronic Lung Infections
AR-501, an investigational inhaled formulation of gallium citrate to treat chronic lung infections in patients with cystic fibrosis (CF),...
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...
FDA Approves Kalydeco for Treatment of 6- to 12-month-old Infants with Cystic Fibrosis
The U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) for the treatment of infants from six to 12 months old, who...
Toddlers Age 1-2 With CF Can Now Be Treated With Kalydeco in Canada
Health Canada has approved the use of Kalydeco (ivacaftor) in toddlers age 1-2 years with certain genetic mutations that prevent the...
FDA puts Translate Bio’s rare disease mRNA drug on hold
The FDA has placed a clinical hold on the IND for Translate Bio’s mRNA treatment for ornithine transcarbamylase (OTC) deficiency, a...
FDA Approves AzurRx Biopharma Request to Test MS1819-SD in CF Patients with EPI
AzurRx BioPharma is planning a multi-center Phase 2 trial to be conducted in the United States and Europe to evaluate its lead candidate...
FDA Expands Kalydeco’s Approval to 1-year-old Toddlers with Cystic Fibrosis
The U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) as the first-ever therapy to treat the underlying cause of...
Vertex Wins FDA Approval for Expanded Cystic Fibrosis Treatment
Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) shares saw a handy gain on Thursday after the U.S. Food and Drug Administration (FDA) gave the...
