Patients & Caregivers
New Designation Accelerates Development of Antifungal Drug
Pulmatrix's Pulmazole (PUR1900), designed to fight pulmonary fungal infections, has been designated as a Qualified Infectious Disease Product by the FDA.
PBS Documentary Gene Doctors Explores Rare Diseases
The Gene Doctors
is available. Film explores the scientific breakthroughs that are transforming rare disease patients
Total Hospital Costs for Cystic Fibrosis Patients in U.S. Cost $1.1 Billion per Year
As CF patients are living longer, their hospital costs are also increasing. Costs were $500 million in 2003 vs $1.19 billion in 2013.
FDA Accepts New Drug Application for Treatment of Rare Lung Condition
Aradigm announced the FDA acceptance of its NDA for Linhaliq to treat patients with non-cystic fibrosis bronchiectasis with chronic infections from P. aeruginosa.
Summary of the Clinical Data Of Subcutaneous C1-Inhibitor For Prophylaxis of Hereditary Angioedema
Administration of CSL830 twice weekly at doses 40 IU per kilogram or 60 IU per kilogram has a significant preventive effect
SteadyMed Received a Refuse to File Letter for Their PAH Drug
The company is optimistic it can meet the requirements asked by the FDA without the need for more studies.
New Combination Therapy for Cystic Fibrosis Being Reviewed by FDA and EMA
Vertex announced that the FDA and EMA have accepted applications for tezacaftor/ivacaftor combination treatment in a subset of patients with cystic fibrosis.
Treatment Development for Respiratory Syncytial Virus Discontinued
Regeneron's Suptavumab (REGN2222) in respiratory syncytial virus did not meet its endpoint, and the company will discontinue development of the drug.
Kadmon Granted FDA Orphan Drug Designation for Rare Lung Cancer Treatment
The FDA granted orphan drug designation to tesevatinib for treatment of non-small cell lung cancer with epithelial growth factor receptor-activating mutations.
Aradigm Submits NDA for Linhaliq (ciprofloxacin) to Treat Bronchiectasis
Aradigm has submitted its NDA for Linhaliq for the treatment of non-cystic fibrosis bronchiectasis (NCFBE) due to P. aeruginosa.
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