Cystic Fibrosis News Today Weekly Digest

Cystic Fibrosis Weekly Update Contents: PTI-808 and PTI-801 Combo Improves Lung Function in CF Patients, Phase 1 Data Show Santhera’s Potential CF Inhalation Therapy Favored for Orphan Drug Status in EU PTI-808 and PTI-801 Combo Improves Lung Function in CF Patients, Phase 1 Data Show Oct 24, 2018 07:00 am | Alice Melão Treatment with a combo of Proteostasis Therapeutics’ investigational CFTR modulator therapies — PTI-808, a  potentiator, plus PTI-801, a corrector — can promote significant improvements in lung function and reduction of sweat chloride in patients with cystic fibrosis (CF), Phase 1 data show. So far in the Phase 1 trial (NCT03140527), 21 patients carrying two copies of the F508del mutation […] The post PTI-808 and PTI-801 Combo Improves Lung Function in CF Patients, Phase 1 Data Show appeared first on Cystic Fibrosis News Today. Read More Santhera’s Potential CF Inhalation Therapy Favored for Orphan Drug Status in EU Oct 23, 2018 07:00 am | Ana Pena Santhera Pharmaceuticals’ investigative inhalation therapy for cystic fibrosis (CF), POL6014, has been favored for orphan drug designation within the European Union. A recommendation supporting the designation, which offers financial and regulatory incentives to develop treatments for rare diseases, was made by  Committee for Orphan Medicinal Products (COMP), a branch of the European Medicines Agency (EMA). The positive opinion is […] The post Santhera’s Potential CF Inhalation Therapy Favored for Orphan Drug Status in EUappeared first on Cystic Fibrosis News Today. Read More   Recent News Compliance Is One of Our Toughest Mental and Physical Battles FDA Approves AzurRx Biopharma Request to Test MS1819-SD in CF Patients with EPI #NACFC2018 — CFF Announces $100 Million ‘Infection Research Initiative’ We Need to Widen Inclusion Criteria for Cystic Fibrosis Clinical Trials Orkambi May Be More Effective in CF Patients with Inflamed Airways, Study Suggests