News

Vertex Pharmaceuticals announced it plans to request by year’s end that the label for Symdeko (tezacaftor/ivacaftor and ivacaftor), its recently approved disease-modifying therapy for cystic fibrosis (CF), be expanded to include children as young as 6 years old, the company announced in a recent investor report. It also plans to…

A Phase 1b/2a, dose-escalating clinical trial evaluating the effects of POL6014 in patients with cystic fibrosis (CF) was recently launched, according to Santhera Pharmaceuticals, developer of the investigational therapy.  The Phase 1b/2a…

Adding the investigational therapy VX-445 to tezacaftor (VX-661) and Kalydeco (ivacaftor) led to a roughly 10 percent improvement in the lung function of cystic fibrosis (CF) patients with F508del mutations in the CFTR gene in a proof-of-concept Phase 2 clinical trial. …

With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Creditand affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate, or…

Once-daily acebilustat, an investigational oral therapy for cystic fibrosis (CF) under development by Celtaxsys, reduces the frequency and delays the onset of pulmonary exacerbations in CF patients, results from a Phase 2 study show. Patients with better lung function…

Treatment with lenabasum can effectively prevent inflammation and induce pro-resolving signals in airway immune cells from patients with cystic fibrosis (CF), results from a preclinical study show. Lenabasum’s more recent preclinical data was discussed at the 32nd North American Cystic Fibrosis Conference, in a poster presentation titled “…

Vertex Pharmaceuticals’ VX-659 or VX-445 in combination with Kalydeco (ivacaftor) and tezacaftor (VX-661) can improve the lung function of patients with cystic fibrosis (CF) with one or two copies of the CFTR F508del mutation, Phase 2 data shows. Updated results of the studies were discussed…

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…

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 …

AzurRx BioPharma is planning a multi-center Phase 2 trial to be conducted in the United States and Europe to evaluate its lead candidate MS1819-SD as a treatment for patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). This announcement follows the approval by the U.S. Food and…