News

Symkevi (tezacaftor/ivacaftor combo), by Vertex Pharmaceuticals, has been approved in the European Union for patients with cystic fibrosis (CF) ages 12 or older who carry F508del mutations. Importantly, the European Commission’s decision to grant marketing authorization makes Symkevi the first treatment in Europe for CF patients with only one F508del copy plus another mutation that confers minimal…

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 …