Author Archives: Marisa Wexler MS

Phase 1 Study of KB407 Green-lighted in Australia

A research ethics committee in Australia has given Krystal Biotech the go-ahead to start a Phase 1 clinical study of KB407, the company’s investigational inhaled gene therapy for cystic fibrosis (CF). Enrollment for the trial is expected to start soon, according to Krystal, following approval from the…

Trikafta Now Covered in 2 More Canadian Provinces

The publicly funded drug access programs in the Canadian provinces of Alberta and Saskatchewan are now covering the triple-combination therapy Trikafta for eligible patients with cystic fibrosis (CF). These provinces join Ontario as the first to confirm coverage of Trikafta in Canada. “It’s a relief to know that…

Patients in Ontario Can Now Access Trikafta Through Public Plan

People with cystic fibrosis (CF) in Ontario, Canada, will now have access to the triple-combination therapy Trikafta through the province’s publicly funded drug program. “Our government has taken urgent action to ensure all cystic fibrosis patients will have more timely access to the effective and lifechanging treatments they need,”…

Adults With CF Can Connect at BreatheCon

A two-day virtual event called BreatheCon will offer adults with cystic fibrosis (CF) an opportunity to connect and share their experiences through open and honest dialogue. Registration for the Sept. 24–25 event, which is hosted by the Cystic Fibrosis Foundation, is free online. “CF can be extremely…

ELX-02, Potential CF Nonsense Mutation Therapy, on FDA Fast Track

The U.S. Food and Drug Administration (FDA) has given fast track designation to ELX-02, an investigational treatment for people with cystic fibrosis (CF) caused by nonsense mutations. This FDA designation aims to speed the development and regulatory review of potential treatments for serious or life-threatening diseases with an unmet medical…

Relizorb Will Be Covered by Medicare

The Centers for Medicare and Medicaid Services (CMS) has published a local coverage determination confirming that Relizorb — an enzyme cartridge to help in digestion for cystic fibrosis (CF) patients receiving their nutrition through enteral feeding — will be covered by Medicare.