Research Update

Orkambi Coverage Approval
The New York Medicaid Drug Utilization Review (DUR) board met November 19th to review Orkambi. The New York DUR Board makes determinations to ensure prescriptions for outpatient drugs are appropriate, medically necessary and not likely to result in adverse medical consequences. The Board unanimously approved continued coverage aligned with the FDA label indications (age 12 or older, two copies of F508del mutation). This decision is in line with CFF's position for state Medicaid programs to cover a drug according to the FDA recommendation without additional restrictions or requirement.

FDA Approves New Device for Improving Absorption of Essential Fats in Tube Feeding
People with CF who have difficulty maintaining a healthy weight often rely on supplemental nutrition through a special feeding tube, known as a gastronomy tube (or G-tube), to meet their nutritional needs. The U.S. Food and Drug Administration has approved a device that helps break down beneficial fats in supplemental nutrition provided via G-tubes.

Orkambi Being Studied in Children
A Phase 3 clinical trial underway could potentially expand the number of people with CF in the United States who can take the lumacaftor/ivacaftor combination drug (Orkambi™). Vertex Pharmaceuticals Inc. is conducting a clinical trial that, if successful, would make the drug available to children ages 6 to 11 with two copies of the F508del gene mutation.

Second Combination Drug in Phase 3 Studies
Four Phase 3 clinical trials are testing ivacaftor (Kalydeco™) in combination with another potential therapy, VX-661. Like lumacaftor, VX-661 helps the defective CFTR protein fold correctly so that it can move to its proper place at the cell surface. Three trials will study the combination treatment in people ages 12 and older who have one copy of the F508del mutation and a second CF mutation that is either a residual function mutation, gating mutation or minimal function mutation, while another trial has enrolled people with two copies of F508del.  

Trial Targeting RNA
ProQR Therapeutics has started a Phase 1 clinical trial to test the safety of a potential messenger RNA therapy for people with CF who have two copies of the F508del mutation. The potential therapy aims to repair messenger RNA so that a normal CFTR protein can be made.  

If you would like to learn more, please visit CFF.org for further information.