Cystic Fibrosis In The News
Learn about the latest CF medical advances, treatments and pharmaceuticals.
FDA Approves SYMDEKO (tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older with Certain Mutations in the CFTR Gene
February 12, 2018 — Vertex announced that the U.S. FDA approved SYMDEKO (tezacaftor/ivacaftor and ivacaftor) for treating the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the CFTR gene or who have at least one mutation that is responsive to tezacaftor/ivacaftor. SYMDEKO is Vertex’s third medicine approved to treat the underlying cause of CF. Vertex is ready to launch SYMDEKO and will begin shipping it to pharmacies in the United States this week.
January 31, 2018 — Vertex announced the selection of two next-generation correctors, VX-659 and VX-445, to advance into Phase 3 development as part of two different triple combination regimens for people with cystic fibrosis (CF). This decision was based on positive initial Phase 2 data. Regulatory discussions are ongoing to finalize the design of Phase 3 programs. Upon completion of these discussions, Vertex plans to initiate the first Phase 3 triple combination program in the first half of 2018 to evaluate VX-659 in triple combination, and a second Phase 3 program in mid-2018 to evaluate VX-445 in triple combination.
This is 29 year old Kristina Caudill. She has cystic fibrosis. I (Laura Bonnell) met with her Monday to talk about how many times her managed medicaid plan denied her request to get on Orkambi, an approved drug for CF by the FDA (July). Caudill and her CF doctor will try for a 5th time to get her on the medication. The state medicaid panel met Tuesday and after months of people advocating to get the drug approved, the panel made a recommendation to have it listed as an acceptable drug. The state Director, Chris Priest (approachable, media accessible and with a desire to help) says it won’t be a long wait but couldn’t say exactly when it will be in the hands of the approximately 30 people who don’t have access to the meds. These are people on average whose yearly income is below $30,000. I also talked to Governor Snyder today and he said the new drugs coming out for people with CF and other diseases is exciting. As Priest stated, and the Governor agreed, they have to listen to the recommendation of the panel (and they approved it), pay attention to costs and talk with legislators.
Listen to the audio of Laura and Kristina’s talk:
The U.S. Food and Drug Administration said Thursday it had approved the use of Vertex Pharmaceuticals’ Orkambi, a drug aimed to treat patients with cystic fibrosis, on patients who are at least 12 years old.
The company’s shares were temporarily halted, up 3.73 percent at $130.90, during late-morning trading for pending news ahead of the announcement at 11:47 a.m. ET. Trading started again around 3 p.m. ET, and Vertex closed the day slightly higher than its halt price.
“…The approval of ORKAMBI represents a fundamental change in the treatment of the most common form of CF, marking significant progress for us and for the entire CF community,” Dr. Jeffrey Leiden, Vertex’s chairman, president and CEO, said in a statement.
With the approval, Orkambi will be available to about 8,500 patients in the United States, according to Reuters.
Cystic fibrosis is caused by a defective gene that disrupts the function of the lungs and digestive system, producing a build-up of thick, sticky mucus leading to inflammation and recurrent bacterial infections..
WWJ Newsradio 950’s Marie Osborne reported on The Bonnell Foundation’s holiday fundraiser. Lois Teicher, sculptor and Grandmother to Molly and Emily Bonnell (who both have cystic fibrosis and are the daughters of our Founder, Laura Bonnell) has designed holiday cards for Christmas and Hanukkah, which are currently for sale.
Some health concerns if your child is taking Kalydeco. Read more. Click here: http://www.fda.gov/Drugs/DrugSafety/ucm316693.htm Please check back or email us with any questions. firstname.lastname@example.org
UMHS one of six Michigan facilities linked to New Hampshire hepatitis C investigation
The Michigan Department of Community Health has been investigating the employment history of a Hepatitis C-positive health care worker who was recently arrested in New Hampshire for allegedly obtaining injectable narcotics at hospitals there, and infecting patients with the Hepatitis C virus through used syringes. To read more please click below:
Check out all the stories about fabulous people staying healthy with cystic fibrosis.
Health and Human Services (HHS) Secretary Kathleen Sebelius today announced $4.9 million in Affordable Care Act funding to support Family-to-Family Health Information Centers, primarily non-profit organizations run by and for families with children with special health care needs.
“These centers provide the information that families need to make health care decisions that are right for their children,” Secretary Sebelius said. “Family-to-Family Health Information Centers are a good investment, and have a measurable and positive impact on families, and communities.”