ICYMI: FDA Approves New Treatment for Cystic Fibrosis

On February 13th, the U.S. FDA announced the approval of Symdeko, a novel combination of two previously used agents to treat cystic fibrosis (CF). Symdeko combines a drug that has been on the market for a while, Kalydeco with a new drug called tezacaftor. Kalydeo was used alone before.

Boston-based Vertex Pharmaceuticals Inc. is the company that developed this new treatment. Vertex markets three treatments for CF: Kalydeco (launched in 2012), Orkambi (launched in 2015) and the new Symdeko (2018). Symdeko is the next step in Vertex’s eventual plan to use a triple-combination treatment for the rare genetic lung disease that inhibits a patient’s ability to breathe and is also a source for many lung infections.

All three of these treatments deal with the actual disease rather than just treating symptoms, but current therapies only can be used by approximately 45% of patients with CF. Researchers believe that the triple-combination treatment, when approved and released will be able to treat about 90% of patients with CF.

“This approval is an important milestone in our journey to treat every person with CF, and we remain committed to urgently advancing our efforts to develop new medicines that treat the underlying cause of CF””
— Jeffrey Leiden, M.D., Ph.D., Vertex’s CEO

In Symdeko’s clinical studies, the combined therapy proved a better therapy than Orkambi in lung function improvements. Additionally, Symdeko doesn’t have requirements for monitoring blood pressure in CF patients and doesn’t include a warning on respiratory events like Orkambi does.

The Phase 3 clinical trials for Symdeko were published in the New England Journal of Medicine in November of last year. The trials involved 750 CF patients and followed them for up to 48 weeks. Patients showed a statistically significant improvement in lung effectiveness and improved drug safety.

Critics of the treatment will most likely focus on the yearly price tag for treatment which will be around $292,000 per year. Vertex has launched a guidance and patient support program to help doctors, patients and their families receive education on insurance issues, co-payment programs and other financial assistance.

According to the Cystic Fibrosis Foundation Patient Registry, there are 1,000 new cases of CF diagnosed in the United States each year. There are approximately 30,000 people living with CF in the United States. Most of these patients were diagnosed before the age of 2.

There is no cure for CF, but the advancements in treatment have made a huge difference since the 1950’s. Back then, most children diagnosed with CF never made it to elementary school. Today the median survival age is 40 years old. Many patients who are building adult lives, by working, graduating from college and starting families, would have never had that chance not that long ago.

Donald Blake

Donald Blake

Donald Blake has a BS in Communication Studies. He has a lengthy tenure in the healthcare, media and education fields. He is dedicated to improving the lives of those with rare diseases through his knowledge of healthcare and communications.

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