Protect those with CF by quickly approving new therapies

Last week, CFRI’s executive director, Sue Landgraf, met with Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research at the United States Food and Drug Administration (FDA) in Washington DC, to encourage that the FDA expedite the review and approval of new drugs that can benefit the CF community. They discussed FDA policies and procedures around this topic and the possibility of expediting Vertex’s tezacaftor/ivacaftor combination treatment that met both Phase Three trials' primary endpoints, with statistically significant improvement in lung function and no adverse side effects for those with CF that have two copies of the F508del mutation, as well as those that have one copy of the F508del mutation and one residual CFTR mutation.

Dr. Woodcock encouraged members of our CF community to contact her to share their opinion on this issue. Let’s keep up the momentum from last week’s meeting. Thank you for writing to Dr. Woodcock today to ask that the FDA approve – as quickly as possible – new medications that have successfully met the clinical trial endpoints. Lives are depending on them.

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Janet Woodcock, M.D.

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Protect those with cystic fibrosis by quickly reviewing and approving new life-saving therapies.

Dear [Decision Maker],

Thank you for recently meeting with Sue Landgraf, CFRI's Executive Director, regarding expediting new drugs that have met their Phase Three trials with significantly statistical success and outcomes. Many thanks to the role you have played in reviewing and approving medications for the cystic fibrosis community. The FDA has approved for use some of the most effective drugs very quickly - such as ivacaftor - thus bringing relief to those in the CF community who have specific CFTR mutations.

I write today to ask you to continue your admirable work and to please review and approve the latest drugs for rare diseases that met their goals in the clinical trials, specifically Vertex's tezacaftor/ivacaftor combination treatment that met both trials' primary endpoints, with statistically significant improvement in lung function for those with CF that have two copies of the F508del mutation, as well as those that have one copy of the F508del mutation and one residual CFTR mutation. I realize that the FDA is extraordinarily busy and impacted by funding shortages, but I hope that the FDA review and approval process can be expedited once the drug application is submitted and after the 60-day application review period. So many in the CF community are in need of this latest breakthrough and simply cannot wait months for access.

There is much hope in the CF community these days, as many companies are in clinical trials for a number of promising new drugs to treat this progressive disease. The FDA has an opportunity to make a difference today in the lives of so many with CF that are waiting for a cure. While this new drug combination does not cure CF, it has the potential to be the most effective medication to date that treats the root cause of the disease in its target population.

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The needs of the CF community are often overlooked because this complex disease impacts approximately 30,000 people in the U.S. and is considered a "rare disease" by the Federal government. Approximately one in 31 Americans is believed to be a silent carrier of the mutated CF gene that leads to a defective exchange of fluids across cells, causing secretions to clog ducts and passageways in the lungs, pancreas, sinuses, and reproductive systems. The primary cause of death is respiratory failure.

As you are well aware, the treatment of this devastating disease is both time-consuming and expensive; CF has a devastating emotional and financial impact upon American families impacted by the disease. The median age of death for those with cystic fibrosis is only 30 years.

I am proud to be part of Cystic Fibrosis Research, Incorporated's (CFRI) efforts to increase access to new therapies and medications. You can make a difference in the lives of 30 million people affected by rare diseases in America, including 30,ooo with cystic fibrosis, by quickly reviewing and approving new drugs that met their clinical trial goals. Thank you for recognizing the overwhelming challenges of these life-shortening diseases and improve our access to new potentially life-saving therapies.

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[Your Address]
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