9-Year Old Cystic Fibrosis Patient Talks with Bennet About "Miracle Drug"?

Drug receiving expedited approval to treat more patients thanks to Bennet's bipartisan "breakthrough therapy"? provision

Yesterday, Senator Michael Bennet visited 9-year old Annelise Heinicke at her elementary school classroom to talk to her about living with cystic fibrosis (CF), the drug that is improving her quality of life, and a new FDA designation that will allow it to help more people more quickly.

Annelise was diagnosed with a rare form of CF as an infant.  In 2012 she began taking Kalydeco after it was approved by the Food and Drug Administration (FDA) to treat a small percentage (4 percent) of CF patients with a specific genetic mutation.  The success of this drug on patients like Annelise has led to the creation of new drug treatments pairing Kalydeco with other treatments as well as using Kalydeco on patients with other gene mutations.  These new therapies could treat as many at 65 percent of patients who have CF.

These new therapies received a “breakthrough therapy” designation from the FDA, which accelerates the approval of drugs that show early, dramatically positive results.  These therapies are now on a faster track to full FDA approval so that they can quickly get to patients that need them.  This new “breakthrough therapy” was written into law by Colorado U.S. Senator Michael Bennet, along with Senator Orrin Hatch and Richard Burr.  They included it in the FDA Safety and Innovation Act that passed into law in 2012.

“These types of game-changing drugs hold the potential to help thousands of people like Annelise live longer, healthier lives.  Too often they are mired in in an unnecessarily burdensome approval process, ” Bennet said.  “The new designation is allowing these lifesaving therapies to safely and more quickly get into the hands of patients, where they can make a meaningful difference.”

Since undergoing the new treatment, Annelise’s mother Jen Heinicke has called Kalydeco a “miracle drug” and reported positive differences in Annelise’s energy and endurance.

“Kalydeco is a game-changer for Annelise and our entire family,” Jen Heinicke said. “We are eternally grateful to the policy makers, like Senator Bennet, as well as the researchers and health care providers that are working so hard on so many levels to find a cure for the other 96 percent of CF patients.”

Background:

  • The average FDA approval process can take more than a decade.
  • Since the passage of Bennet’s provision more than 40 drugs have received this breakthrough therapy designation.
  • In addition to CF, breakthrough therapies have been approved to treat several illnesses, including hepatitis C and leukemia.
  • CF is a life-threatening genetic disorder that affects roughly 70,000 people worldwide and approximately 30,000 in the United States, including nearly 1,000 Coloradans.