Is the New FH Drug Inspired by Star Wars? Who Cares, It Works

Standing in front of a crowd and talking about your HoFH can be intimidating. Imagine if that crowd was a panel of doctors and scientists with the US Food and Drug Administration (FDA).

For Colleen McCready, that was reality one day in June. A passionate mother, she stood before EMDAC, the FDA Endocrinologic and Metabolic Drugs Advisory Committee, and spoke about her long journey with homozygous familial hypercholesterolemia (HoFH)

McCready details when she finally found relief through an emerging medicine after her LDL levels hit over the 300s! Source: The FH Foundation.

Like many others in the familial hypercholesterolemia (FH) community, when a routine physical showed that her LDL cholesterol level was in the 300s, McCready tried every therapy her doctor recommended. However, no matter what combination of therapies she tried, her body just could not tolerate them.

Unfortunately, McCready isn’t alone. Other members of the community were there to share similar experiences, and those who could not attend sent letters and emails to make sure their voices were heard. The message was clear: The FH community needs more options for treatment.

As McCready told the committee, “These new drugs represent hope to myself and many, many others. I don’t have a lot of options.”

The new drugs that sound like they should belong in a Star Wars movie, PCSK9 inhibitors, are being reviewed by the committee to become a part of a whole new class of hypercholesterolemia treatment:

  • PCSK9 inhibitors work with traditional statins
  • Efficacy data shows they may possibly decrease LDL cholesterol levels by 60%(more than any other drug on the market), with an average decrease of 47%
  • So far, safety data in short-term studies has also been positive
  • Long-term studies for efficacy and safety are currently ongoing

Even though the FDA typically prefers data from long-term studies,  the FH community needs more options…and they need them now!

EDMAC echoed that sentiment by recommending approval of PSCK9 inhibitors. For a community in such need, the benefits of these treatments outweighed the risks. Now, while the FDA does not have to follow recommendations by their advisory committees, it often does.

This marks a huge step forward in treatment for HoFH! The FDA is expected to make its decisions on these drugs by July and August 2015.

It just goes to show you what a community can do when they band together. As Deborah McCall, a member of EDMAC, told Colleen McCready, “You really made a difference by coming today.”

Read the full length article from the NIH to learn more about this breakthrough treatment looking to alleviate the FH community.

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