This Woman Takes on the FDA in One Epic Battle

No one knows the dangers of bad cholesterol better than patients with familial hypercholesterolemia (FH).

According to the FH Foundation, there are two forms of FH. 1) HeFH, and 2) HoFH, being the rarest of the two. Those with HoFH have inherited it from both of their parents and experience symptoms brought upon by extremely high cholesterol. Without treatment, LDL (a.k.a. the bad cholesterol) levels of patients with HoFH can range anywhere between 500 and 1000, which can lead to a whole host of health issues. It goes without saying that treatment can be the difference between life and death for these people.

Good news! As reported by Xconomy, after a two-day panel discussion about two new cholesterol-reducing drugs, Katherine Wilemon, founder and president of the FH Foundation, feels hopeful. The goal of the outside advisors on the panel was to convince the FDA of approving BOTH drugs, as each could be the saving grace for someone with FH.

For some people with a form of FH, regular old statins can do the trick, but for those who need more, the approval of these cholesterol-fighting drugs must be expedited. And subsequently, with the potential promise of a bigger and better financial gain, pharma companies can’t argue with THAT, now can they?

Statins to treat FH
Statins have long been the primary source to lower LDL cholesterol in the FH community. People like Wilemon are hoping to get the FDA to grow that list. Source: Tabletsmanual.com

Having FH, herself, Wilemon speaks from experience. After many years of taking statins, Wilemon did not see the results she wanted. It was only after participating in a clinical study for a PCSK9 inhibitor that her LDL levels began to finally decrease.

During the two-day panel, advisors agreed that in order to be approved, the drugs must not only lower lipid levels, but the risk of cardiovascular disease as well, providing that the benefits outweigh the side effects. However, questions posed include, “For what populations of people should these drugs be indicated? Those with simply high cholesterol? Those with HeFH? HoFH? Those who cannot tolerate statins?” And ultimately, “Should the approval of these drugs be expedited with the risk of an inadequate supply of data?”

Read more from the full discussion to see how the lives of FH patients may change forever.

And I’m sure we’re all wondering what the insurance companies think about these new developments. You’ve probably already guessed it… insurers have practically promised to make paying for PCSK9 inhibitors a living hell–no matter how well the drugs do in clinical trials. Ultimately, they wonder, with statins so cheap and prevalent on the market, how mandatory are these additional treatments really?

But first and foremost, you have to convince the FDA, which calls for not only a significant reduction in LDL levels but also a balanced safety profile. For this reason, some think clinical trials should be prolonged in order to collect sufficient data. Otherwise, doctors may not even feel comfortable prescribing them, thus making pharma’s efforts in speeding the process of approval all for naught…

Still, Wilemon is confident that the FDA and insurers will see the light in “understand[ing] the need for additional treatment options.”

As of July, according to the FDA, one of the drugs, Praluent, was approved for the treatment of HeFH. However, no FDA decision has been made as of yet on the approval of the other, Repatha.


The FH community has been waiting for new drug approvals for a long time. Share the good news with those in your social networks!

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