Will the FDA Hear These FCS Patients?

Lindsey Sutton showed signs of familial chylomicronemia syndrome as early as five weeks old.

Sutton is now 28 years old. Because of her condition, she must adhere to a very careful diet. Despite this, a number of hospital visits are still necessary to control the pancreatitis resulting from her condition. A drug therapy by Akcea Therapeutics Inc. led to a drastic improvement in her symptoms. The United States Food and Drug Administration (FDA), however, rejected the treatment in August. Despite the drug’s positive effect in Sutton’s life, the FDA had big questions. How can this be resolved? Keep reading to learn more, or follow the original story here for more information.

Three Years of Freedom

Familial chylomicronemia syndrome (FCS) causes the body to store and buildup fats. As a result, people with FCS must be very careful of their diet. Sutton, for example, limits her intake of fats to about a tablespoon of olive oil every day. This aids with some symptoms, but ultimately isn’t enough to spare her from multiple hospital visits for complications including pancreatitis.

Starting on a new treatment did, however, help with the hospitalizations. Waylivra, a product of Akcea Therapeutics Inc, kept Sutton out of hospitals for three consecutive years. It also improved her other symptoms. Pain and fatigue were both lessened by the effects of the drug. Sutton reported feeling “normal” for the first time in her life. This is why patients like Sutton are so perplexed and passionate about the FDA decision to not approve Waylivra. For patients like Sutton, the drug has literally been life-changing.

Inside the FDA

One reason the FDA decided against Waylivra pertains to a side effect of the drug. Waylivra has been shown to cause a decline in platelets. According to Akcea’s CEO, the FDA seeks to better understand this side effect before it will consider approving Waylivra. There are also questions about the dosage of Waylivra. The proposed dosing regulations do not match the ones originally studied according to the FDA.

Akcea plans to continue pursuing Waylivra. The company’s CEO hopes to see positive results from an upcoming open label extension study. On top of the six months of patient data, this study will provide the company believes that patients using the drug through the compassionate use program will also demonstrate further evidence of its viability. Akcea hopes to meet with FDA regulators before the year ends to reopen the issues surround Waylivra.

While the FDA does not comment on pending drug applications, it did issue a broad statement. A spokesperson for the FDA announced via email that patient advocates play an important role in the agency’s considerations.

“The FDA carefully considers patient input as it fulfills its role in the drug development process.”

Sutton and her colleagues have already gathered 12,000 signatures in support for the treatment. Their petition is to be presented to the FDA as well.


 

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