A patient advocacy organization is putting its weight behind an effort to urge the U.S. Food and Drug Administration (FDA) to reconsider its recent decision to reject a treatment for the rare lipid disorder familial chylomicronemia syndrome (FCS).
Last week the FDA issued a stunning rejection of Akcea Therapeutics’ Waylivra, an experimental treatment that reduced triglycerides by 77 percent in FCL patients during late-stage trials. The drug had already been supported by an FDA advisory committee in May, which typically sets the stage for full FDA approval. However, the company and FCS patients were stunned by the FDA’s rejection. That rejection leads to a round of layoffs at Akcea. This week the company announced in a filing with the U.S. Securities and Exchange Commission that it was reducing its workforce by 10 percent. The cuts will primarily impact those employees who had been working on Waylivra, the company noted in its filing.
When Akcea, a subsidiary of Ionis Pharmaceuticals announced it had received the Complete Response Letter at the end of August, the company did not disclose why the regulatory agency rejected the drug. The CRL may have to do with the safety profile of Waylivra. During the advisory committee meeting, there were concerns over significant drops in platelet counts in some patients. When Akcea announced Phase III results, the company noted that five patients were forced to withdraw from the trial due to a drop in platelets.
Now though, as Akcea works with the FDA on a path forward, an FCS advocacy group is also urging action – hoping that its voice will lead to the FDA reconsidering the medication. The FCS Foundation announced Thursday that it was demanding the FDA reconsider its decision regarding Waylivra. The advocacy group said FCS patients are “desperately in need” of access to a treatment.
“This FDA decision leaves FCS patients with no clinical treatment for the disease,” the foundation said in a statement to BioSpace.
The FCS Foundation noted that when the FDA advisory committee initially reviewed the Waylivra clinical data in May, there were numerous members of the FCS community who testified in support of approval. Many of those speakers participated in the Waylivra clinical trials, the foundation said. In a statement sent to the FDA, foundation members pointed to a lack of treatments available to FCS patients, other than a strict low-fat diet. Even with adherence to the diet, the foundation noted that the burdens of FCS remain.
“Despite the FDA having a department that is committed to understanding the impact and burden of rare diseases, it appears they have not taken the patient experience or testimony into consideration in their decision to not approve Waylivra,” the foundation said.
FCS is a severe, rare disorder where patients have extremely high levels of triglycerides in their blood, causing a range of symptoms including potentially fatal attacks of acute pancreatitis. With limited LPL function, people with FCS cannot breakdown chylomicrons, lipoprotein particles that are 90 percent triglycerides. FCS patients are at risk of chronic complications due to permanent organ damage. They can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognition that affect their ability to work.
“If the FDA truly listened to our patient stories and experiences with FCS and treatment with Waylivra, this drug would not have been rejected,” Melissa Goetz and Lindsey Sutton, co-founders and presidents of the FCS Foundation said in a statement.
Under Commissioner Scott Gottlieb, the FDA has been known to reverse decisions. Last month, four days after rejecting a new dosing label for Regeneron’s Eylea, the regulatory agency overturned its decision. Other companies that have also benefitted from an about face include a New Drug Application by Alkermes, as well as reversals for Amicus‘ Fabry disease treatment, as well as Eli Lilly’s rheumatoid arthritis drug baricitinib and TherapeuticsMD, Inc.’s vaginal pain medicine TX-004HR.
