Rejected by the FDA, Ionis and Akcea's Waylivra finds new life with EU approval

With its chances for an FDA approval dead in the water, Ionis and Akcea’s ultrarare lipid disorder treatment Waylivra seemed doomed. Fortunately, the drug found new life in Europe.

On Wednesday, the European Commission approved Waylivra for adjunct treatment of familial chyromicronemia syndrome (FCS) after diet adjustment and triglyceride therapy. FCS is an ultra-rare inherited disorder that prevents the breakdown of fats, leading to abnormally high triglyceride levels in the blood.  

With its first major approval under its belt, Waylivra will enter the market as the only approved treatment for FCS in Europe, the company said.  Akcea CEO Paula Soteropoulos said the drug is scheduled for launch in Germany this year, with other launches set for 2020.

Ionis and Akcea’s success in Europe followed a surprise FDA overturn in August, after an advisory committee’s split vote of confidence in the drug. In response, Akcea axed 10% of its workforce after building up its sales team in anticipation of an FDA approval.

In its briefing rejecting the drug’s application, the FDA flagged several concerns, including the risk of serious bleeding and low blood platelet count. The agency questioned whether the drug needed a risk evaluation and mitigation strategy, and whether FCS was a specific enough diagnosis for the drug’s approval.

The FDA also knocked Akcea for unexpectedly submitting an amendment to its approval filing that offered up a new dosing and platelet monitoring strategy not tested in clinical trials.

As Waylivra has suffered its setbacks, Ionis and Akcea’s other ultra-rare disorder drug Tegsedi, which launched last fall, has faced some pricing pushback, and soon could confront new competition.

Tegsedi is one of three approved treatments for variants of transthyretin-mediated amyloidosis (ATTR), alongside Alnylam’s Onpattro and Pfizer’s tafamidis, which won an FDA nod earlier this week. 

In April, the National Institute for Health and Care Excellence (NICE)—England’s drug-cost watchdog—reversed its initial guidance and recommended Tegsedi for treatment of hereditary ATTR, an ultra-rare disease that affects about 150 U.K. citizens.

NICE’s initial guidance knocking Tegsedi for its lack of affordability in October 2018 closely followed the FDA’s nod for the drug, which bears a U.K. list price of $7,737 per week. The drug is priced at $450,000 per year in the U.S., or $8,653 per week.

Now that Pfizer's new ATTR drugs, dubbed Vyndaqel and Vyndamax, are approved, it could be just a matter of time before Tegsedi has to line up against the Big Pharma's marketing power. Pfizer already decided to offer its meds at a list price of $225,000 per year, a 50% discount over both Tegsedi and Onpattro. But as of yet, Pfizer can't challenge Tegsedi directly—or vice versa—because they're cleared to treat different forms of ATTR. Tegsedi's cleared for hereditary ATTR polyneuropathy (hATTR-PN), while Pfizer is working with the FDA to see whether its drugs can come back from a rejection in that indication.

If Pfizer is able to find a path to that approval, analysts figure the oral product could best its competitors’ subcutaneous injection delivery in terms of drawing patients if it faces them head to head.

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