- Patients with a rare, hereditary disease known as transthyretin amyloidosis now have a second new treatment option, after the Food and Drug Administration on Friday cleared Akcea Therapeutics and Ionis Pharmaceuticals' Tegsedi.
- Approval of Tegsedi puts Akcea in direct competition with Alnylam Pharmaceuticals, which won an FDA OK this summer for its drug Onpattro. Both therapies work by targeting RNA, degrading the genetic instructions responsible for production of a misfolded protein, which builds up in the organs of patients with the disease.
- Tegsedi will carry a black box warning, however, cautioning physicians and patients of the risk of low blood platelet counts and acute inflammation of the kidneys. By comparison, Onpattro's label is cleaner and without any major warnings.
Approval of Tegsedi is a needed win for Akcea and Ionis, which recently saw the FDA reject another drug that uses the same antisense technology as Tegsedi. Ionis is the majority owner of Akcea.
The approval is several months later than the companies had hoped for, though. A decision was originally expected earlier this year before the FDA pushed back its review to go over additional data supporting Akcea's submission for approval.
That delay cost Akcea a chance at launching its drug ahead of Alnylam's Onpattro, which has been on the market since early August.
The two drugs are each approved specifically for polyneuropathy of hereditary transthyretin amyloidosis, or ATTR. In patients with this condition, toxic deposits of misfolded TTR protein build up in the organs and peripheral nerves.
By targeting RNA, Alnylam's and Akcea's drugs reduce production of that protein, ideally allowing for the clearance of the problematic deposits.
Both therapies showed strong efficacy in their respective clinical studies, although Alnylam's demonstrated Onpattro could reverse neurological impairment in a little more than half of studied patients. In its trial, Tegsedi showed a substantial slowing — but not reversal — of disease progression on the same measure.
Akcea priced Tegsedi, which was developed by and later licensed from Ionis, at a maximum of $450,000 per year, the companies announced in a conference call Friday. That matches the cost of Onpattro, which also carries a list price of $450,000 per year. (Rebates, however, bring Onpattro's price down to an estimated net price of $345,000)
Both those prices, while not unheard of for ultra-rare diseases, have drawn fire from the Institute for Clinical and Economic Review (ICER). The group has called on both drugmakers to price their therapies lower, recommending a steep reduction in annual list prices of between 90% and 97% to meet its cost-effectiveness thresholds.
With only a few thousand Americans diagnosed with ATTR-related polyneuropathy, how competition between the two drugs shakes out is an question analysts and investors will be eager to have answered.
Akcea CEO Paula Soteropoulous, however, argues the dual approvals should help expand the market, spurring higher diagnosis rates.
"The market opportunity will grow much faster than it has in the past with other rare diseases," said Soteropoulos in an interview with BioPharma Dive ahead of the FDA's decision.
In the U.S, Akcea says there are 3,000 identified ATTR patients with polyneuropathy, and estimates another 12,000 could be diagnosed in the near future.
Soteropoulous also emphasized the convenience to patients of Tegsedi's once weekly injection regimen, which can be done at home. Onpattro is given by infusion once every three weeks.
The FDA, however, will require Tegsedi to be distributed through a Risk Mitigation and Evaluation Strategy (REMS) program, which will require weekly platelet testing and twice weekly monitoring for glomerulonephritis.
Akcea will partner with Express Scripts to distribute Tegsedi through the pharmacy benefit manager's specialty pharmacy, Accredo.
"By introducing competition into the marketplace this should drive down the total cost of care," said Steve Miller, chief medical officer at Express Scripts, in a statement from Akcea.