The U.S. Food and Drug Administration (FDA) gave Akcea Therapeutics and its parent company Ionis Pharmaceuticals the green light for Tegsedi, its treatment for polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
The FDA approval marks the third for Tegsedi, which was previously green-lit in Canada and the European Union. While the companies snagged approval, Tegsedi will be hobbled with some safety concerns. Tegsedi can be associated with serious bleeding issues caused by low platelet counts, as well as potential kidney concerns. Because of those issues, Tegsedi will only be available through a restrictive program called the Tegsedi Risk Evaluation and Mitigation Strategy, the company said on Friday.
Despite the safety concerns, Akcea and Ionis are upbeat about Tegesdi’s approval, particularly with the August FDA rejection of Waylivra, a treatment for the rare lipid disorder familial chylomicronemia syndrome. Paula Soteropoulos, chief executive officer at Akcea Therapeutics, said Tegsedi is the “first and only RNA-targeting therapeutic” that reduces the production of TTR protein through a once-per-week subcutaneous injection. She said the company believes the drug’s efficacy, combine with its ease of self-administration, will make Tegsedi an “excellent choice” for many patients.
“As we execute on our multi-country launch, we are committed to delivering a comprehensive treatment experience for people taking Tegsedi. Beyond the drug itself, this includes ensuring patients who need this therapy have access to it, offering personalized support programs and enabling patients to administer treatment on their own terms,” Soteropoulos said in a statement.
The new Ionis/ Akcea drug will square off against Alnylam’s hTTR amyloidosis treatment, Onpattro (patisiran), which was approved by the FDA in August. Pfizer could also have a treatment for hTTR amyloidosis treatment approved. The company has secured approval in Europe for Vyndaqel (tafamidis), but not yet in the U.S.
Tegsedi was approved by the FDA following Phase III results that demonstrated the efficacy of the drug in patients with hATTR amyloidosis with symptoms of polyneuropathy in comparison to placebo. In hATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body. Tegsedi is designed to target the disease at its source by reducing the production of TTR protein. In the NEURO-TTR study, treatment with Tegsedi produced up to a 79 percent mean decrease from baseline in serum TTR protein in patients. Akcea licensed Tegsedi (inotersen) from its parent company earlier this year, along with AKCEA-TTR-LRx, a potential treatment for hereditary and wild-type types of ATTR. Following the FDA approval, Ionis will receive a $50 million milestone payment that may be made in Akcea common stock or cash. Commercial profits and losses from TEGSEDI will be split 60 percent to Ionis and 40 percent to Akcea.
In addition to the regulatory approval, Akcea also announced that it had struck a dealwith Accredo specialty pharmacy, a subsidiary of Express Scripts, as the distributor of Tegsedi. Steve Miller, chief medical officer of Express Scripts, said as a self-administered medication, Tegsedi should reduce administration costs and drug wastage.
“By introducing competition into the marketplace this should drive down the total cost of care,” Miller said in a statement.
To assist hATTR amyloidosis patients in gaining access to Tegsedi, Akcea created Akcea Connect, a patient support program. Akcea Connect is accepting patient enrollment and authorization forms, the company said. Sarah Boyce, president of Akcea Therapeutics, said the company is ready to help patients through every stage of treatment with Tegsedi.