Wainua now available in US through Orsini specialty pharmacy

Wainua is first and only approved FAP treatment that can be self-administered

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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A few weeks after its approval in the U.S., Wainua (eplontersen) will now be available to adults with familial amyloid polyneuropathy (FAP).

Orsini was selected as the exclusive specialty pharmacy partner to provide Wainua, which was co-developed by Ionis Pharmaceuticals and AstraZeneca. The medication will also be made available through integrated delivery networks within qualifying specialty pharmacies.

Wainua is the first and currently only approved FAP treatment that patients can self-administer by using an autoinjector.

“Orsini is proud to be the exclusive specialty pharmacy offering access to Wainua as a new treatment option for patients living with [FAP],” Brandon Tom, CEO of Orsini, said in a company press release. “We are on a mission to compassionately serve rare disease patients, and Wainua is an important addition to our Neurology Center of Excellence.”

FAP, also known as hereditary transthyretin-mediated amyloid polyneuropathy, is a rare, inherited disorder caused by mutations in the TTR gene. As a result, abnormal clumps of the transthyretin protein build up in tissues, particularly in nerve cells, leading to damage.

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Wainua designed to reduce transthyretin protein production

Wainua is an RNA-targeted therapy designed to reduce transthyretin protein production, and thereby prevent the buildup of toxic transthyretin clumps and slow disease progression. With a mode of action similar to Tegsedi (inotersen), Wainua was engineered to be more effective at reaching the liver, where most transthyretin is produced.

Both therapies are administered under the skin, or subcutaneously, but Tegsedi is given once a week while Wainua is administered once a month.

Data from the Phase 3 NEURO-TTRansform (NCT04136184) trial, which supported the therapy’s approval, showed that after over a year of treatment with Wainua, transthyretin levels were reduced by over 81%. This was accompanied by an easing of disability related to nerve damage in 47% of patients, and improvements in quality of life in 58% of participants.

In terms of safety, Wainua is generally well tolerated. According to the therapy’s prescribing information, the most common side effects reported include reduced levels of vitamin A and vomiting. Therefore, people starting on the therapy should supplement with vitamin A.

Wainua is currently being tested in the Phase 3 CARDIO-TTRansform trial (NCT04136171) in patients with transthyretin-mediated amyloid cardiomyopathy — an FAP-related disorder in which toxic transthyretin clumps mainly damage the heart muscle.