Amvuttra for FAP Among Specialty Treatments Offered by Soleo Health

Vutrisiran, injection therapy, eases symptoms of familial amyloid polyneuropathy

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by Andrea Lobo |

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vutrisiran | FAP News Today | Soleo Health offering Amvuttra | illustration of person on injection therapy

Soleo Health, a U.S. national provider of pharmacy services, announced that Amvuttra (vutrisiran), an approved treatment for adults with familial amyloid polyneuropathy (FAP), has been added to its portfolio of specialty treatments.

Amvuttra was developed by Alnylam Pharmaceuticals to treat FAP, also known as hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy. It was approved in the U.S. in June and more recently for patients in the European Union.

“This marks the fourth therapy Soleo Health now offers from Alnylam Pharmaceuticals’ rare disease product portfolio further strengthening our ability to care for more patients living with rare disease who benefit from innovative new therapies like Amvuttra,” Drew Walk, Soleo Health’s CEO, said in a company press release.

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UC San Diego Health 1st in US to Offer Amvuttra for FAP

FAP is caused by mutations in the TTR gene, which encodes a protein called transthyretin (TTR). These mutations lead to the production of an abnormal version of TTR whose toxic protein deposits (amyloid fibrils) accumulate in tissues, often nerve cells. Over time, damage from this buildup leads to disease symptoms that typically include limb numbness, tingling, and weakness.

To produce a protein, a gene’s DNA sequence is used to create a temporary molecule, called messenger RNA, that is then used by specific components of cell machinery to make a protein.

Vutrisiran’s US and EU approvals supported by Phase 3 HELIOS-A trial

Amvuttra binds to, and silences, the TTR gene’s messenger RNA, reducing TTR protein production. Its mechanism of action is similar to that of Onpattro (patisiran), another approved FAP treatment by Alnylam. However, Amvuttra is designed to be more stable and to last longer in the body, possibly providing more powerful and longer-lasting therapeutic effects.

Amvuttra’s approvals were supported by data from the Phase 3 HELIOS-A trial (NCT03759379) that assessed the safety and efficacy of the therapy, then known only as vutrisiran, in 164 adults with FAP.

After 18 months of treatment, 48% of patients treated with Amvuttra saw their nerve symptoms ease, as measured by a reduction in the modified Neuropathy Impairment Score (mNIS+7), while only 4% of those on an external placebo group — part of an earlier Phase 3 trial — achieved the same outcome. Moreover, 57% of Amvuttra-treated patients reported quality of life improvements, compared with 10% of those given a placebo.

Amvuttra’s recommended dose is 25 mg, given once every three months as an under-the-skin (subcutaneous) injection by a healthcare professional.

Soleo Health, a Texas-based company, provides complex pharmacy services that can be administered at home or treatment sites, following a physician’s recommendation. The company has 21 pharmacy locations and is licensed in all 50 U.S. states.

Soleo reported that its interdisciplinary team is trained in the clinical profile and administration of Amvuttra. Its specialty health care services are provided by experienced pharmacists, nurses, reimbursement specialists, and patient ambassadors.