Orsini Chosen for Distribution of Approved FAP Treatment Amvuttra

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Alnylam Pharmaceuticals has selected Orsini Specialty Pharmacy to distribute Amvuttra (vutrisiran), which was recently approved by the U.S. Food and Drug Administration (FDA) to treat familial amyloid polyneuropathy (FAP).

“We are honored to work with Alnylam in providing access to this important treatment option or medication for adults with [FAP],” Mike Fieri, founder and CEO of Orsini, said in a company press release.

“Our dedicated Care Team will ensure patients and their caregivers receive Amvuttra utilizing the full breadth of Orsini’s specialty pharmacy services, which encompass distribution, education, monitoring, benefits assistance, and experienced, compassionate support,” Fieri said.

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FAP, also known as hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy, is caused by mutations in the TTR gene, which provides instructions for making a protein called transthyretin. Because of these mutations, an abnormal form of the transthyretin protein is produced, which forms toxic clumps in the body’s tissues, ultimately leading to disease symptoms.

Amvuttra is designed to reduce transthyretin production by binding and destroying TTR mRNA, an intermediate molecule produced when the gene is “read” and used as a template to make the protein.

This mechanism is basically identical to that of Onpattro (patisiran), an older FDA-approved treatment for FAP developed by Alnylam. Compared with Onpattro, Amvuttra is intended to be more stable in the body, allowing for a longer-lasting and more powerful therapeutic effect.

The FDA approved Amvuttra to treat FAP in June. Alnylam has set the therapy’s list price at $463,500 per year. The medication also recently received a recommendation for approval in the European Union for FAP patients with stage 1 or 2 polyneuropathy and is being considered for similar approvals in Japan and Brazil.

The FDA’s approval was supported by data from an ongoing Phase 3 clinical trial called HELIOS-A (NCT03759379), which enrolled 164 people with FAP and randomly assigned them to treatment with Amvuttra or Onpattro for 18 months.

Amvuttra was administered via under-the-skin injections every three months, while Onpattro was given via an infusion into the bloodstream every three weeks.

Top-line data from the trial showed that after nine months of treatment, Amvuttra improved walking, eased neurologic impairment, and bettered life quality, compared to historical data for FAP patients on a placebo.

More recent data from the ongoing study indicated these benefits were maintained after 18 months of treatment, with participants reporting less disabling symptoms and better quality of life. Exploratory trial data also suggest that Amvuttra may help limit heart damage in patients with cardiac involvement.