FDA Approves New Drug for Rare Nerve Disease: 4 Things MCOs Should Know

MHE PublicationManaged Healthcare Executive
Volume 28
Issue 10

Here are four other things that MCOs should know about a first-of-its kind targeted RNA-based therapy to treat a rare disease.

FDA’s approval of a new drug, Onpattro (patisiran) infusion, to treat peripheral nerve disease (polyneuropathy) on August 10 also marks FDA’s first approval of a new drug class called small interfering ribonucleic acid (siRNA) treatment.
The drug, manufactured by Alnylam Pharmaceuticals, Inc., was approved for adults with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR), a debilitating and often fatal genetic disease characterized by the buildup of abnormal amyloid protein in peripheral nerves, the heart, and other organs. Affecting about 50,000 people worldwide, hATTR is a rare condition.

“The disease results in patients developing numbness and weakness in their limbs, difficulty with gut movement and digestion, and heart failure,” says Michael J. Polydefkis, MD, professor of neurology at John Hopkins University in Baltimore and a lead investigator of the phase 3 APOLLO study for Onpattro. As an inherited disease, if someone has hATTR each of their children has a 50% chance of getting the disease-causing gene.

Here are four other things that MCOs should know about this new drug.

  • siRNAs are a new way of treating disease. The treatment alters the genetic drivers of disease. RNA inhibitors (RNAi) can essentially target any gene in the human genome and prevent it from turning on. “Targeting the disease’s source (the expressed gene) versus a surface membrane protein (which is how biologics work) provides an off switch for the disease,” says Kellie Rademacher, PharmD, vice president of Payer Access Solutions at Precision for Value, a healthcare company that supports commercial excellence for pharmaceutical clients.

“RNAi has the potential to be disease modifying not only for hATTR, but also for other diseases caused by gene expression, such as hemophilia, cancer, and age-related vision loss,” Rademacher says. “RNAi technology is precision medicine at its foundation.” 

  2.  A new treatment for hATTR was needed. Currently, drugs to treat hATTR slow its progression, but don’t stop it. “The gold standard of treatment is liver transplant or multi-organ transplant (depending on the disease’s severity), because the TTR protein is primarily produced in the liver. Data released for Onpattro showed a reduction in the development and deposits of TTR amyloids, resulting in maintenance or improvement in ambulation, nutritional status, activities of daily living, and quality of life, Rademacher says. 

Polydefkis believes that patients who take Onpattro will experience less disability, be more productive, and ultimately live longer.

  3.  It comes with a hefty price tag. The average annual list price for Onpattro before insurer discounts is estimated at $450,000. The effective net price is $345,000 annually, taking into account mandatory government discounts. According to a press release by the manufacturer, “As a therapy designed to treat less than 3,000 currently diagnosed patients in the United States, Onpattro is priced relative to the population it will treat.”

Rademacher says the pricing isn’t outside the realm of what she has seen with other rare disease agents, especially where technological scientific advancements have been made. “Even though only a small number of people have this disease, payers will likely want to manage Onpattro carefully because of its cost and specificity,” she says. “Onpattro is a lifelong therapy without any realized outcomes yet. Clinical trial data look promising, but whether or not real-world outcomes translate into savings for payers and patients remains to be seen.”

  4.  Methods to pay for pricy drugs are needed. Rademacher says how the system reimburses for Onpattro and other new exciting gene-related technologies needs to be reassessed. “Perhaps costly drugs could be financed similar to how other large purchases are financed,” she says. “How the healthcare system provides access and reimburses for these advances will require all stakeholders to collaborate.”


Karen Appold is a medical writer in Lehigh Valley, Pennsylvania.

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