Alynlam Gets FDA Approval for Second RNA Interference Drug, Givlaari.

The United States Food and Drug Administration, this week, approved a new groundbreaking drug: the second to be based on a Nobel prize-winning technique called RNA interfence.  

This emerging treatment intends to treat a very rare genetic condition known as acute hepatic porphyria, which can cause bouts of severe abdominal pain.  Branded as Givlaari, the drug is from Alnylam Pharmaceuticals Inc, who also released the first RNAi-based drug—Patisiran–last year.  While the technology to pioneer this treatment has been around for at least a couple decades, the development is of recent mastery. 

Alnylam chief executive officer John Marganore comments, “We believe the approval of Givlaari represents a landmark event for the advancement of precision genetic medicines, providing new hope for patients and their caregivers living with the debilitating manifestations of AHP and unpredictable nature of AHP attacks, as well as for the doctors who diagnose and treat these patients.”

Of course, that is to say if you can afford this medicine.  Ultra-conditions like this painful metabolic disorder often receive subsidies and other financial assistance to help ensure patients have access but the average price for a year of Givlaari is around $575,000.  This breaks down about $39,000 per vial. After discounts, the company said the drug’s price will drop to $442,000.

For a little perspective, last year’s RNA-i treatment (Alynlam’s patisiran) costs upwards of $450,000 per year. That is, of course, before any other considerations are made. For example, the price is subject to insurer agreements over how well individuals could respond to it. Givosiran will have the same type of speculation with Alynlam offering rebates to some insurers who may cover a higher-than-expected proportion of qualifying patients. 

This type of thing has never been coordinated before in the rare disease treatment world.  However, Marganore asserts, this is something the company believes to be a responsible and appropriate method for devising ultra-rare orphan drug pricing in this market.