How a 15-year-old Genzyme drug shortage became a smorgasbord of pharma’s thorniest issues

A manufacturing problem from 15 years ago and the resulting prolonged drug shortage is at the heart of newly revived litigation against Sanofi’s Genzyme. An appeals court overturned the dismissal of a 2020 lawsuit brought by patients who allege they were harmed by Genzyme’s handling of a shortage of its drug Fabrazyme.

The case combines many of today’s thorniest issues in pharma: Rare disease treatments, drug shortages, a market monopoly and even march-in rights.

Here’s what’s happening as patients and Genzyme await more action from courts.

The beginning

The trouble began in 2009 with Genzyme’s drug Fabrazyme, which at the time was the only FDA-approved treatment for Fabry disease, a rare genetic disorder. In June of that year, Genzyme discovered viral contamination at its manufacturing facility, and the company halted Fabrazyme production, causing a shortage. 

A few months later, the FDA dinged Genzyme for more contamination, this time in the form of particulates, including steel fragments, which made the shortage even worse. Genzyme eventually agreed to a $175 million fine and an FDA consent decree to resolve the manufacturing woes.  

How Genzyme managed the shortage

Genzyme attempted to mitigate the Fabrazyme shortage with a rationing plan that provided reduced doses to stretch out the drug’s supply. 

In 2011, the shortage worsened when Genzyme diverted some of the supply to the European market while still rationing for U.S. patients. There was also a period in August 2011 when U.S. patients didn’t receive any Fabrazyme doses at all. In March 2012, Genzyme restored full supplies of Fabrazyme to U.S. patients.

Alleged ‘mishandling’ of the shortage

Patients allege that the drug shortage harmed them both physically and financially, and that Genzyme knew that low doses of Fabrazyme wouldn’t treat Fabry disease effectively. 

Patients also allege that despite knowing this, Genzyme continued to misrepresent “both the effectiveness of its low-dose regimen and the expected duration of the shortage,” according to the new decision.

A market monopoly

In addition to saying they were harmed by the low doses, patients claim that Genzyme diverted doses away from the U.S. market because Fabrazyme was the only approved Fabry disease treatment there, and diverting the drug wouldn’t harm their U.S. market share. In Europe, however, there was another approved Fabry disease treatment, so they provided doses there to stave off competition, patients alleged.

Invoking march-in rights

March-in rights are another part of the Fabrazyme story. When a patent holder creates an invention that was created using taxpayer funds, the federal government can invoke so-called march-in rights to force the patent holder to license their patent to other applicants. For example, march-in rights are being floated as a way for the U.S. government to drive down prescription drug prices. 

Although they’ve never been used before, the FTC recently called them “an important check on companies charging Americans inflated prices for drugs developed with taxpayer-funded research.” 

March-in rights were part of the Fabrazyme conversation more than a decade ago, too, when patients tried to get the federal government to invoke them and allow other companies to manufacture the drug. They were unsuccessful because the government didn’t believe invoking march-in rights would increase Fabrazyme supplies in the timespan needed to relieve the temporary shortages.

Mounting lawsuits

Patients have filed lawsuits against Genzyme in the years since the Fabrazyme shortage, but they’ve often been dismissed due to lack of standing. Among them was a 2020 lawsuit that was dismissed, in part, because the court said the plaintiffs failed to specify how they were harmed. In overturning that dismissal last week, the appeals court disagreed, saying the complaint “makes specific allegations about the particular injuries suffered by each individual plaintiff.”

Where things stand now

The appeals court wrote that the dismissal was incorrect and vacated the judgment, sending the case back to the district court. That means, for the plaintiffs, their once-dismissed lawsuit is suddenly back in play.