Agalsidase Beta

Genzyme was dealing with fallout from the shortage and related manufacturing woes back when it sold to Sanofi in 2011. It’s been 15 years since Genzyme began rationing the Fabry disease treatment Fabrazyme after a shortage caused by contamination at a manufacturing site, marking the start of many years of litigation. Now, after a quiet period, an appeals court has put one case back on the table. More than two dozen plaintiffs are included in the lawsuit at hand, which was filed in 2020 and seeks monetary damages for injuries allegedly stemming from Genzyme’s “mishandling” of a Fabrazyme shortage between 2009 and 2012. The U.S. Court of Appeals for the First Circuit ultimately decided that 22 members of the plaintiff group have legal standing to assert their claims. The court's opinion (PDF), from Feb. 15, reverses a prior ruling dismissing the case from the U.S. District Court for the District of Massachusetts. Sanofi has no comment on the ongoing litigation, a company spokesperson said. The French drugmaker picked up Genzyme in 2011, giving the then-struggling company a lifeline after its manufacturing shortfalls led it ink an FDA consent decree and pay a $175 million fine. The legal hubbub began after viral contamination in a bioreactor at Genzyme’s Fabrazyme manufacturing site started a shortage of the drug back in 2009.  At the time, Fabrazyme was the only commercial treatment for Fabry disease, a rare inherited disease that can cause heart and kidney failure among other life-threatening complications. When administered every two weeks, Fabrazyme works to slow the progression of the disease. Later that year, the FDA found a separate instance of contamination at the plant, further threatening the already low supply. In response, Genzyme began “arbitrarily” rationing the drug, a lawsuit from 2009 alleged. While drug rationing is often a forced consequence of a supply squeeze, the legal complaints stem from certain aspects of the company's response. In the lawsuits, the plaintiffs say Genzyme sold a reduced dosage despite its alleged knowledge that lower doses are not effective. In 2011, Genzyme “worsened” the U.S. shortage by diverting some supply to Europe, plaintiffs have argued. Fabrazyme supply recovered in 2012, but not before patients had gone through long periods of time without getting full doses, according to the lawsuit. Since the shortage began, the issue has resulted in multiple class-action lawsuits, appeals and dismissals, plus settlement talks. Plaintiffs in the 2020 suit, which includes Fabry patients as well as spouses of deceased Fabrazyme users, specifically describe several injuries attributed to the situation. The plaintiffs argue the company “knowingly misrepresented” both the expected timeline of the shortage and the effectiveness of a low-dose treatment regimen.

An appeals court ruling from late last week will revive a lawsuit over a Sanofi subsidiary’s contamination issues and a subsequent shortage of a key rare disease drug. At issue in the case is a series of alleged blunders related to the supply of Genzyme’s drug for treating Fabry disease, a rare neurological disorder that can lead to kidney failure, heart failure or stroke. The revived lawsuit, filed in 2020 in a Massachusetts federal court, seeks monetary damages for more than two dozen Fabry patients who allege they were injured by the company’s handling of the contamination and subsequent shortage of the drug, marketed as Fabrazyme, that occurred between 2009 and 2012. Sanofi acquired Genzyme for about $20 billion in 2011.