After stepping in to relieve a drug shortage that forced Fabry's disease patients to ration their drug supplies, Shire has pulled its drug, Replagal, from FDA review. The company was set to go before the agency next month, but preliminary talks indicated the FDA was going to require more testing.
Spokesperson Jessica Cotrone told MM&M that additional costs would be in the millions and “would take years to conduct and finish, then file, then wait for approval.” It would have also been the second time the company was asked to go back for more information – Shire withdrew a BLA application for Replagal in 2010 when the agency asked for more data. The drug is approved in 46 other countries to treat the rare disease, which renders sufferers unable to break down a specific type of fat and is linked with kidney damage, heart attack and stroke.
Jerry Walter, founder and president of the National Fabry's Disease Foundation, told MM&M he understood Shire's position but is disappointed.
The global shortage was triggered by contamination and quality control issues at Genzyme plant in Allston, Mass, almost three years ago. The problems cut Genzyme's Fabrazyme distribution by about 70% and patients were told to scale back on their medication.
As a backstop, Shire got the FDA's approval for limited distribution of its drug Raplagal, which was provided to about 140 of the country's Fabry patients for free. It was also an opportunity for Shire to break into the Fabry treatment space, and it filed a BLA that would put its drug on equal footing to compete with Genzyme, which costs patients between $200,000 and $250,000 a year.
Although Shire has walked away, Genzyme, which was purchased by Sanofi last year, has started to rebound. The FDA approved a new Genzyme facility in Framingham, MA, in January. Genzyme expects all US patients to return to full doses this month, and for output to meet full global demand beginning in the second quarter of this year.
“We applaud management's plan to shut down Fabrazyme's relatively ineffective Allston manufacturing in order to more quickly return to full supply via this approval,” Leerink Swann analyst Seamus Fernandez said in a January research note.
Fernandez said the clearance has an added benefit. “This is a critically important step in the validation of the [Genzyme] acquisition as Framingham was the key gating factor in a recovery toward historic peak sales of $5B,” he said.
Walter praised the increased production, but said Shire's withdrawal is still a concern.
“It may take years now for us to get another option to protect ourselves from things like a drug shortage” he said.