In the brief history of Risk Evaluation and Mitigation Strategies, QOL Medical’s enzyme replacement therapy Sucraid stands out for the unusual reason behind the REMS requirements in the first place, and because it was the first and only product to date to be released from its risk management obligations.

The Sucraid example demonstrates FDA’s willingness to release a sponsor from a REMS, even one that contains elements beyond a mere MedGuide, when required assessments demonstrate that strict risk management measures are no longer necessary.

Sucraid (sacrosidase), an orphan drug, was first approved in April 1998 for treatment of genetically determined sucrase deficiency, which is part of congenital sucrase-isomaltase deficiency (Also see "Orphan Medical Will Launch Sucraid For Sucrase Deficiency In June" - Pink Sheet, 20 April, 1998.).

More than 10 years post-approval, the agency required a REMS in conjunction with a manufacturing change involving the active ingredient.

In a November 2008 press release, FDA cited concerns that the newly manufactured product may contain increased levels of the enzyme papain, which can cause allergic reactions. The agency said that to avoid a shortage of Sucraid, it allowed the manufacturing change but required a REMS to communicate information about the change and assess whether there was an increase in allergy-related adverse events.

The REMS included a communication plan in the form of a physician letter and questionnaire, along with elements to assure safe use and an implementation plan.

Under the REMS, Sucraid could only be prescribed by providers who attested that they had read and understood information about the manufacturing change, evaluated patients receiving the new product for adverse events and completed a survey about each patient’s experience. The product was shipped directly to each patient from a single central pharmacy, and patients were requested to complete a questionnaire about their experience using the new formulation.

REMS assessments were to be conducted at 18 months, three years and seven years – a standard schedule.

In 2010, QOL Medical submitted a supplemental NDA and its first REMS assessment. The company proposed that the REMS was no longer necessary, and FDA agreed. In a Dec. 9, 2010 letter, FDA said the patient mailings, telephone survey and physician surveys conducted as part of the REMS showed no reports of adverse events.

QOL said Sucraid is still distributed through an exclusive specialty pharmacy for commercial reasons unrelated to any regulatory requirement.

By Sue Sutter

QOL Medical’s Sucraid Is First To Lose Its REMS

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