A Fabry disease charity organization has voiced its disappointment over Shire’s (NASDAQ:SHPGY) withdrawal of its biologics license application to treat the disease after talks broke down between the drug company and the U.S. Food and Drug Administration.
Recent interactions with the FDA led Shire to believe that it would be required to conduct additional controlled trials for approval, the statement said. The drug company concluded that the likely additional studies would cause a significant delay, and an approval of Replagal for U.S. patients would only be possible in the distant future, according to the statement. Shire’s move comes just as a drug shortage dating back to 2009 appears to be easing.
The country’s only FDA-approved treatment, Fabrazyme, is made by Genzyme, but it has faced manufacturing shortages since 2009 until the company could make a new Massachusetts factory fully operational this year. Genzyme is owned by Sanofi. Its treatment has been on the market since 2003.
Jerry Walter, the founder and president of the National Fabry Disease Foundation who also has Fabry disease, said Genzyme had resumed full doses of the drug earlier this month following three years in which the drug was rationed to half or one-third of the full dose. Walter said he was disappointed by Shire’s move to withdraw the drug but was not surprised, based on the drug company’s belief that the FDA would not approve the drug. In a comment on the association’s Facebook page, Walter said: “While Fabrazyme is now available at a full dose, the Fabry community won’t be able to relax until other treatment options are in place to provide security from another drug shortage.”
Fabry disease is a rare genetic lysosomal disorder caused by an enzyme deficiency. Childhood symptoms can include pain in the hands and feet, fatigue and gastrointestinal upset, among other symptoms. People with the disease have a high risk of strokes, heart attacks and kidney disease.
Other pharmaceutical companies are also developing treatments for the disease. Amicus Therapeutics has partnered with GlaxoSmithKline on a treatment that is currently in a phase 3 study. Protalix Biotherapeutics is set to commence clinical trials for its plant-based therapy this year.
Although Shire’s therapy for Fabry disease has been available in Europe since 2001 and is available in 46 markets worldwide, the drug company has failed to get it approved by U.S. regulators. It said the FDA had encouraged the company to submit applications for Replagal in 2009 and again in 2011.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
During the shortage, Shire had been providing Replagal free to 140 U.S. patients, or 20 percent of the U.S. patient population being treated for the disease, through treatment access programs.
Shire’s decision to drop the application just two weeks before it was scheduled to meet with the FDA’s Cardiovascular and Renal Drugs Committee could be the product of a calculation that with an uncertain path to approval, the financial burden of additional clinical trials was not worth the financial benefit of what is essentially an orphan drug with a patient population estimated to be a small, niche market that is already effectively supplied by an entrenched competitor, with more set to come.
