Tuesday, June 6, 2017

Good News for Sickle-Cell Patients, But How Will Emmaus Make Money? A Look at the Business Side of Pharmaceutical Policy

 Last week’s most interesting story was that a the FDA Oncologic Drug Advisory Committee voted 10-3 to recommend that the FDA approve Emmaus Medical’s L-Glutamine for the treatment of Sickle Cell Disease.  The Phase III Clinical Trial showed that patients receiving L-Glutamine had a clinically significant reduction in the number of Sickle Cell Crises over the course of the 48-week trial. There were also positive results on other measures of the drug’s benefit, while the safety issues were relatively minor. So this is good news for Sickle Cell Disease patients. There are few therapeutic options for this serious disease and the likely approval of another meaningful treatment may make a real difference in their lives. 

BUT, this is one of the most unusual drug development studies I have seen. While other Orphan Drug makers have taken older compounds through development and relied on the 7 years of Orphan Drug exclusivity to return a profit on their investment, L-Glutamine is not just an older compound. Unlike repurposed older drugs, it is widely available and used as a dietary supplement. So while Emmaus is to be commended for undertaking the costly and risky process of establishing the effectiveness of L-Glutamine for Sickle Cell Disease and will be the only manufacturer allowed to label L-Glutamine for treating Sickle Cell Disease, they will not be the only manufacturer selling L-Glutamine in bulk quantities sufficient to treat Sickle Cell Disease. A quick search online revealed numerous online sellers.  For example, one online seller is offering 250 grams of L-Glutamine (advertised as pharmaceutical-grade) for $9.95.  Since the Emmaus studies dosed patients at between 20 to 60 grams a day, depending on body weight, the 250 grams being sold online would be last between 4 to 12.5 days.  Even assuming that most patients require 60 grams per day, the price for 1800 grams online would be a bit more than $60 per month.  I have not yet seen an announcement of Emmaus’ intended pricing for their product post-approval, however, their current pricing for the same drug, approved for short bowel syndrome, would be over $2000 per month at the higher dose.  While the Rx form is likely to be covered by insurance, it still seems quite possible that a significant market share could be lost to patients simply buying L-Glutamine online. That would still be good for patients, but not very good for Emmaus’ bottom line. It will be very interesting to follow Emmaus’ sales over the next few years to see whether taking a dietary supplement through the FDA approval process can actually work for companies as well as patients.

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