July 10, 2017
By Mark Terry, BioSpace.com Breaking News Staff
A private company in Torrance, Calif., Emmaus Life Sciences, won the first approval for a drug for sickle cell disease in nearly 20 years.
The U.S. Food and Drug Administration (FDA) approved Endari (L-glutamine oral powder) for the complication of sickle cell disease in adults and pediatric patients five years and older. The drug reduces oxidant damage to red blood cells.
There is a caveat, however. The drug’s active ingredient, L-glutamine, has been around for years and can be bought over the counter. Its availability has the potential to undercut the company’s efforts to get insurance companies to pay for the drug.
The drug, in clinical trials, reduced the frequency and length of hospital stays for patients with pain caused by sickle cell disease compared to patients on placebo. Adverse effects included constipation, nausea and headache.
John Carroll, with Endpoints News, contacted the company and asked if they had any kind of commercial organization or sales force. The company responded in a statement, saying, “Endari is a significant milestone for the sickle cell patient community. It is the first advancement in treatment of this devastating disease in nearly 20 years and the first-ever treatment option for children. We expect to begin marketing Endari in the fourth quarter of 2017 and have plans to price Endari at a very reasonable amount to ensure patient access. We will apply for European market approval in late 2017 and we’re working with various groups and regulatory entities to provide access to Endari in the Middle East and Africa.”
The company also indicates it expects to market Endari for $11,000 to $18,000 per year. L-glutamine can be bought online for prices ranging from $7.70 to $58.60. Emmaus sells L-glutamine powder as NutreStore for Short Bowel Syndrome (SBS).
“A sickle cell crisis is the most common acute complication for patients and the number one cause of emergency room visits,” said Wally Smith, Florence Neal Cooper Smith Professor of Sickle Cell Disease, Division of General Internal Medicine, Virginia Commonwealth University, in a statement. “Endari has clinically shown to reduce sickle cell crises and hospitalizations, representing a significant medical advancement for patients with limited therapeutic options that have many side effects.”
Emmaus is sure to get some coverage for the approval, as well as a few peculiarities. Carroll writes, “In one of many odd facts I’ve collected about this company, new FDA chief Scott Gottlieb was on the board at Emmaus in the second half of 2015, one of many board postings he took over the past decade. Niihara’s bio [Yutaka Niihara, the company’s chairman and chief executive officer] also cites a role as a professor at the David Geffen School of Medicine at UCLA. A spokesperson for the school tells me he’s a volunteer clinical professor at the school of medicine who works at Harbor-UCLA Medical Center.”
Scientific American notes that Emmaus and Endari focus on “reducing the complications of sickle cell disease,” while “Bluebird Bio is developing a gene therapy to treat the disease’s underlying cause and potentially cure patients. Research using the CRISPR gene-editing technology aims to accomplish the same goal.”
Global Blood Therapeutics and Novartis are also working on sickle cell drugs.
