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FDA gives go-ahead to first sickle cell treatment in 20 years

Published on 10/07/17 at 09:11am
Image: OpenStax College, Anatomy & Physiology

Emmaus Medical has been handed approval for the first new treatment for sickle cell disease in two decades. The active ingredient is the only major question mark over the drug, with L-glutamine being the primary ingredient in Endari and already cheaply available over-the-counter (OTC) in the US.

The pivotal trial that led to the drug’s approval showed strong benefits in those suffering from sickle cell disease, with a 25% reduction in sickle cell crises and hospitalisations lowered by 33%. Beyond this, it was also found that cumulative hospital days were on lowered by 41% and incidence of acute chest syndrome minimised to 60%.

The study ran for 48 weeks, involving 239 adults and children. The worst of the side-effects exhibited were constipation, nausea, headaches and abdominal pain.

“The approval of Endari is a significant milestone for the sickle cell patient community who has not had an advancement in treatment for nearly 20 years and which now, for the first time ever, has a treatment option for children,” said Yutaka Niihara, Chairman and CEO of Emmaus Life Sciences.  “Endari reinforces our commitment to discovering innovative therapies that help to improve the lives of people with rare diseases. We thank the FDA for its prompt review and look forward to making treatment available to patients as early as this fourth quarter.”

As mentioned by Niihara, the treatment has been approved for children of the ages five and upwards. The company are expected to price the treatment at between $11,000 to $18,000 per year depending on dose.

This is potentially where things might get complicated. Emmaus references its treatment as ‘prescription grade L-glutamine’ but what the difference could be between this and OTC L-glutamine is unclear. This will be of particular interest given that, at the cheaper end of the scale, that could mean a $10,990 different in cost compared with OTC price.

What is not in question is the obvious demand for such a treatment for sickle cell disease, with 100,000 people born with the inherited disorder in the US. The shape of patient’s blood cells are indicated by the name of disorder and this odd shape restricts flow in blood vessels; this can lead to blockages and oxygen starvation of certain organs, resulting in severe pain.

Ben Hargreaves

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