A new drug called Altemia is being developed to treat sickle cell diseases in children, reports BioPortfolio. It has recently been awarded Orphan Drug designation in both the United States and European Union following clinical trials with encouraging results.

Sickle cell diseases (SCD) are life-long genetic conditions that cause red blood cells to be deformed. This leads to symptoms including anaemia, sickle cell crises (extremely painful episodes), and a raised infection risk, amongst others. SCD are more common in people of African or Asian descent, and are thought to affect approximately 100,000 people in the US. Treatment options available to patients at the moment include over-the-counter or prescribed painkillers for pain crises, daily antibiotics to reduce the chance of infections, blood transfusions to counter anaemia, and hydroxycarbamide, an oral medicine that can reduce symptoms. However, hydroxycarbamide does not suit all patients, and it’s usefulness for preventing chronic complications from SCD remain unclear.

Sancilio Pharmaceuticals Company, Inc. is developing a new medicine, called Altemia to provide a new treatment option for children with SCD. Altemia is a pill made up of a mixture of lipids (fats) that works by replacing the lipids destroyed by SCD. By replacing the lost lipids, the medication should reduce anaemia, painful crises, and organ damage in patients.

Following successful results from Phase 2 clinical trials in November 2017, Altemia has been awarded Orphan drug status by the European Union and the US. The Orphan Designation is awarded to drugs being developed to treat serious rare diseases. To qualify in the European Union, the disease needs to affect no more than five people out of every 10,000. Being granted Orphan drug status gives the developers several benefits for developing the drug, including several years of marketing exclusivity, cuts to fees required during the process of bringing the drug to market, and assistance with drug development, although the precise terms differ between the US and EU.

Receiving an Orphan Drug designation is an encouraging sign for the development of Altemia. It shows that the early stages of testing have produced positive results, and that the EU and US are prepared to support the drug throughout the process to make it widely available to patients. If successful, this drug will bring another much-needed treatment option to SCD patients.

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