Altemia Granted Orphan Drug Status in EU as Potential Therapy for Pediatric Sickle Cell Disease

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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The European Medicines Agency recently granted orphan drug status to Sancilio PharmaceuticalsAltemia for the treatment of sickle cell disease (SCD) in pediatric patients, the company announced.

Altemia is a specific formulation of lipids designed to replenish the elements needed for healthy red blood cells that are damaged in SCD.

The therapy is encapsulated in a small, soft gelatin capsule taken once daily. Using Sancilio’s proprietary Advanced Lipid Technologies platform, Altemia is a lead candidate for the treatment of SCD.

Topline data from a Phase 2 trial (NCT02973360) announced in November 2017 showed that Altemia achieved both primary and secondary goals in SCD patients ages 5 to 17.

The trial’s primary goal was to measure the change in the concentration of fatty acids, an important component of cellular membranes, in the red blood cell membrane of patients treated with Altemia or a placebo. Investigators tested three doses of Altemia. The concentration of fatty acids changed significantly after four weeks of treatment.

Top-line results also showed a clinically meaningful reduction of vaso-occlusive episodes. After eight weeks of treatment, additional clinical manifestations of SCD also showed statistically significant improvement.

No serious adverse events were observed during the study, and most participants received the green light to continue receiving treatment in an open-label extension study to evaluate Altemia’s safety and effectiveness.

“The European Commission designating Altemia as an Orphan Medicine for the EU continues to advance the pathway for our lead clinical compound,” Geoffrey Glass, chief executive officer at Sancilio, said in a press release. “The pediatric population of sickle cell disease patients needs and deserves more therapeutic options, and we are excited about the opportunity to gain advice from the European Commission on advancing Altemia in the EU markets.”

Orphan drug designations are granted in Europe for treatments or products that seek to improve the lives of patients living with rare diseases. These are diseases that affect no more than five per 10,000 European Union citizens. Orphan drugs may receive up to 10 years of market exclusivity upon approval, as well as a reduction in fees and assistance with applications and protocols.

“We are extremely encouraged to receive this designation, further supporting the significant clinical and regulatory progress made in advancing Altemia’s development program,” said Adrian L. Rabinowicz, chief medical officer at Sancilio.

The U.S. Food and Drug Administration granted Altemia rare pediatric drug designation in September 2017. The agency also granted Altemia orphan drug designation in 2015.