Orphan Drug Status for MMV Drug DHA-Piperaquine

Status is excellent news for the upcoming submission for registration of this drug.

25 Jul 2007

The European Agency for the Evaluation of Medicinal Products (EMEA) granted orphan drug status to Dihydroartemisinin-Piperaquine (DHA-PPQ) in July 2007.

DHA-PPQ, an artemisinin combination therapy, was developed over three years by MMV in collaboration with pharmaceutical partners Sigma Tau Industrie Farmaceutiche reunite S.p.A of Italy and Holley Pharmaceuticals of China, and with Guangzhou University (China) and the University of Oxford.

Dihydroartemisinin is known to have anti-malarial activity. It is a derivative of artemisinin, a naturally occurring substance extracted from the Chinese plant Artemisia annua.

Piperaquine is a member of the 4-aminoquinolone group of anti-malarial drugs. It was synthesised in the 1960’s and was extensively used over a 20-year period in China and Indochina for both prophylaxis and treatment of malaria.

Combination therapy is a strategically viable option for delaying development and selection of resistant parasites, and artemisinin combination therapy has been recommended by the World Health Organization as first-line treatment for malaria.

DHA-PPQ has successfully completed Phase III clinical trials in adults and children, to the highest international Good Clinical Practice level, and is now ready for registration. One Phase- III trial in Southeast Asia (India, Laos and Thailand) compared DHA-PPQ with artesunate mefloquine in 1050 patients. The second Phase-III trial with 1500 patients at 5 sites in Africa used artemether-lumefantrine as the comparator drug.

DHA-PPQ specifically offers:

  1. An absorption not dependent on food. This drug does not require co-administration with food. This is medically significant for the proposed patient population.
  2. Improved compliance. For a 60 kg patient, the treatment requires only three doses of three tablets over three days (nine tablets in all). This provides clear benefits in terms of patient compliance and tolerability.

It is hoped that the orphan status for DHA-PPQ will facilitate faster registration of this efficacious new antimalarial, and thus enable quicker access to patients awaiting new medicines for malaria.