EU Grants Orphan Drug Designation to Cannabinoid Cream PPP004 for EB

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by Patricia Inácio, PhD |

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Tetra Bio-Pharma’s investigational cannabinoid cream PPP004 (THC-CBD) has been granted orphan drug status by the European Medicines Agency (EMA) to treat epidermolysis bullosa.

This follows the orphan drug designation granted to PPP004 by the U.S. Food and Drug Administration (FDA) for the same purpose in April 2020.

These designations are meant to support the clinical development of potential rare disease therapies by providing several benefits. In the European Union, this designation is granted to treatments addressing life-threatening or chronic debilitating disorders or conditions that affect less than five in 10,000 people. The benefits include cost reduction in drug development, an accelerated review process, and market exclusivity for 10 years upon regulatory approval.

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“This important designation is a milestone in the development of PPP004 as Tetra continues to execute its regulatory strategy in Europe. It also highlights the critical need for effective treatment options for people living with EB,” Guy Chamberland, PhD, Tetra’s CEO and chief regulatory officer, said in a press release.

The cannabis plant contains hundreds of components, of which more than 100 are called cannabinoids. Cannabidiol (CBD) and tetrahydrocannabinol (THC) are the main cannabinoids. While CBD lacks psychoactive activity (meaning it does not cause a high) and has anti-inflammatory as well as analgesic properties, THC has been reported to increase anxiety, sedation, and psychotic symptoms (having difficulty in recognizing what’s real and what’s not).

PPP004 is composed of THC and CBD. Its formulation is manufactured per good manufacturing practices, Tetra said, which ensures production according to quality standards.

The treatment candidate was designed to help manage pain and itch in epidermolysis bullosa, two symptoms known to have detrimental effects on patients’ daily lives, despite current treatments.

If approved, PPP004 will be made available in an airless pump system. According to Tetra, this helps ensure the delivery of the same dose of both active ingredients, CBD and THC. Moreover, it protects the compounds from degradation, triggered by exposure to light and oxygen.

According to the press release, Tetra is ready to test PPP004 in clinical trials.

PPP004 containing only CBD has previously been granted orphan drug status by both the FDA and the EMA for treatment of EB.