The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Tetra Bio-Pharma’s investigational cannabinoid cream PPP004 (THC-CBD) for the treatment of epidermolysis bullosa, the company has announced.
Orphan drug status aims to encourage the development of therapies for rare diseases (those affecting less than 200,000 patients in the U.S.), through benefits such as exemption from FDA fees, financial incentives for clinical development and commercialization, and seven years of market exclusivity if granted approval.
“We are very pleased to have received this new Orphan Drug Designation (ODD) from the FDA,” Guy Chamberland, PhD, CEO and chief regulatory officer of Tetra, said in a press release. “Orphan designation qualifies PPP004 for development incentives that … are critically important to a small biopharma.”
Cannabinoids are compounds found in the cannabis plant. The most common are cannabidiol, or CBD, and tetrahydrocannabinol, or THC. While CBD does not produce psychoactive activity and has analgesic and anti-inflammatory properties, THC is classified as a mild sedative at low doses and a hallucinogen at high doses.
PPP004 cream is made of equal parts THC and CBD, and is available in three different strengths of 2%, 1%, and 0.5%. Both THC and CBD are sourced from manufacturers following good manufacturing practices, Tetra said.
The cream will be available in predefined doses delivered using an airless pump system. According to the company, this guarantees the delivery of the same dose of the active pharmaceutical ingredients, CBD and THC, while avoiding their degradation due to light and oxygen.
PP004 is meant to help manage pain and itch in epidermolysis bullosa, which impair the daily lives of patients despite current treatments.
Tetra expects to launch a proof-of-concept clinical trial for PPP004 in the near future.
“Our preclinical research was completed for taking PPP004 into human clinical trials. We have a vast experience in the development of topicals … and expect to rapidly bring this product into trials,” said Melanie Kelly, PhD, chief scientific officer of Tetra. “In the coming days, we will be reaching out to physicians specialized in the treatment of this disease to discuss a potential proof of concept clinical trial.”
Added Chamberland: “Tetra will be assessing to launch a proof of concept trial in the coming months and will be requesting a Type B meeting with the US FDA to discuss the marketing requirements for this Orphan Drug indication.”
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