The FDA-Approved Medical Marijuana Clinical Trial for Kids with Epilepsy

In line with the Federal Food, Drug and Cosmetic (FD&C) Act, the role of the FDA is to evaluate research carried out by scientific investigators or manufacturers by reviewing data submitted to them in the form of an application for approval. FDA approvals are aimed at assuring the public that a drug product is in compliance with statutory standards put in place to ensure both safety and efficacy. On FDA’s website, the organization responds to concerns regarding the FDA and Marijuana. According to the FDA through information provided under its 2 of 3 main divisions; CDER and CBER, it is important to note that they have not to date approved any marketing application for a drug product that contains or is a derivative of botanical marijuana. The two of the three main divisions are the Center for Drug Evaluation and Research, the Center for Biologics, Evaluation, and Research. The FDA’s position remains that products derived from marijuana have not been found to be effective or safe for any indication or the treatment of any condition or disease.

Be that as it may, the FDA agrees that to assess the effectiveness and safety of marijuana for medical use, the study of marijuana in clinical trial settings is a requirement. Towards this endeavor, the FDA has pledged support and will continue facilitating companies whose work is committed to introduce appropriately, quality products that are effective and safe into the market. Research concerning the use of marijuana for medical reasons should include scientific foundations. GW Pharmaceuticals is one such company that has been allowed by the FDA to carry out studies on an “Investigational New Drug.”GW Pharmaceuticals as a biopharmaceutical company that has proprietary cannabinoid product platform from which they are keen to make discoveries, develop, and commercialize novel therapeutics. The drug product being investigated by GW Pharmaceuticals is being developed to treat seizure disorders and contains cannabidiol. Through clinical trials enrollment or the expanded access program for marijuana for medical use, manufacturers carrying out research on investigational drugs can provide access of their drug product to individual patients according to the provisions of FD&C Act. The ultimate decision to provide patients with experimental therapies lies with the sponsor company, the FDA is only tasked with granting permission for the use of an experimental therapy.

GW Pharmaceuticals has a drug known as Epidioxeldeveloped for children under the category of pediatric epilepsy, through which the potential of the cannabinoid CBD is being investigated.DravetSyndrome is a form of childhood epilepsy considered treatment –resistant, catastrophic, and rare, that Epidiolex is expected to be able to treat. Since the FDA had already awarded the designation of orphan drug for Epidiolex in Dravet Syndrome’s treatment and approved the company’s application for ‘Investigational new Drug,’ the company is confident and is moving to develop a program with the aim of seeking FDA’s market authorization. This market authorization if granted by the FDA would allow the company to provide children with Dravet syndrome and who have exhausted all other therapeutic options with a cannabidiol prescription. This will however have to wait until phase 2 and phase 3 of the FDA-approved medical marijuana clinical trials are complete in 2015 and feedback from FDA in the future. Under a long-term open label extension, all patients (80 patients) that participate in the study will be eligible to receive Epidiolex.

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