The U.S. Food and Drug Administration has granted orphan drug designation to RaphaLX™ for the treatment of patients with amyotrophic lateral sclerosis (ALS). Under the U.S. Orphan Drug Act, the FDA’s Office of Orphan Products Development provides special status and incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the U.S. Orphan Drug Designation (ODD) conveys up to seven years of marketing exclusivity if the compound receives regulatory approval from the FDA. Other incentives from ODD include development incentives such as tax credits related to clinical trial expenses, exemption from the FDA-user fee and FDA assistance in clinical trial design. The granting of Orphan Designation does not alter the standard regulatory requirements, timing or process for obtaining marketing approval. The safety and effectiveness of a drug must be established through adequate and well-controlled studies.
FDA Approval Process
Investigative New Drug (IND) Phase
The IND phase is the means by which the company obtains permission to ship an experimental drug or a biological products across state lines (usually to clinical investigators) before it can approved to be administered to humans. The FDA reviews the IND application for safety to assure that research subjects will not be subjected to unreasonable risk. If the application is cleared, the candidate drug usually enters a Phase 1 clinical trial.
PHASE 1: Toxicity study
PHASE 2: Efficacy trials
PHASE 3: Toxicity study with larger population sampling
PHASE 4: Commercialization development