Edgewise Therapeutics, a clinical-stage biopharmaceutical company based in Boulder, Colorado, has recently achieved significant milestones for its EDG-5506 muscular-dystrophy program. The U.S. Food & Drug Administration (FDA) has granted two important designations to the company.
Orphan-Drug Designation for Duchenne and Becker Muscular Dystrophy
EDG-5506 has been granted orphan-drug designation by the FDA for the treatment of Duchenne and Becker muscular dystrophy. This program provides special status to drugs and biologics that focus on diseases and disorders affecting fewer than 200,000 people in the United States. As a result, Edgewise Therapeutics is eligible for an extended marketing exclusivity period against potential competitors.
Rare-Pediatric-Disease Designation for Duchenne
The FDA has also awarded rare-pediatric-disease designation to EDG-5506 for the treatment of Duchenne. This designation allows the company to receive a priority-review voucher upon drug approval. These vouchers can be used to expedite the review process of another drug or sold to other companies.
Advancing EDG-5506 in Multiple Phase 2 Studies
Edgewise Therapeutics is currently progressing with the development of EDG-5506, a small molecule designed to prevent muscle damage caused by contraction in dystrophinopathies like Duchenne and Becker. This promising treatment is being evaluated in multiple Phase 2 studies.
Addressing Unmet Medical Needs
Duchenne and Becker muscular dystrophy are neuromuscular genetic diseases that currently lack cure options. Edgewise Therapeutics aims to address these unmet medical needs with their innovative EDG-5506 program.
