Cytokinetics Receives Orphan Drug Status for ALS Treatment

Cytokinetics, a biotechnology company developing small molecule therapeutics for multiple diseases, has been granted orphan drug status by the U.S. Food and Drug Administration for its drug candidate to treat amyotrophic lateral sclerosis, or Lou Gehrig’s disease. The San Francisco-based company plans to start a Phase II clinical trial of the drug, CK-2017357, on ALS patients in the first half of 2010. Orphan drug status is given to drugs that offer the potential to treat rare diseases. An estimated 30,000 Americans suffer from ALS, a neurogenerative disease that affects the nerve cells in the brain and the spinal cord that control muscle movement. Patients may experience muscle weakness, twitching, speech problems, and, in later stages, difficulty breathing and swallowing.  ALS typically affects patients between the ages of 40 and 70. There is no cure for the disease. Other companies working in this space include Synapse Biomedical and Obura Company. Cytokinetics is developing a class of drugs to activate the skeletal sarcomere, a cytoskeletal structure instrumental in skeletal muscle contraction. Promising data from a Phase I clinical trial of CK-2017357 was announced in January. The company also specializes in treatments for cardiac muscle contractility, smooth muscle contractility, and oncology.

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