ALBUQUERQUE, N.M.--(BUSINESS WIRE)--Dec. 15, 2004--The Office of Orphan Products Development of the U.S. Food and Drug Administration has granted Orphan Drug status to Maas BiolAB, LLC for cyclosporin for the treatment of amyotrophic lateral sclerosis and its variants. Pre-clinical studies in the amyotrophic lateral sclerosis (ALS) transgenic mouse model have shown impressive neuroprotective properties of cyclosporin in slowing ALS and extending lifespan.

Orphan Drug designation is given by the FDA to products that treat rare diseases affecting less than 200,000 Americans. The designation provides significant advantages including a seven-year exclusivity after marketing approval, reduced filing fees, 50% clinical development tax incentives, and eligibility for grant funding.

ALS, or Lou Gehrig's disease, together with its variants, affects between 30,000 to 50,000 Americans, with 5,000 new cases of ALS diagnosed yearly. This neurodegenerative disease causes progressive death of motor neurons in the brain and spinal cord. The loss of strength leading to complete paralysis reduces survival to an average of 3 years. The ability to think normally and feel sensation is preserved until the end of life. While ALS mostly strikes adults, some variants such as spinal muscular atrophy afflict children.

Cyclosporin is a unique neuron-protecting drug, with the advantage that it is the only one in development that is a direct mitochondrial neuroprotectant. Cyclosporin targets the mitochondria for stabilization and preservation, blocking both the apoptotic and necrotic pathways that cause neuronal death. The mitochondria are "waterproofed" by cyclosporin and neurons are able to continue functioning under the conditions of attack seen in ALS. Maas BiolAB is raising funds to conduct Phase 2a clinical trials in ALS.