Rare Diseases: Breakthrough Therapy Business Developments (10.16.13)
I – Epidermolysis Bullosa
Scioderm, a North Carolina startup pharmaceutical company, is focusing on developing therapies for orphan products. The company’s lead product is SD-101, an investigational topical cream, which is being developed for the treatment of the rare inherited disease, Epidermolysis Bullosa (EB). In April 2013, Scioderm receives the FDA Breakthrough Therapy Designation for EB treatment.
EB is a rare genetic connective tissue condition that involves very fragile skin that blisters or tears with the slightest trauma or friction. EB patients are known as “Butterfly children” due to the fragile nature of the skin, similar to the fragility of the wings of a butterfly. There is currently no cure or effective treatment, outside of daily wound care, pain management, and preventative bandaging.
On October 15, Scioderm announces the selection of Novella Clinical, a Quintiles full service Clinical Research Organization (CRO), to manage a Phase IIb trial for the treatment of EB. The trial will evaluate SD-101 for the treatment of skin blistering and erosions associated with EB. In January 2014, the clinical trial is scheduled to have the first patient enrolled. The clinical trial will enroll 36 patients at 7 sites in the United States. The trial examines the safety and efficacy of SD-101 with primary endpoint of target lesion closure at 1-month of treatment.
II – Lambert-Eaton Myasthenic Syndrome (LEMS)
Catalyst Pharmaceutical Partners, is a Florida-based pharmaceutical company focusing on developing and commercializing novel drugs to treat rare neuromuscular and neurological diseases, including:
• Lambert-Eaton Myasthenic Syndrome (LEMS)
• Infantile Spasms
• Tourette’s Disorder.
In August 2013, Catalyst’s orphan drug Firdapse (Amifampridine Phosphate) receives the FDA Breakthrough Therapy Designation for LEMS. The drug receives FDA Orphan Drug Designation in November 2009 for LEMS.
LEMS is a rare autoimmune disease with primary symptoms of muscle weakness of the limbs. It is a result of an autoimmune reaction in which antibodies are formed against presynaptic voltage-gated calcium channels in the neuromuscular junction. The prevalence of LEMS is 3.4/1,000,000. Approximately 60% of patients with LEMS have an underlying malignancy, most common of which is small cell lung cancer. Patients who develop LEMS are usually over 40, though it can occur at any age.
On October 15, Catalyst Pharmaceutical Partners announces the recommendation of the Data Monitoring Committee to continue the pivotal Phase III clinical trial for Firdapse for LEMS. The Company expects to complete enrollment in the trial by the end of the fourth quarter of 2013 and to announce top line data from the trial during the second quarter of 2014.
Please Note: “Spinning Device To Mix Tissue Culture Medium” by National Cancer Institute [Public domain] | Wikimedia Commons.