Porphyria is a group of disorders including cutaneous porphyria and acute porphyria. Cutaneous porphyria affects the skin while acute porphyria affects the nervous system. Porphyria cutanea tarda is the most common type of porphyria prevalent in the U.S. The common symptoms of porphyria are abdominal pain, chest pain, increased blood pressure, increased heart rate, muscle weakness, cramping, blisters, itching, swelling, constipation, vomiting, mental disorders, agitation, and seizures. The risk factors associated with porphyria are alcohol abuse, excessive intake of iron, hepatitis C, Human immunodeficiency virus (HIV), menstrual problems, stress, sun exposure, and smoking. Alnylam Pharmaceuticals Inc. is in the process of developing Givosiran, as a subcutaneously administered, investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias (AHPs).
The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the detailed analysis of pipeline and clinical trials.
Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology, and others.