GC Pharma today announced that the European Medicines Agency (EMA) granted orphan drug designation (ODD) to Hunterase ICV (intracerebroventricular, generic name: idursulfase-beta (recombinant)) for the treatment of mucopolysaccharidosis type II (Hunter syndrome) in the European Union.
Source:- GC Pharma
Worldwide, ~2,500 patients are expected to be suffering from this disease. As per the National MPS Society, US, hunter syndrome affects 1 in 100,000 to 1 in 170,000 primarily males. Hunter Syndrome or Mucopolysaccharidosis II (MPS II) is a very rare disease and hence the reported cases incidence/prevalence is very less across all key geographies. As per Thelansis research, the disease is expected to have ~800 prevalent cases in the US and ~850 cases across Europe. In Japan, the prevalence of Hunter Syndrome is estimated to be around ~400 patients
Comprehensive insight on patient segmentation has been provided into the epidemiology section of the Hunter syndrome (Mucopolysaccharidosis Type -II) and its treatment in the 8 MM countries, covering the United States, EU5 (Germany, Spain, France, Italy, UK), Japan, and China
Apart from an approved therapy, Takeda is also investigating SHP631 in Phase I in collaboration with ArmaGen Technologies. SHP631 targets insulin receptors and sulfated alpha-L-iduronic acid. Another product in development, TAK-609, is being suspended. JCR Pharmaceuticals has 3 products under development in its Hunter Syndrome portfolio out of which, 1 product is in Phase II/III, 1 in pre-clinical stages, and another one in development outside of the US. JR-141, which is in Phase II/III stage also targets sulfated alpha-L-iduronic acid and has orphan drug designation. JR-141 is expected to be next in line with the approval for Hunter Syndrome. JCR Pharma is developing JR-032 outside the US and is evaluating JR-441 in Phase I
Thelansis is specialized in pharmaceutical market research and market Insight Report Company, published reports across the therapeutic area which includes both rare / ultra-rare and mainstream indication. Over the period of time, we have built a strong repository of 6,000+ Bio-pharma reports which essentially covers Epidemiology study and Market forecasting based on the KOL opinions. Competitive intelligence and track of trial results throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the major focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making.
Gurugram- Onework Gold Souk mall, Phase I, Sector 43, Haryana-122002, India
Contact no.: +91(124) 404-1731
183-Asylum Street Hartford, CT-06103, USA
USA +1 (267) 244-6955,