The Japanese biopharmaceutical company, Takeda, has launched its enzyme replacement therapy portfolio for lysosomal storage disorders (LSD), to address the unmet needs of patients living with rare diseases in India.
The country has been recording a prevalence in lysosomal storage disorders such as Hunter’s syndrome, Gaucher disease and Fabry disease.
Considering the prevalent form of LSDs in India, Takeda, which is one of the leaders in the rare disease space, brings to the market Idursulfase , Velaglucerase Alpha and Agalsidase Alfa for treating Hunter syndrome, Gaucher disease and Fabry disease respectively.
Hunter syndrome is a serious genetic disorder that interferes with the body’s ability to break down specific mucopolysaccharides, also known as glycosaminoglycans or GAGs. It primarily affects males.
Gaucher is a rare inherited metabolic condition which affects approximately 1 in 100,000 people in the general population. Patients with type 1 Gaucher disease may experience varying symptoms and degrees of disease severity, making it difficult to diagnose.Fabry disease interferes with the body’s ability to break down a specific fatty substance (globotriaosylceramide or Gb3).
The U.S. Food and Drug Administration in 2006 had granted Takeda to market Idursulfase for patients with Hunter syndrome. The use of Idursulfase has shown an increased life expectancy of 11.8 years with 54% reduction in mortality for patients, reported the company.
Velaglucerase Alpha was approved by the U.S. FDA in 2010, showcasing Gaucher patients reach normalization across various clinical parameters like, liver volume, spleen volume, lumbar Spine BMD Z score, blood platelet count and Hb count with a marked reduction in Lyso-Gb1 level 3.