According to a press release from Takeda Pharmaceutical Company published by For Press Release, the Company has launched a collection of enzyme replacement therapies for use in the treatment of lysosomal storage disorders that are of particular prevalence in India.
The collection comprises three medications — idursulfase for the treatment of Hunter syndrome, velaglucerase alpha for Gaucher disease, and agalsidase alfa for Fabry disease. These lysosomal storage disorders are particularly concerning in India, where their increasing prevalence has been observed for several years now.
About Lysosomal Storage Disorders
Lysosomal storage disorders (LSD) are genetically inherited conditions characterized by the buildup of toxic or other substances in dangerous concentrations in the body’s lysosomes — sacks of digestive enzymes found in almost every cell in the body. Often, these buildups are the result of enzyme deficiencies that make it inefficient, if not outright impossible, for the body to break down certain substances.
There are almost 50 lysosomal storage diseases that affect a diverse array of sites around the body, including the skeleton, heart, and central nervous system — and new LSDs are frequently being discovered.
Hunter syndrome, also known as mucopolysaccharidosis type II, affects a number of sites around the body. Patients are likely to develop a number of identifying abnormal physical characteristics, and may suffer from macrocephaly and hydrocephalus (enlarged head and brain-fluid buildup, respectively), narrowing of the spinal column (which can damage the spinal cord), or enlarged liver and spleen. Almost all affected are men.
Gaucher disease is, most accurately, a group of related conditions whose symptoms and prognosis can vary significantly between types and individual cases. Individuals with non-neurological cases may only experience certain symptoms like liver/spleen enlargement, anemia, or bone abnormalities. Other types of Gaucher disease are called neuronopathic, and can affect the central nervous system in various ways — potentially causing eye dysfunction, seizures, and brain damage.
Fabry disease is an inherited condition characterized by the buildup of a globotriaosylceramide, a kind of fat, in cells throughout the body. Although those born with Fabry disease may show no symptoms early in life, as globotriaosylceramide builds up, it can wreak havoc on vital organs. Some complications of Fabry disease can be potentially fatal, such as kidney failure, cardiac arrest, and stroke.
The launch of this lysosomal storage disease portfolio highlights Takeda’s commitment to India’s unique health challenges, and could mean good things for the thousands of Indians living with LSDs.
What do you think of this exciting news? Do you think the simultaneous roll-out of three lysosomal storage disease drugs will lead to significant improvement in patient outcomes? Share your thoughts with Patient Worthy!