Gaucher Disease, sometimes referred to as Lipodystrophy is one of many inherited lipid accumulation diseases also called lipid deficiency diseases. Gaucher Disease either doesn't develop sufficient of the enzyme lipolysis required to break down fats or has insufficient enzymes which don't function properly. Gaucher disease is a very rare disease and thus research and development for its drug discovery have become a major priority for many pharmaceutical companies. As a result of this, regulatory agencies are increasingly approving safe and effective drugs for treatment. Recently, in March 2019, the U.S. Food and Drug Administration (FDA) approved a new orphan drug Elelyso (taliglucerase alfa), manufactured by Pfizer Inc., for the treatment of Gaucher disease. Hence, such factors can stimulate the growth of the Gaucher disease treatment market.
When it comes to the regional footprint, the U.S. seems to be exhibiting lucrative traction in the Gaucher disease treatment market. This is typically due to increasing awareness regarding diagnosis and treatment of the disease combined with the growing patient pool in the country. Other countries including France and Germany are showing promising signs due to rising disposable income. Recently, in February 2021, M6P Therapeutics received the U.S. FDA orphan drug status for its M6P Gene Therapy intended for Gaucher disease.
There are three types of Gaucher Disease: Gaucher Type I, Gaucher Type II and Gaucher Type III. Gaucher Type I is the least common and occurs most often in males. Symptoms include fat deposits around the chest and abdomen, increased appetite, muscular weakness and stiffness of ankles and knees, constipation, swelling, and jaundice. However, there are certain hurdles present that could limit the development of the market. For instance, a lot of people are not aware of this disease as well as its symptoms, diagnosis, and treatment, especially in the rural population. Therefore, these factors can potentially inhibit the growth of the Gaucher disease treatment market shortly.
Patients with Gaucher Disease are at risk for many complications if not diagnosed early and treated adequately. Unfortunately, there is no cure for Gaucher Disease, and those who suffer from this disease are faced with a life expectancy of about a year or less. The Gaucher disease, even though it is rare, the incidence has gone up in the recent past. As per the National Organization for Rare Disorder (NORD), Gaucher disease affects around 6,000 people in the U.S. every year. Hence, such factors can potentially bolster the growth of the Gaucher disease treatment market shortly.