Gaucher Disease Drugs Market Analysis

Therapy Type (Enzyme Replacement Therapy, Substrate Reduction Therapy)

By therapy type, the enzyme replacement therapy (ERT) segment is expected to hold over 73.8% gaucher disease drugs market share by the end of 2037 and is projected to maintain a steady growth rate during the forecast period. The growth of the segment is attributed to the efficacy of ERT in managing symptoms and improving patient quality of life. ERT reduces the lipid buildup in patients by supplementing synthetic glucocerebrosidase.

The FDA has approved Cerezyme (imiglucerase), VPRIV (velaglucerase alfa), and Elelyso (taliglucerase alfa) as ERT drugs. The drugs have shown success in stabilizing symptoms such as enlarged liver/spleen (hepatosplenomegaly) leading to increasing adoption in treatment. Additionally, increasing research in ERT and coverage by government-backed plans on ERT drug costs is positioned to maintain a steady growth of the segment in the global gaucher disease drugs market. For instance, in June 2022, Pharmac, the government agency deciding the funding of medicines in Aotearoa, New Zealand, announced the decision to manage funded access to ERT to standard special authority allowing clinicians a streamlined approach to apply for funded access to ERT for patients.

Substrate reduction therapy (SRT) segment of the market is poised to increase its revenue share by the end of 2037. The growth of the segment is attributed to the rising adoption of SRT as an alternative to ERT for patients who cannot undergo regular blood infusions. SRT enables the body to partially block the production of glucocerebroside. The therapy has emerged as ideal for children and pregnant women, boosting its adoption and fueling the growth of the segment.

Cerdelga (eliglustat) and Zavesca (miglustat) are two FDA approved drugs for SRT. The segment benefits from increasing approval by regulatory bodies of SRT drugs benefitting the growth of the market. For instance, Breckenridge Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration granted final approval of its abbreviated new drug application for Zavesca or Miglusat capsules of 100 mg which will be commercialized by a manufacturer based in the U.S.

Disease Type (Type 1, Type 3)

Based on disease type, the type 1 segment registered the largest revenue share in the gaucher disease drugs market. The segment’s growth is attributed to type 1 gaucher disease being the most prevalent and manageable form of the disease. Type 1 is non-neuronopathic because it usually does not affect the spinal cord and the brain. Additionally, type 1 gaucher disease is responsive to ERT and SRT treatments, leading to a high diagnosis rate that expands demands for targeted treatment of the disease. Pharmaceutical companies are leveraging the burgeoning demands of treatment for type 1 gaucher disease by investing in clinical trials and pushing for regulatory approval so that commercialization can be initiated. For instance, in January 2022, Freeline announced FDA clearance of an investigational new drug application for FLT201 for gaucher disease type 1 and it is the first AAV-mediated gene therapy for patients.

Our in-depth analysis of the global market includes the following segments: