This approach is called substrate reduction therapy.Eliglustat (Cerdelga) (approved in 2014)  is also a small molecule. The compound is believed to work by inhibition of glucosylceramide synthase. Epidemiology
The National Gaucher Foundation (United States) states the incidence of Gauchers disease is about one in 20,000 live births. Around one in 100 people in the general US population is a carrier for type I Gauchers disease, giving a prevalence of one in 40,000. Among Ashkenazi Jews, the rate of carriers is considerably higher, at roughly one in 15.Type II Gauchers disease shows no particular preference for any ethnic group.Type III Gauchers disease is especially common in the population of the northern Swedish region of Norrbotten, where the incidence of the disease is one in 50,000. History
The disease was first recognized by the French doctor Philippe Gaucher, who originally described it in 1882 and lent his name to the condition. In 1902, its mode of inheritance was discovered by Nathan Brill. The neuronal damage associated with the disease was discovered in the 1920s, and the biochemical basis for the disease was elucidated in the 1960s by Roscoe Brady. The first effective treatment for the disease, the drug alglucerase (Ceredase), was approved by the FDA in April 1991. An improved drug, imiglucerase (Cerezyme), was approved by the FDA in May 1994 and has replaced the use of Ceredase. October is National Gauchers Disease Awareness Month in the United States.
History
Results of a Phase I clinical trial were reported in 2016.In December 2019, enfortumab vedotin was approved in the United States for the treatment of adult patients with locally advanced or metastatic urothelial cancer who had previously received a programmed cell death receptor-1 (PD-1) or programmed death ligand 1 (PD-L1) inhibitor and a platinum-containing chemotherapy.Enfortumab vedotin was approved based on the results of a clinical trial that enrolled 125 patients with locally advanced or metastatic urothelial cancer who received prior treatment with a PD-1 or PD-L1 inhibitor and platinum-based chemotherapy. The overall response rate, reflecting the percentage of patients who had a certain amount of tumor shrinkage, was 44%, with 12% having a complete response and 32% having a partial response. The median duration of response was 7.6 months.The U.S. Food and Drug Administration (FDA) granted the application for enfortumab vedotin accelerated approval, priority review designation, and breakthrough therapy designation. The FDA granted the approval of Padcev to Astellas Pharma US Inc.In July 2021, the FDA approved enfortumab vedotin for adults with locally advanced or metastatic urothelial cancer who have previously received a programmed death receptor-1 (PD-1) or programmed death-ligand (PD-L1) inhibitor and platinum-containing chemotherapy, or; are ineligible for cisplatin-containing chemotherapy and have previously received one or more prior lines of therapy.