EU5, USA, and Japan Alpha Mannosidosis (Enzyme Replacement Therapy) ERT Market: By Type (Type I and Type II), and Country

EU5, USA, and Japan Alpha Mannosidosis (Enzyme Replacement Therapy) ERT Market is estimated to account for US$ 33.7 million in 2023 and is projected to grow at a significant CAGR over 2023-2030 owing to the launch of Velmanase alfa, the one and only approved enzyme replacement therapy for alpha mannosidosis by the EMA in all the European countries. The increasing number of diagnosed patients with Type 1 and Type 2 AM in the UK, Germany, France, the United States, and other countries coupled with the availability of reimbursement policies for ultra-rare disorder drugs in the United States, Germany is expected to create lucrative opportunities for the growth of the market. In addition, there is no ERT in clinical trials or in the market for alpha mannosidosis, in the USA, there is an increase in trend for the co-insurance of the drugs and payers showing interest towards the drugs that are providing co-insurance programs. For instance, Kanuma (Sebelipase Alfa), offering a commercial co-pay program can limit out-of-pocket expenses to $5 for eligible patients with commercial insurance. Moreover, more than 70 patients in Germany are readily eligible for Alpha Mannosidosis ERT treatment and in the UK, according to NICE, more than 30 patients are eligible for ERT and many more are yet to be diagnosed. However, a very limited number of patients for ultra-rare diseases and rejection for drug reimbursement by the HTA bodies such as NICE (, and the high cost of enzyme replacement therapy are anticipated to restrain the growth of the market over the forecast period.

Rising number of patients diagnosed with Type I and Type II Alpha Mannosidosis:
The growing incidence of Alpha Mannosidosis disease globally is projected to drive the sales of Alpha Mannosidosis therapeutics market over the forecast period. According to Germany Health Technology and Assessment (HTA), approximately 70 – 140 people are affected with Alpha Mannosidosis in Germany. According to NICE reports, approximately 460 people in European countries are affected with Alpha Mannosidosis. Moreover, according to National Organization for Rare Disorders, Alpha Mannosidosis incidence is generally placed at approximately 1 in 500,000 births across the globe. Additionally, according to NICE, more than 30 patients are already diagnosed in England and around 13 patients are eligible for Enzyme Replacement Therapy soon after the launch of the product in the market. Moreover, among the disease types, the incidence rate of Type 3 disease diagnosed patients' number is very less common compared with Type 2 that contribute revenue generation for Velmanase alfa (Lamzede) over the forecast years as the product is intended to treat Type 1 and Type 2 alpha mannosidosis diagnosed patients.

Key Developments:

• In February 2023, the US Food and Administration approved velmanase alfa-tycv for the treatment of non-central nervous systems manifestations of alfa-mannosidosis in adult and pediatric patients.
• For instance, in April 2018, Chiesi has announced that the European Commission granted the marketing authorization for Lamzede® (Velmanase alfa), the first enzyme replacement therapy for the treatment of non-neurological manifestations in patients with mild to moderate alpha?mannosidosis (AM), an ultra-rare progressive and debilitating disease)
• In 2013, Chiesi announced that it had signed a definitive agreement with Sunstone Capital to acquire Zymenex Holding A/S and its related group of companies.