Alpha-mannosidosis

Historically, treatment for alpha-mannosidosis has mostly been based on treating and managing the different symptoms that people with the condition have.

A hematopoietic stem cell transplant (HSCT) can also be used to treat some of the symptoms of alpha-mannosidosis. These transplants can improve the levels of alpha-mannosidase in the body to treat some of the symptoms of the condition. A HSCT does not usually improve the problems with the bones, problems with the eyes, or the problems that people with alpha-mannosidosis can have with their mental development and the brain. HCST has significant risks to the person undergoing the procedure and is not done on a routine basis. The risks and benefits of HSCT should be discussed with your doctor.

Treatment options for alpha-mannosidosis using enzyme replacement therapy emerged in January 2018. The European Medicines Agency (EMA) granted a marketing authorization under exceptional circumstances in the European Union for Lamzede (velmanase alfa, Chiesi Farmaceutici S.p.A.) to treat alpha-mannosidosis. Lamzede is a long-term intravenous enzyme replacement therapy for adults, adolescents, and children with mild to moderate forms of alpha-mannosidosis. Details available from European Medicines Agency announcement.

In September 2022, Chiesi Global Rare Diseases announced that the U.S. Food and Drug Administration (FDA) accepted a Biologics License Application for velmanase alpha, Chiesi's enzyme replacement therapy, for treating alpha-mannosidosis. Velmanase alpha was granted priority review, with the FDA setting an action date in the first half of 2023 under the Prescription Drug User Fee Act. The FDA gives priority review to therapies with the potential to provide important improvements in the diagnosis, prevention, or treatment of serious conditions. With this designation, the review period required by the FDA is shortened. To view more detailed information see the Chiesi Global Rare Diseases announcement.

Treatment for alpha-mannosidosis is mostly based on treating and managing the different symptoms that people with the condition have. However, in recent years, newer treatment options have arisen.

A hematopoietic stem cell transplant (HSCT) can also be used to treat some of the symptoms of alpha-mannosidosis. These transplants can improve the levels of alpha-mannosidase in the body to treat some of the symptoms of the condition. A HSCT does not usually improve the problems with the bones, problems with the eyes, or the problems that people with alpha-mannosidosis can have with their mental development and the brain. HCST has significant risks to the person undergoing the procedure and is not done on a routine basis. The risks and benefits of HSCT should be discussed with your doctor.

There are orthopedic surgeries that individuals with alpha-mannosidosis can have to relieve some of the pain and other complications from their bone problems. There are antibiotics and vaccinations available for people with alpha-mannosidosis that can prevent some of the infections that may be common for people with this condition. Hearing aids can be used to improve nerve related hearing loss, and glasses can be used to help with vision problems.

Depending on a person's specific symptoms of Alpha-mannosidosis, there may be other treatment methods that they might find helpful.

Treatment options for alpha-mannosidosis using enzyme replacement therapy emerged in January 2018. The European Medicines Agency (EMA) granted a marketing authorization under exceptional circumstances in the European Union for Lamzede (velmanase alfa, Chiesi Farmaceutici S.p.A.) to treat alpha-mannosidosis. Lamzede is a long-term intravenous enzyme replacement therapy for adults, adolescents, and children with mild to moderate forms of alpha-mannosidosis. Details available from European Medicines Agency announcement.

In September 2022, Chiesi Global Rare Diseases announced that the U.S. Food and Drug Administration (FDA) accepted a Biologics License Application for velmanase alpha, Chiesi's enzyme replacement therapy, for treating alpha-mannosidosis. Velmanase alpha was granted priority review, with the FDA setting an action date in the first half of 2023 under the Prescription Drug User Fee Act. The FDA gives priority review to therapies with the potential to provide important improvements in the diagnosis, prevention, or treatment of serious conditions. With this designation, the review period required by the FDA is shortened. To view more detailed information see the Chiesi Global Rare Diseases announcement.