The U.S. Food and Drug Administration (FDA) on February 17, 2023 announced the approval the first-ever enzyme replacement therapy (ERT) for the treatment of rare alpha-mannosidosis, a genetic disorder that affects an individual’s ability to break down complex sugars.
Alpha-mannosidosis is a rare lysosomal storage disorder that is caused by a deficiency in the enzyme alpha-mannosidase. This deficiency leads to the accumulation of complex sugars in the body, which can cause a range of symptoms such as intellectual disability, skeletal abnormalities, hearing loss, and recurrent infections. This approval marks a significant milestone in the treatment of rare diseases.
Trials Indicate Improved Quality of Life
The newly approved ERT, known as Lamzede (velmanase alfa) and manufactured by Chiesi, is a recombinant human alpha-mannosidase enzyme that replaces the missing enzyme in the body. The therapy is administered through weekly 10mg injection.
The approval of Lamzede was based on the results of a clinical trial involving 25 patients with alpha-mannosidosis. The trial demonstrated that Lamzede improved patients’ endurance, walking ability, and respiratory function. Additionally, the treatment was well-tolerated with no serious adverse reactions reported.
The approval of Lamzede is a significant breakthrough for individuals living with alpha-mannosidosis. It provides a new treatment option that has the potential to improve their quality of life and slow the progression of the disease.
Not a Cure
However, it is important to note that Lamzede is not a cure for alpha-mannosidosis. Patients will still require ongoing medical management and monitoring. Additionally, as with all medications, there is a risk of side effects with Lamzede. Patients should discuss the risks and benefits of the treatment with their healthcare provider.
Conclusion
In conclusion, the FDA’s approval of Lamzede marks a significant milestone in the treatment of rare diseases. This new therapy provides a promising new treatment.
Click here to read the full FDA Announcement