What is LAMZEDE prescribed for?
For the treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients.
What is the name of the drug and what does it do?
LAMZEDE (generic name: velmanase alfa-tycv), pronouonced “lam-zed,” is a medicine made from human genes that helps treat the non-brain symptoms of alpha-mannosidosis, like skeletal abnormalities, hearing loss, and intellectual disability in both adults and kids.
How does it work?
LAMZEDE is a medicine that helps people with alpha-mannosidosis, a rare genetic condition.
In people with alpha-mannosidosis, the body doesn’t have enough of a special enzyme called alpha-mannosidase.
This enzyme normally helps break down certain substances in the body. When these substances build up, it can cause problems like intellectual disability, bone issues, and hearing loss.
LAMZEDE steps in to provide the missing enzyme.
By giving the body more alpha-mannosidase, LAMZEDE helps break down these substances better.
This can help reduce symptoms and improve the health of people with alpha-mannosidosis.
So, LAMZEDE works by filling in for the missing enzyme, making it easier for the body to handle these substances and hopefully making people feel better.
What did the research discover?
In the clinical trials, 15 patients tried LAMZEDE for 52 weeks in Trial 1. One patient (7%) had kidney issues.
In Trial 2, 5 kids used LAMZEDE for about 121 weeks, with one child (20%) having serious problems.
In Trial 3, 33 patients took LAMZEDE for around 89 weeks if they were adults and 155 weeks if they were kids.
Some common issues were stomach pain, bruises, and weight gain.
3% of patients had kidney problems, one got a skin condition called immunoglobulin A vasculitis, and one had seizures that might be linked to LAMZEDE.
Reactions during infusions were more likely in patients with certain antibodies. In Trial 1, 1 out of 5 patients with these antibodies had a bad reaction.
In Trial 2, 2 out of 4 patients with these antibodies had reactions. In Trial 3, 3 out of 33 patients had reactions.
In Trial 2, 4 out of 5 kids developed antibodies, and 3 of them had antibodies that stopped LAMZEDE from working.
In Trial 3, 5 patients had antibodies before treatment, and 4 got them after. The trials showed that LAMZEDE helped patients do better in tests like climbing stairs and walking.
What are some of the side effects?
The side effects listed are from the product manufacturer. As health scientists, we share this information to keep you informed without causing undue worry. Many side effects diminish over time, and not all patients experience them. If prescribed medication, take it as directed and consult your healthcare professional if you have any concerning side effects.
- Breathing difficulty
- Low blood pressure
- Common cold
- Fever
- Headache
- Joint pain/discomfort
The recommended dosage of LAMZEDE is 1 mg/kg (actual body weight) administered once every week as an intravenous infusion.
Source:
Novel Drug Approvals for 2023 [Internet]. U.S. Food and Drug Administration. [cited 2024 Jul 1]. Available from: https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2023
