LAMZEDE is an enzyme replacement therapy that is used for the treatment of adult and pediatric patients with alpha-mannosidosis.
Alpha-mannosidosis is a rare disease where patients have decreased activity of the enzyme alpha-mannosidase, which breaks down mannose containing oligosaccharides (a complex sugar). Oligosaccharides can build up in organs and tissues causing a weakened immune system, frequent lung infections, skeletal abnormalities, and muscle weakness.
LAMZEDE is administered by a healthcare provider using a needle placed in a vein (known as intravenous infusion) once every week.
The FDA approved LAMZEDE based primarily on evidence from one clinical trial (Trial 1) of 25 patients with alpha-mannosidosis. The trial was conducted at seven sites in Europe.
All 25 patients were evaluated for efficacy and side effects.
LAMZEDE was evaluated in one clinical trial of 25 patients with alpha-mannosidosis.
The benefit and side effects of LAMZEDE were evaluated primarily in one clinical trial. Trial 1 enrolled children and adults with alpha-mannosidosis who had a genetic and corresponding enzyme deficiency. The enzyme deficiency was determined to be a good target for therapy with LAMZEDE based on laboratory data. The clinical trial compared patients who received LAMZEDE (the missing enzyme) with patients who never received treatment to replace the missing enzyme. Patients were randomized to receive LAMZEDE or placebo every week for 12 months. Neither the patients nor the healthcare providers knew which treatment was given until after the trial was completed.
The benefit of LAMZEDE was assessed after 12 months of treatment by assessing 3-minute stair climbing test (3MSCT), 6-minute walking test (6MWT), forced vital capacity (FVC), and serum oligosaccharides at 12 months. Patients treated with LAMZEDE experienced a larger average increase in steps on the 3MSCT, a larger average distance in the 6MWT, and larger increase in FVC after 12 months of treatment, compared to patients who received placebo for 12 months. Patients treated with LAMZEDE also experienced a larger average decline in serum oligosaccharides after 12 months of treatment.
Figure 1 summarizes how many males and females were enrolled in the clinical trial used to evaluate the efficacy of LAMZEDE.
Figure 2 summarizes how many patients by race were enrolled in the clinical trial used to evaluate the efficacy of LAMZEDE.
Figure 3 summarizes how many patients by age were enrolled in the clinical trial used to evaluate the efficacy of LAMZEDE.
LAMZEDE improved walkable distance, ability to climb stairs, and lung function in children and adults with alpha-mannosidosis. LAMZEDE also reduced blood levels of oligosaccharides (a complex sugar which is abnormally high in patients with alpha-mannosidosis) in both children and adults with alpha-mannosidosis.
The pivotal trial enrolled only White patients and patients younger than 65 years of age. Only subgroup analyses by sex can be conducted. However, the trial population was too small to enable interpretation of these subgroup analyses .
LAMZEDE may cause serious side effects such as hypersensitivity reactions and infusion associated reactions.
The most common side effects are nasal and throat irritation (nasopharyngitis), fever (pyrexia), headache, and joint pain (arthralgia).
CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.
COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or "sugar pill" that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.