Have you been paying attention to the news? If not, you might have missed that INCRELEX (mecasermin) is now commercially available in Canada! In a news release, biopharmaceutical company Ipsen Biopharmaceuticals Canada Inc. (“Ipsen”) shared that the treatment has now been commercialized following its December 2020 approval. The treatment, designed for pediatric patients with severe primary insulin-like growth factor-1 deficiency (SPIGFD), is the first approved IGF-1 treatment in Canada for this particular patient population.
Normally, our bodies produce a substance called insulin-like growth factor-1 (IGF-1). This substance plays a role in both bone and muscle health and growth. But in patients with SPIGFD, a number of genetic abnormalities prevent the body from producing enough – or any – IGF-1. INCRELEX is a human IGF-1 therapy which helps promote growth in patients. Currently, the treatment is indicated for patients with SPIGFD between the ages of 2-18 years old. The treatment is administered subcutaneously.
Altogether, the approval hinged on data from a clinical trial evaluating the efficacy of INCRELEX for previously untreated patients. 81 patients enrolled. Researchers determined that INCRELEX treatment helped sustain growth for up to 8 years. However, the most response and growth is seen within the first year of treatment. Although INCRELEX was relatively safe and well-tolerated, some adverse reactions did occur. These included:
- Injection site inflammation
- Low blood sugar
- Benign or malignant abnormal tissue growth
Severe Primary Insulin-Like Growth Factor-1 Deficiency (SPIGFD)
Altogether, SPIGFD is caused by genetic abnormalities, such as those within the GHR gene, post-GHR signaling pathway, or other genes which impact IGF-1. According to the Child Growth Foundation:
Severe primary IGF-1 deficiency (SPIGFD) is a medical term used when IGF-1 levels are extremely low or undetectable in the blood. The classical (most well recognised) form of SPIGFD is a genetic condition called Laron syndrome, [where patients] have an abnormal growth hormone receptor (GHR) which does not function properly.
SPIGFD can be diagnosed when patients have a short stature and failure to grow, and when doctors can rule out hypopituitarism, hypothyroidism, malnutrition, or other potential causes. Signs and characteristics include:
- A short stature (2 standard deviations below the average for the age)
- Fatigue and/or lethargy
- Low blood sugar
- Delayed puberty
- Poor muscle strength or endurance