Health Canada Approves INCRELEX First and Only Recombinant Human Insulin-like Growth Factor-1 (IGF-1) Therapy in Canada for the Treatment of Severe Growth Failure Associated with Ultra-Rare Condition in Children and Adolescents

MISSISSAUGA, ON, March 16, 2021 /CNW/ - Ipsen Biopharmaceuticals Canada Inc. today announced the commercial availability of INCRELEX^® (mecasermin) for the treatment of growth failure in children and adolescents from 2 to 18 years with confirmed severe primary insulin-like growth factor-1 deficiency (SPIGFD), following its Health Canada approval in December 2020.¹

INCRELEX^® is the first and only recombinant human IGF-1 therapy approved in Canada for children and adolescents with SPIGFD, an ultra-rare condition that affects the growth of fewer than five people per 10,000 globally.²

“Until now, there has been no treatment for children and adolescents with SPIGFD in Canada, and now we can do more to help patients,” said Dr. Cheri Deal, past Chief of Pediatric Endocrinology and Diabetes at CHU Ste-Justine. “Arriving at an accurate diagnosis can be complex and may take several trips to multiple physicians before a short stature disorder is diagnosed. It’s critical we investigate the children at the lower end of the growth chart and diagnose early because treatment is ineffective once the growth plates close. INCRELEX^® is an important milestone, providing hope for families who have a SPIGFD diagnosis.”

The approval of INCRELEX^® by Health Canada was based on results from a clinical study involving 81 treatment-naïve patients with SPIGFD, with a primary efficacy endpoint of height velocity, which was compared with pre-treatment levels.³ The trial showed that long-term INCRELEX^® treatment resulted in a sustained increase in height velocity above pre-treatment levels for up to eight years, with the largest response observed in the first year.⁴

IGF-1 is a hormone important for the growth of bones and muscles and is normally produced by the body.⁵ A synthetic form of IGF-1, INCRELEX^® stimulates statural growth in children who are IGF-1 deficient, enabling growth.⁶

“We are very pleased INCRELEX^® has received Health Canada approval, which is great news for the SPIGFD community,” said Ed Dybka, General Manager, Ipsen Biopharmaceuticals Canada. “Our mission is to give hope to those with serious and difficult-to-treat diseases – no matter the size of the patient population – which is why Ipsen is proud to make INCRELEX^® available to Canadian children and adolescents with SPIGFD who have previously had no treatment available to them. With patients at the heart of everything we do, the approval of INCRELEX^® is an example of our commitment to meeting the needs of Canadians with rare and serious diseases and our drive to provide new therapies for the rare disease community.”

About SPIGFD
SPIGFD is defined by a height standard deviation score ≤ –3.0; basal IGF-1 levels below the 2.5th percentile for age and gender; growth hormone sufficiency; and exclusion of secondary forms of IGF-1 deficiency, such as malnutrition, hypopituitarism, hypothyroidism, or chronic treatment with pharmacologic doses of anti-inflammatory steroids.⁷

Children with SPIGFD have abnormally low levels of IGF-1, a hormone made primarily in the liver. IGF-1 regulates growth by affecting development of body tissues such as the growth plates of the bones.⁸ SPIGFD presents as growth failure and can potentially be associated with metabolic and dysmorphic abnormalities which can vary in severity based on the underlying genetic issue.⁹

SPIGFD includes patients with mutations in the GH receptor (GHR) gene/Laron’s syndrome, post-GHR signaling pathway, and IGF-1 gene defects; they are not GH deficient, and therefore, they cannot be expected to respond adequately to exogenous GH treatment.¹⁰

Short stature is defined as a height more than two standard deviations below the mean for age.¹¹ Studies show compared to those with normal height, children with short stature experience poorer health-related quality of life.¹² Their size can impact their social relationships, self-esteem, and cognitive functioning.¹³

About INCRELEX^®
INCRELEX^® (mecasermin) is indicated for the treatment of growth failure in children and adolescents from 2 to 18 years with confirmed SPIGFD.¹⁴ INCRELEX^® is administered by subcutaneous injection.¹⁵

The most common adverse events (AEs) reported were hypoglycemia, hypersensitivity, injection site hypertrophy, headache and snoring. Hypoglycemia was the most frequently reported AE.¹⁶ There is an increased risk of benign and malignant neoplasia in children and adolescents treated with INCRELEX^®, since IGF-1 plays a role in the initiation and progression of benign and malignant tumours.¹⁷ INCRELEX^® is contraindicated for children or adolescents with active or suspected neoplasia, or any condition or medical history which increases the risk of benign or malignant neoplasia.¹⁸

About Ipsen in North America
Ipsen (Euronext: IPN; ADR: IPSEY) is a global biopharmaceutical company focused on innovation and specialty care. The company develops and commercializes innovative medicines in three key therapeutic areas – Oncology, Neuroscience and Rare Diseases. At Ipsen, we focus our resources, investments and energy on discovering, developing and commercializing new therapeutic options to provide hope for patients whose lives are challenged by difficult-to-treat diseases. Ipsen’s North American operations are located in Cambridge, Massachusetts, one of the company’s three global hubs. Based in the heart of Kendall Square, our fully integrated biopharmaceutical business includes Commercial, Research & Development, Manufacturing, and Global External Innovation and Partnering. Combined with Ipsen Biopharmaceuticals Canada Inc., our Canadian headquarters in Mississauga, Ontario, and other locations, Ipsen employs approximately 600 people in North America. For more information, please visit www.ipsenus.com or www.ipsen.ca.

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SOURCE Ipsen Biopharmaceuticals Canada Inc.