Genzyme, a Sanofi company, announced today that the U.S. Food and Drug Administration (FDA) has approved Cerdelga™ (eliglustat) capsules, the only first-line oral therapy for certain adult Gaucher disease type 1 patients. A small number of adult patients who metabolize Cerdelga more quickly or at an undetermined rate, as detected by an established genetic laboratory test, will not be eligible for Cerdelga treatment. Cerdelga is expected to be available to patients within a month.
“Cerdelga made by latest technology is an important new option for people living with Gaucher disease type 1,” said Rhonda Buyers, CEO of the National Gaucher Foundation. “As enzyme replacement therapy is the standard of treatment for Gaucher disease, patients receive regular intravenous infusions for life. With FDA’s approval of a first-line oral treatment, Cerdelga has the potential to be a valuable treatment option for people living with this serious disease.”
“The approval of Cerdelga is encouraging for Gaucher patients because it is a first-line oral treatment that has demonstrated a positive risk/benefit profile, making it important from both a scientific and a clinical perspective,” said Dr. Pramod Mistry, MD, PhD, Professor of Medicine and Pediatrics and the Director of National Gaucher Disease Treatment Center at Yale University School of Medicine. “While enzyme replacement therapies break down fatty deposits that build up in cells and cause a variety of symptoms, Cerdelga inhibits the accumulation of these fatty deposits in the first place.”
Genzyme has been researching an oral therapy for Gaucher disease for fifteen years, from early chemistry and preclinical research through clinical development. The Cerdelga clinical development program is the largest ever conducted in Gaucher disease, with approximately 400 patients treated in 29 countries.
“More than twenty years ago, Genzyme introduced the world’s first treatment for Gaucher disease. We are proud to build on this legacy and continue to improve Gaucher patients’ lives through ongoing research and new therapies,” said Genzyme President and CEO, David Meeker, M.D. “The approval of Cerdelga represents our unwavering commitment to the Gaucher patient community.”
The FDA approval was based on efficacy data from two positive Phase 3 studies for Cerdelga: one in patients new to therapy (Trial 1), and the other in patients switching from approved enzyme replacement therapies (Trial 2). The filing also incorporated four years of efficacy data from the Cerdelga Phase 2 study.
In Trial 1, improvements were seen across the following endpoints after 9 months on Cerdelga: spleen size, platelet levels, hemoglobin levels, and liver volume. Patients continue to receive Cerdelga in the extension period, and the majority of patients have been on treatment for over eighteen months.
Trial 2 met the pre-specified criteria for non-inferiority to an enzyme replacement therapy (imiglucerase), which was a composite endpoint of each of the following parameters: spleen volume, hemoglobin levels, platelet counts, and liver volume. Patients continue to receive Cerdelga in the extension period, and the majority of patients have been on treatment for over two years.
The most common adverse reactions (≥10%) are fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain.
Cerdelga is a specific ceramide analogue inhibitor of glucosylceramide synthase (IC50 = 10 ng/mL) with broad tissue distribution. It reduces the production of glucosylceramide, the substance that builds up in the cells and tissues of people with Gaucher disease.