TORONTO, Nov. 20, 2015 (GLOBE NEWSWIRE) -- Aegerion Pharmaceuticals, Inc., a biopharmaceutical company dedicated to the development and commercialization of innovative therapies for patients with debilitating rare diseases, today announced that the Régie de l'Assurance Maladie du Québec (RAMQ) has added JUXTAPID™ (lomitapide) capsules for the treatment of homozygous familial hypercholesterolemia (HoFH) to its formulary effective November 20, 2015. The listing was based on a recommendation from l'Institut National d'Excellence en Santé et en Services Sociaux (INESSS) and is subject to a Listing Agreement between the Quebec Minister of Health and Social Services and Aegerion Pharmaceuticals (Canada) Ltd. The Listing Agreement is based on Aegerion’s price for JUXTAPID in Canada, which is a fixed daily cost irrespective of the dose prescribed and strengths dispensed.
“We are pleased with decision of the RAMQ to add JUXTAPID to its formulary, furthering Aegerion’s goal of increasing access to our medicines for adult HoFH patients in need,” said Sandford D. Smith, Chief Executive Officer of Aegerion.
HoFH is a serious, rare genetic disease that impairs the function of the receptor responsible for removing LDL-cholesterol from the body, which results in extreme elevation of blood cholesterol levels. Patients with HoFH often develop premature and progressive atherosclerosis, a narrowing or blocking of the arteries.
“HoFH is a challenging disease, where patients continue to experience severe elevations in LDL-C despite the use of available therapies,” commented Dr. Jacques Genest, MD, FRCPC, Cardiologist at McGill University Health Centre, and Professor, Departments of Biochemistry, Medicine and Genetics at McGill University. "JUXTAPID has the ability to help reduce the cholesterol in adult HoFH patients and its approval on the RAMQ formulary is especially encouraging for the Quebec region, where there is a higher prevalence of HoFH based upon a founder effect.”
“FH Canada Patient Network knows first-hand the challenges of living with HoFH and applauds the Quebec Health Minister for accepting the INESSS recommendation to make this very important option available to patients with unmet needs,” added Durhane Wong-Rieger, Executive Director, FH Canada Patient Network.
The Regroupement Québécois des Maladies Orphelines (RQMO) has been representing patients with rare diseases in Quebec for more than 5 years. “It is comforting to see the Quebec government completing the full circle from assisting in bench side discovery to bedside availability of medicines. Having a medication like JUXTAPID funded for adult patients with HoFH breaks down the final barrier, and allows appropriate patients access to this innovative medicine,” said Gail Ouellette, geneticist, President and Executive Director of the RQMO.
About Aegerion Pharmaceuticals
Aegerion Pharmaceuticals is a biopharmaceutical company dedicated to the development and commercialization of innovative therapies for patients with debilitating rare diseases. Our first approved product, JUXTAPID, is an oral once-daily capsule that offers a treatment option to patients with homozygous familial hypercholesterolemia (HoFH) – a severe lipid disorder. For more information about the company, please visit www.aegerion.ca.
Important Safety Information
- Five (17%) of the 29 patients with HoFH that participated in the clinical trial discontinued treatment due to an adverse reaction.
- The most common adverse reactions were gastrointestinal, reported by 27 (93%) of 29 patients.
- Adverse reactions reported by ≥8 (28%) patients in the HoFH clinical trial included diarrhea, nausea, vomiting, dyspepsia, and abdominal pain.
- Gastrointestinal reactions contributed to the reasons for early discontinuation from the trial for 4 (14%) patients.
- To reduce the risk of gastrointestinal adverse events, patients should adhere to a low-fat diet supplying <20% of energy from fat and the dosage of Juxtapid should be increased gradually.
Transaminase Elevations
JUXTAPID™ can cause elevations in alanine aminotransferase (ALT) and aspartate aminotransferase (AST). During the HoFH clinical trial:
- 10 (34%) of 29 patients had at least one elevation in ALT and/or AST ≥3x ULN.
- No clinically meaningful elevations in total bilirubin or alkaline phosphatase were observed.
- Dose adjustments were required for patients with elevated levels of transaminases per study protocol. Transaminases typically fell within one to four weeks of reducing the dose or withholding JUXTAPID™.
Hepatic transaminases, i.e., serum ALT, AST, should be measured before initiation of treatment with JUXTAPID™, and prior to each dose escalation. After the patient has been stabilized on an individualized dose, transaminases should be measured periodically, i.e., monthly during the first year of treatment and every three months after the first year.
Please consult the complete Product Monograph at http://www.aegerion.ca/Product_monograph_lomitapide.htm for contraindications, warnings, precautions, adverse reactions, interactions, dosing, and conditions of clinical use.
The product monograph is also available by calling us at 1-855-303-2347.