BRIDGEWATER, N.J., Jan. 30, 2019 /PRNewswire/ -- Continued research and development is critical to advancing care for people with rare diseases who have a significant need for earlier diagnosis, better disease management opportunities, and new treatment options. Sanofi Genzyme, the specialty care global business unit of Sanofi, has been a pioneer in rare diseases with a focus on lysosomal diseases, a group of extremely rare genetic diseases, for more than three decades.
New research on the company's approved treatments for a number of lysosomal diseases, along with ongoing clinical studies of treatments in development for a broader range of rare diseases, will be presented at an upcoming key scientific meeting, the 15th Annual WORLDSymposium™ 2019, being held February 4 – 8, 2019, in Orlando, FL.
"Expanding the depth and breadth of knowledge related to rare genetic diseases, particularly lysosomal diseases, are focus areas for Sanofi," said Sébastien Martel, Global Head of Rare Diseases, Sanofi Genzyme. "We are committed to bringing new therapeutic options to patients living with rare genetic diseases and look forward to sharing our progress at WORLDSymposium this year."
"We made significant progress this year advancing innovative therapies in clinical development for several rare diseases," said Gianluca Pirozzi, Head of Development for Rare Disease and Translational Gene Therapy, Sanofi. "We are on track with the enrollment of our clinical studies and we are excited to share our results."
Olipudase Alfa Development Program
Data presented at WORLDSymposium include the latest research regarding olipudase alfa, an investigational enzyme replacement therapy being studied for patients with non-neurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B and A/B. Both the Phase 2/3 clinical trial in adults (ASCEND) and the Phase 1/2 clinical trial in children (ASCEND-Peds) have successfully completed enrollment for the targeted number of patients for each of these trials.
Olipudase Alfa Poster at WORLDSymposium:
Venglustat Development Program
Venglustat is an investigational oral therapy in development for multiple rare disorders associated with mutations in the glycosphingolipid metabolic pathway including Gaucher disease type 3, GBA-related Parkinson's disease, Fabry disease and autosomal dominant polycystic kidney disease (ADPKD). Preliminary data from the Phase 2 clinical trial in Gaucher disease type 3 (LEAP) will be presented at WORLDSymposium along with data from the Phase 2 clinical trial in Parkinson's disease patients with a GBA mutation (MOVES-PD):
Avalglucosidase Alfa Development Program
Avalglucosidase alfa is an investigational enzyme replacement therapy that has been designed for selective receptor targeting and increased enzyme uptake in muscles, with the aim of enhancing glycogen clearance in patients with both late-onset Pompe disease and infantile-onset Pompe disease. Data from both the late-onset and infantile-onset studies will be shared at WORLDSymposium:
Olipudase alfa, venglustat and avalglucosidase alfa are investigational agents and have not been approved by the US Food and Drug Administration (FDA) or any other regulatory agency worldwide for the uses under investigation.
Sanofi Genzyme Rare Disease Registries
A disease registry is an observational database where clinical information is collected per routine clinical practice and voluntarily reported on patients with a specific condition. This information can be retrospectively analyzed and the results can help healthcare professionals better understand the disease and its management. The Sanofi Genzyme Rare Disease Registries program started in 1991 for patients with Gaucher disease and since that time additional registries have been added for Fabry, MPS I, and Pompe diseases. Multiple analyses of registry data will be presented at WORLDSymposium:
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CERDELGA® (eliglustat)
Indication
CERDELGA is a prescription medicine used for the long-term treatment of Gaucher disease type 1 (GD1) in adults who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test. Your doctor will perform a test to make sure that CERDELGA is right for you.
Limitations of Use:
Important Safety Information
Certain patients should not use CERDELGA based on their CYP2D6 metabolizer status due to an increased risk of side effects, including heart problems. Do not use CERDELGA if you are:
Your doctor will perform a test to help determine if CERDELGA is right for you.
CERDELGA can affect the way other medicines work and other medicines can affect how CERDELGA works. Using CERDELGA with other medicines or herbal supplements may cause an increased risk of side effects, including changes in electrical activity of your heart (ECG changes) and irregular heart beat (arrhythmias). Especially tell your doctor if you take St. John's Wort, or medicines for fungal infections, tuberculosis, seizures, heart conditions, high blood pressure, or depression or other mental health problems. Your doctor may need to prescribe a different medicine, change your dose of other medicines, or change your dose of CERDELGA. Tell your doctor about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements before you start taking them.
Before taking CERDELGA, tell your doctor about all of your medical conditions, including heart problems (including a condition called long QT syndrome), a history of heart attack, kidney or liver problems. If you are pregnant or plan to become pregnant or breastfeed, talk to your doctor. It is not known if CERDELGA will harm your unborn baby. Talk to your doctor if you are breastfeeding or planning to breastfeed. It is not known if CERDELGA passes into your breast milk. You and your doctor will decide if you should take CERDELGA or breastfeed. You should not do both.
CERDELGA, used with certain other medicines, may cause changes in the electrical activity of your heart (ECG changes) and irregular heart beat (arrhythmias). Tell your doctor if you have new symptoms such as palpitations, fainting, or dizziness.
The most common side effects (≥10%) of CERDELGA include: tiredness, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain. Call your doctor for medical advice about adverse effects.
Tell your doctor if you have any side effect that bothers you or that does not go away. These are not all the possible side effects of CERDELGA. Call your doctor for medical advice about side effects.
It is not known if CERDELGA is safe and effective in children.
Please see the full Prescribing Information, including the Patient Medication Guide, for CERDELGA.
Cerezyme® (imiglucerase for injection)
Indication & Usage
Cerezyme® (imiglucerase for injection) is indicated for long-term enzyme replacement therapy for pediatric and adult patients with a confirmed diagnosis of Type 1 Gaucher disease that results in one or more of the following conditions:
Important Safety Information
Approximately 15% of patients have developed immune responses (antibodies) to Cerezyme during the first year of therapy. These patients have a higher risk of an allergic reaction (hypersensitivity). Your doctor may periodically test for the presence of antibodies. Serious allergic reactions (anaphylaxis) have been reported in less than 1% of patients. Symptoms suggestive of allergic reaction happened in approximately 7% of patients, and include itching, flushing, hives, swelling, chest discomfort, shortness of breath, coughing, cyanosis (a bluish discoloration of the skin due to diminished oxygen), and low blood pressure. If you have had an allergic reaction to Cerezyme, you and your doctor should use caution if you continue to receive treatment with Cerezyme.
High blood pressure in the arteries of the lungs (pulmonary hypertension) and pneumonia have been observed in less than 1% of patients during treatment with Cerezyme. These are also known complications of Gaucher disease regardless of treatment. If you experience symptoms such as shortness of breath or chest pain, with or without fever, contact your doctor.
Approximately 14% of patients have experienced side effects related to treatment with Cerezyme. Some of these reactions occur at the site of injection such as discomfort, itching, burning, swelling or uninfected abscess. Other side effects, each of which was reported by less than 2% of patients, include nausea, abdominal pain, vomiting, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and rapid heart rate. Temporary swelling in the legs has also been observed with drugs like Cerezyme.
Please see Full Prescribing Information (PDF).
Fabrazyme® (agalsidase beta)
Indication and Usage
Fabrazyme® (agalsidase beta) is used to treat patients with Fabry disease. Fabrazyme lowers the amount of a substance called globotriaosylceramide (GL-3), which builds up in cells lining the blood vessels of the kidney and certain other cells.
The lowering of GL-3 suggests that Fabrazyme may improve how Fabry disease affects your body; however a relationship of lower GL-3 to specific signs and symptoms of Fabry disease has not been proven.
Important Safety Information
Fabrazyme can cause serious side effects, including:
Severe Allergic Reactions (anaphylaxis): Life-threatening severe allergic (anaphylactic) reactions have been seen in patients during Fabrazyme infusions. Approximately 1% of patients who have received Fabrazyme either during a clinical study or after Fabrazyme was approved have experienced anaphylactic or severe allergic reactions during their infusion.
Infusion-Associated Reactions: In clinical studies with Fabrazyme, 59% of patients experienced infusion-associated reactions during Fabrazyme administration, some of which were severe.
Pre-existing Heart Problems: People with advanced Fabry disease may have heart problems, which may put them at a higher risk for severe complications from infusion-associated reactions. These patients should be watched closely during their infusion if the decision is made to give them Fabrazyme.
Immune Response and Continued Treatment After Allergic Reaction: In the clinical studies, a few patients developed IgE antibodies or a reaction to an allergy skin test specific to Fabrazyme. IgE antibodies are usually produced by the body's immune system during an allergic reaction. Your doctor should consider testing for IgE antibodies if you experience suspected allergic reactions. Providing Fabrazyme to patients who have experienced severe or serious allergic reactions to Fabrazyme should only be done after carefully considering the risks and benefits of continuing the treatment, and only under the direct supervision of a qualified healthcare professional and with appropriate medical support readily available.
Common and Other Possible Side Effects:
The safety and effectiveness of Fabrazyme in patients younger than 8 years of age have not been studied.
Please see full prescribing information for Fabrazyme.
SOURCE Sanofi
