| || Press Release|
Source: Sanofi (EURONEXT: SAN) (NYSE: SNY)
CHMP recommends approval of Cablivi(TM) (caplacizumab)
First therapeutic to receive positive CHMP opinion for treatment of a rare blood disorder called acquired thrombotic thrombocytopenic purpura
Paris, France - June 29, 2018 -The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended approval of Cablivi (caplacizumab) in Europe for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood-clotting disorder. Cablivi was developed by Ablynx, a Sanofi Company.
The European Commission will review the CHMP recommendation and a final decision on the Marketing Authorisation Application for Cablivi in the European Union is expected in the coming months.
Directed against von Willebrand Factor (vWF), Cablivi is Ablynx's first Nanobody®-based medicine to receive a positive CHMP opinion and, if approved, will be the first therapeutic specifically indicated for the treatment of aTTP.
aTTP is a life-threatening, autoimmune-based blood clotting disorder characterized by extensive clot formation in small blood vessels throughout the body, leading to thrombocytopenia, ischemia and widespread organ damage especially in the brain and heart. There are currently no products authorised for the treatment of aTTP and despite treatment with plasma exchange (PEX) and immunosuppression, patients remain at risk for thrombotic complications, recurrences and death. The potential of Cablivi to address this unmet need has been demonstrated in 220 patients with aTTP, who participated in the phase II TITAN and phase III HERCULES studies. Data from the TITAN study were published in the New England Journal of Medicine in February 2016 and the positive HERCULES results were presented at the 59th Annual Meeting of the American Society of Hematology (ASH) in December 2017.
If approved, Cablivi will be made available to patients through Sanofi Genzyme, Sanofi's specialty care business, and will be part of the unit's new rare blood disorders franchise that will launch in 2019 and which will also include Bioverativ's treatments for Hemophilia A and B.
About Cablivi (caplacizumab):
Caplacizumab is a bivalent anti-vWF Nanobody that received Orphan Drug Designation in Europe and the United States in 2009. Caplacizumab blocks the interaction of ultra-large vWF multimers with platelets and, therefore, has an immediate effect on platelet adhesion and the ensuing formation and accumulation of the micro-clots that cause the severe thrombocytopenia, tissue ischemia and organ dysfunction in aTTP. This immediate effect of caplacizumab protects the patient from the manifestations of the disease while the underlying disease process resolves.
The efficacy and safety of caplacizumab in addition to daily PEX and immunosuppression were demonstrated in the phase II TITAN and Phase III HERCULES studies. In the HERCULES study, treatment with caplacizumab in addition to standard-of-care resulted in a significantly shorter time to platelet count response (p<0.01), a significant reduction in aTTP-related death, recurrence of aTTP, or at least one major thromboembolic event during study drug treatment (p<0.0001), and a significantly lower number of aTTP recurrences in the overall study period (p<0.001). Importantly, treatment with caplacizumab resulted in a clinically meaningful reduction in the use of PEX and length of stay in the intensive care unit (ICU) and the hospital, compared to the placebo group. In addition, caplacizumab prevents refractory disease and has a positive impact on the normalisation of organ damage markers (lactate dehydrogenase, cardiac troponin I and serum creatinine). Caplacizumab has a favourable safety profile, consistent with its mechanism of action. No deaths were reported during study drug treatment in the caplacizumab group in the TITAN and HERCULES studies, while for the placebo group, two deaths were reported in the TITAN study and three deaths in the HERCULES study.
A three-year follow-up study (NCT02878603) of patients who have completed the HERCULES study is in progress and will further evaluate the long-term safety and efficacy of caplacizumab and repeated use of caplacizumab, as well as characterising the long-term impact of aTTP.
In Europe, a MAA for caplacizumab in aTTP was submitted to the EMA in February 2017 based on the Phase II TITAN study results. The phase III HERCULES study results became available in the course of this procedure and were subsequently added to the Application.
In the United States, Fast Track designation was received from the Food and Drug Administration (FDA) for caplacizumab for the treatment of aTTP in July 2017 and filing of a Biologics License Application (BLA) is planned in 2018.
aTTP is a rare, acute, life-threatening, autoimmune blood clotting disorder. It is caused by impaired activity of the ADAMTS13 enzyme, leaving ULvWF molecules uncleaved (vWF is an important protein involved in the blood clotting process). These ULvWF molecules spontaneously bind to blood platelets, resulting in severe thrombocytopenia (very low platelet count) and clot formation in small blood vessels throughout the body, leading to ischemia and widespread organ damage.
Despite the current standard-of-care treatment consisting of PEX and immunosuppression, episodes of aTTP are still associated with a mortality rate of up to 20%, with most deaths occurring within 30 days of diagnosis. Furthermore, patients are at risk of acute thromboembolic complications (e.g. stroke, myocardial infarction) and of recurrence of disease. Some patients have refractory disease1, which is associated with a poor prognosis for survival of an acute episode of aTTP. Long term, patients are at increased risk for hypertension, major depression, and premature death.
Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.
With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.
Sanofi, Empowering Life
Media Relations Contact
Tel.: +33 (0)1 53 77 46 46
Investor Relations Contact
Tel.: +33 (0)1 53 77 45 45
| Sanofi Forward-Looking Statements|
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates regarding the clinical development of and potential marketing approvals for the product. Forward-looking statements are generally identified by the words "expects", "anticipates", "believes", "intends", "estimates", "plans", "would be" and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development of the product, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve the product or biological application that may be filed for the product as well as their decisions regarding labeling and other matters that could affect the availability or commercial potential of the product, the absence of guarantee that the product if approved will be commercially successful, risks associated with intellectual property, future litigation, the future approval and commercial success of therapeutic alternatives, and volatile economic conditions, as well as those risks discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under "Risk Factors" and "Cautionary Statement Regarding Forward-Looking Statements" in Sanofi's annual report on Form 20-F for the year ended December 31, 2017. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.
© 2018 GlobeNewswire, Inc. All Rights Reserved.