Clinical Data Demonstrates That LYSTEDA™ Significantly Reduced Menstrual Blood Loss and Limitations on Social, Leisure and Physical Activities in Women With Cyclic HMB

SAN FRANCISCO, May 19 /PRNewswire/ -- Five presentations reporting the results of two placebo-controlled clinical trials evaluating Ferring Pharmaceuticals' LYSTEDA™ (tranexamic acid) tablets, a novel, first-in-class, non-hormonal therapy indicated for treatment of women with cyclic heavy menstrual bleeding (HMB), were presented at The American College of Obstetricians and Gynecologists 58th Annual Clinical Meeting in San Francisco, May 15-19, 2010.  It is estimated that up to 22 million women suffer from HMB in the U.S.(1),(2)

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LYSTEDA oral tablets received approval on November 13, 2009 following a Priority Review by the U.S. Food and Drug Administration (FDA).  Ferring recently signed an agreement with Xanodyne Pharmaceuticals, Inc. that will expand its Women's Health product portfolio with the acquisition of the global rights to LYSTEDA, which the company will initially market in the U.S.

"We are extremely pleased by the substantial clinical evidence supporting LYSTEDA as a new therapy for heavy menstrual bleeding.  LYSTEDA was shown to significantly reduce menstrual blood loss in women suffering from cyclic heavy menstrual bleeding," said Andrea Lukes, MD, lead study investigator, Carolina Women's Research and Wellness Center.  "As a result of taking this new treatment, most women in the trial said they experienced a significant, meaningful improvement in their social, leisure and physical activities."

Edward A. Trott, MD, Vice President of Medical Affairs, Ferring Pharmaceuticals, stated, "Heavy menstrual bleeding has long been a widely overlooked and unmet medical need that many women considered normal and did not require special medication.  With the introduction of LYSTEDA, obstetricians and gynecologists can now offer appropriate patients the only non-hormonal therapy that is FDA approved for cyclic heavy menstrual bleeding and taken only during the menstrual phase."

The following results of the oral tranexamic acid formulation (LYSTEDA) were reported from two multicenter, double-blind, placebo-controlled, parallel-group trials of women aged 18-49 years with HMB, defined as mean menstrual blood loss (MBL) greater than 80 mL per cycle, averaged over two cycles and measured by a validated alkaline hematin method.

Dose-Response Analysis

To assess the efficacy and safety of two dosage regimens, 294 women with HMB were randomized (2:2:1) to receive LYSTEDA 1.95 (n=115) or 3.9 g/day (n=112) or placebo (n=67) for up to five days of menstrual bleeding.  MBL was measured during 2 baseline and 3 treatment cycles using a validated alkaline hematin method.  The tripartite primary efficacy endpoint was achieved if MBL reduction from baseline was significantly greater than placebo, met a "meaningful" reduction in MBL threshold of 36 mL/cycle as perceived by women with HMB, and exceeded 50 mL/cycle.

Only women receiving LYSTEDA 3.9 g/day achieved the tripartite endpoint.  Mean reduction of MBL for the 3.9 g/day group was -65.3 mL/cycle compared with -3.0 mL/cycle for women receiving placebo (P<.001).  Both doses of LYSTEDA were well tolerated.

A Meaningful Reduction in MBL

To quantify 'meaningful' reduction in MBL in women with HMB after treatment with LYSTEDA, data from 294 women in the study were used in a blinded receiver operating characteristic (ROC) analysis.  MBL was measured in two ways during the study.  One measured the blood loss using a validated alkaline hematin method at baseline and after treatment with LYSTEDA. The second measured a meaningful reduction in MBL using the Menorrhagia Impact Questionnaire (MIQ), a patient-reported outcome instrument previously validated in an HMB population.  Women in this study were asked if the change in their period after treatment was 'meaningful or important' after treatment compared to their prior pretreatment cycles.  After treatment with LYSTEDA, women with HMB perceived an MBL reduction of 36 mL or greater to be meaningful.  

Effect of LYSTEDA Treatment on Social, Leisure and Physical Activities

To measure changes in social, leisure and physical activities in women with HMB treated with LYSTEDA, women (n=294) were randomized (2:2:1) to receive LYSTEDA 1.95 g/day (n=115), LYSTEDA 3.9 g/day (n=112), or placebo (n=67) for up to five days per menstrual period for three cycles.  The MIQ was used to measure changes at baseline and after three treatment menstrual cycles.  The questionnaire contains questions about limitations on social, leisure, physical, or work activities during menstruation, and the woman's perception of the influence of MBL on those measures.  Women receiving either dose of LYSTEDA experienced significant improvements in all measurements from baseline compared with placebo (P <.01 for each dose).  Improvement in all measurements was greater in women receiving high-dose LYSTEDA compared with low-dose LYSTEDA.  Both dosage regimens were well tolerated.

Effects of BMI, Age and Fibroids on MBL Reduction

Additional post hoc subgroup analyses were conducted using data from a randomized study of 187 women with HMB (LYSTEDA 3.9 g/day (n=115) or placebo (n=72)).  Results showed that LYSTEDA 3.9 g/day significantly and uniformly reduced MBL, regardless of age, the presence of uterine fibroids, baseline MBL or body mass index (BMI).  

About LYSTEDA

LYSTEDA™ (tranexamic acid) tablets are indicated for the treatment of cyclic heavy menstrual bleeding.  Prior to prescribing LYSTEDA, exclude endometrial pathology that can be associated with heavy menstrual bleeding.  For more information, visit www.lysteda.com.

Important Safety Information

LYSTEDA is contraindicated in women with active thromboembolic disease or a history or intrinsic risk of thrombosis or thromboembolism, including retinal vein or artery occlusion; or known hypersensitivity to tranexamic acid.

Concomitant therapy with hormonal contraceptives may further increase the risk of blood clots, stroke, and myocardial infarction.  Women using hormonal contraception should use LYSTEDA only if there is a strong medical need and the benefit of treatment will outweigh the potential increased risk of a thrombotic event.  In case of severe allergic reaction, discontinue LYSTEDA and seek immediate medical attention.  Visual or ocular adverse effects may occur with LYSTEDA.  Immediately discontinue use if visual or ocular symptoms occur.  Concomitant use of LYSTEDA with Factor IX complex concentrates, anti-inhibitor coagulant concentrates or all-trans retinoic acids (oral tretinoin) may increase risk of thrombosis.  Cerebral edema and cerebral infarction may be caused by use of LYSTEDA in women with subarachnoid hemorrhage.

The most common adverse reactions in clinical trials (> 5%, and more frequent in LYSTEDA subjects compared to placebo subjects) were: headache, sinus and nasal symptoms, back pain, abdominal pain, musculoskeletal pain, joint pain, muscle cramps, migraine, anemia and fatigue.

About Ferring Pharmaceuticals Inc.

Ferring Pharmaceuticals Inc. is a subsidiary of Ferring Pharmaceuticals, a privately owned, international pharmaceutical company.  Ferring Pharmaceuticals offers a line of products in the U.S. market.  They include: BRAVELLE® (urofollitropin for injection, purified), MENOPUR® (menotropins for injection, USP) and REPRONEX® (menotropins for injection, USP), NOVAREL® (chorionic gonadotropin for injection, USP), ENDOMETRIN® (progesterone) Vaginal Insert, 100 mg, FIRMAGON® (degarelix for injection), PROSED® DS (methenamine, phenyl salicylate, methylene blue, benzoic acid, hyoscyamine sulfate), DESMOPRESSIN, and EUFLEXXA® (1% sodium hyaluronate).

Ferring Pharmaceuticals specializes in the research, development and commercialization of compounds in general and pediatric endocrinology, urology, orthopaedics, gastroenterology, obstetrics/gynecology, and infertility.  For more information, call 1-888-FERRING (1-888-337-7464) or visit www.FerringUSA.com.

(1)  U.S. Census Bureau, 2006-2008 American Community Survey. Available at: http://factfinder.census.gov/. Accessed April 7, 2010.

(2)  Tufts Medical Center website. Heavy Menstrual Bleeding. Available at: www.tufts-nemc.org. Accessed April 7, 2010.

Please visit www.Lysteda.com for Full Prescribing Information for LYSTEDA.

SOURCE Ferring Pharmaceuticals Inc.