The European Medicines Agency (EMA) has begun a reevaluation of the risk/benefit ratio for ESMYA (Ulipristal acetate), a drug for treatment of uterine fibroids in women of childbearing age. This decision follows five cases of severe hepatic insufficiency in Europe.
According to new recommendations from EMA and the french ANSM (French acronym for “National Agency for the Safety of Medicines and Health Products”) to healthcare professionals, treatment with ESMYA should no longer be initiated in new patients before any published evaluation results are conclusive. Women on treatment are required to be monitored very closely and with the utmost vigilance. The findings of this assessment are expected to be available by the second quarter of 2018.
Since the drug was marketed in 2012, there have been some 50 cases of liver disease reported in patients on ESMYA therapy in Europe. Of these cases, five were critical and included the death of one patient in Germany in October 2017. The remaining four patients had to undergo liver transplantation.
The drug ESMYA (ulipristal acetate), produced by the Hungarian laboratory Gedeon Richter, entered the pharmaceutical market in 2012 and is indicated for the preoperative treatment of uterine fibroids symptoms in adult women of childbearing age that range from moderate to severe. The duration of a treatment cycle is recommended to remain to not exceed 3 months.
Uterine fibroids are benign uterus tumors whose prevalence increases sharply from the age of 30 years. They represent the first cause of hysterectomy in women in some European countries. The symptomatic expression of the disease varies from one patient to another. For some women, the condition remains asymptomatic while for others, a uterine fibroma can cause troublesome symptoms such as metrorrhagia, anemia, and pelvic pain. ESMYA has shown great effectiveness in treating these signs and since its launch, it has been prescribed to 700,000 women worldwide.
As of late February 2019, there is a high degree of uncertainty about this case. According to the EMA, clinical drug trials that were performed on 7000 women did not show hepatic toxicity.
ESMYA is intended for the symptomatic treatment of uterine fibroids and therefore does not fall under the category of curative drugs. For this reason, transitional measures have been taken as a precautionary principle as new tests are awaited. According to these guidelines, health professionals should no longer undertake further treatment with this drug in new patients. For women who are already on treatment, close monitoring should be ensured by performing a monthly liver test. A transaminase level 2 times above normal values should require immediate discontinuation of the drug. In the same way, the appearance of symptoms signifying possible liver injury (nausea, anorexia, asthenia, pain in the right hypochondrium) requires stopping treatment and prescribing a liver test.
The decision to resume a treatment or not will be made based on the results of this assessment. In addition, an information sheet must be given to patients undergoing treatment to make them aware of alert symptoms that may hint at any possible liver damage.