Emmaus complying with FDA postmarketing safety regulations
No issues found in 6 years of marketing Endari for SCD in US, per company
Emmaus Life Sciences, which markets Endari (L-glutamine) for sickle cell disease (SCD), has announced that it is complying with postmarketing safety regulations of the U.S. Food and Drug Administration (FDA).
The announcement followed a 2.5-day routine inspection of Emmaus under the FDA’s Postmarketing Adverse Drug Experience (PADE) compliance program. According to the company, no issues were detected by the regulatory agency in the six years that Endari has been marketed in the country.
“This exemplifies Emmaus’ mission to provide the highest quality Endari … and monitor its safety for our patients living with sickle cell disease,” Charles Stark, Emmaus’ chief scientific officer, said in a company press release.
“These outcomes were possible due to the consistent and dedicated compliance to FDA regulations around surveillance, receipt, evaluation, and timely reporting of safety data by the safety and regulatory colleagues at Emmaus,” Stark added.
The PADE program is intended to ensure that accurate and reliable safety data on therapies are submitted in a timely fashion to the FDA as per postmarketing laws and regulations. It also aims to protect patients from poor quality, unsafe, and ineffective medications, through surveillance actions.
Emmaus receives ‘No Action Indicated’ classification post-inspection
Pharmaceutical companies are selected for inspection based on risk criteria, which include company information and developed products. Inspection history, taking into account companies that have not been inspected before or ones that had issues in previous inspections, also is considered for site selection.
In the case of Emmaus, the inspection by two FDA consumer safety officers found no objectionable conditions or practices, and thereby the company received a “No Action Indicated” inspection classification.
“Based on the 2019 FDA inspection results survey, this places Emmaus within the top 16% of all companies investigated in 2019,” Stark said.
The company is the developer of Endari, an oral therapy approved in the U.S. since 2017 for adults and children, 5 and older, with SCD. The treatment works to reduce acute disease complications, including pain crises, in patients.
SCD is caused by mutations in the HBB gene that lead to the production of a faulty version of hemoglobin (hemoglobin S), the protein responsible for transporting oxygen in red blood cells.
Hemoglobin S tends to clump inside red blood cells, causing them to acquire a sickle-like shape and become more rigid, and more likely to stick together. That can block blood flow and lead to a shortage of oxygen in several tissues, causing acutely painful episodes known as vaso-occlusive crises.
Moreover, sickled red blood cells also tend to die prematurely. This is mainly due to their higher susceptibility to oxidative stress, a type of cellular damage caused by an excess of damaging molecules called free radicals that overcome the cells’ antioxidant defenses.
Endari was designed to boost the production of antioxidants to help reduce oxidative stress in red blood cells. This helps red blood cells regain flexibility and improve their ability to travel through blood vessels and carry oxygen to tissues.
In clinical trials, the therapy was found to be safe and well tolerated, and to effectively reduce pain crises and other SCD-related complications, as well as hospital admissions of SCD patients.
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