Emmaus Life Sciences, a leader in the treatment of sickle cell disease, has announced that it has received the highly sought-after ‘No Action Indicated’ classification from the United States Food and Drug Administration (FDA) following a routine inspection of its Postmarketing Adverse Drug Experience (PADE) compliance program. This classification demonstrates Emmaus’ commitment to patient safety and its dedication to providing high-quality treatments.
The PADE program is designed to protect patients from poor quality, unsafe, and ineffective drugs through proactive compliance strategies and risk-based enforcement actions. One of the program’s key objectives is to ensure that accurate, reliable, and timely safety data are submitted to the FDA in accordance with postmarketing laws and regulations.
During the two and a half-day inspection, two FDA Consumer Safety Officers found no objectionable conditions or practices in the six years that Endari, Emmaus’ flagship product, has been on the market. This resulted in the ‘No Action Indicated’ (NAI) inspection classification. According to Charles Stark, Pharm.D., Chief Scientific Officer of Emmaus Life Sciences, this places Emmaus within the top 16% of all companies investigated in 2019, as per the 2019 FDA inspection results survey.
Emmaus’ mission is to provide the highest quality Endari® (L-glutamine oral powder) and ensure its safety for patients living with Sickle Cell Disease. This achievement is attributed 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, including Rafael Razon, M.D. and Rajani Singh. Emmaus also expresses gratitude to APCER Life Sciences, their long-standing Pharmacovigilance business partner, for their continued support.
Emmaus Life Sciences, Inc. is a commercial-stage biopharmaceutical company that specializes in the treatment of sickle cell disease. The company currently markets Endari®, an FDA-approved L-glutamine oral powder, which is indicated to reduce the acute complications of sickle cell disease in adults and children aged five years and older. In addition to sickle cell disease, Emmaus is engaged in the discovery and development of innovative treatments and therapies for rare and orphan diseases, as well as conditions affecting larger populations.
Sickle cell disease affects approximately 100,000 people in the United States and millions more worldwide. It is not limited to individuals of African descent, as the sickle gene is found in every ethnic group. The genetic mutation responsible for sickle cell disease causes red blood cells to distort into a sickle shape, reducing their ability to transport oxygen throughout the body. This leads to physical symptoms such as severe pain, tissue and organ damage, and early death.
Emmaus estimates that there are around 225,000 sickle cell disease patients in the Middle East North Africa region who could potentially benefit from treatment with Endari®. With their ongoing commitment to compliance and patient safety, Emmaus Life Sciences continues to make significant strides in improving the lives of individuals living with sickle cell disease.