Emmaus Life Sciences (OTCMKTS:EMMA) is a development‐stage biopharmaceutical company primarily focused on advancing novel therapies for individuals living with rare and genetic diseases. Headquartered in Torrance, California, Emmaus Life Sciences has built its portfolio around the commercial launch of Endari® (L‐glutamine oral powder), which received U.S. Food and Drug Administration approval in 2017 for the reduction of acute complications of sickle cell disease in adult and pediatric patients. In addition to Endari, the company maintains a pipeline of investigational candidates aimed at addressing high‐unmet medical needs in areas such as cystic fibrosis and nephropathic cystinosis.
Endari represents Emmaus’s first successful entry into the U.S. market, where it utilizes a direct‐to‐patient specialty distribution model and collaborates with key healthcare providers and patient advocacy groups. Building on this foundation, Emmaus holds exclusive worldwide development and commercialization rights for ELX‐02, a novel aminoglycoside analog licensed from Eloxx Pharmaceuticals. ELX‐02 is designed to promote “readthrough” of certain genetic mutations and is in clinical development for rare diseases caused by nonsense mutations, with initial programs focused on cystic fibrosis and nephropathic cystinosis.
Since its inception in the late 1990s, Emmaus Life Sciences has evolved from a small private venture into a publicly traded entity, expanding its geographic reach beyond North America into Europe and other selected markets through strategic licensing and partnership agreements. The company’s supply chain is supported by contract manufacturing organizations in the United States and abroad, ensuring continuity of product availability and compliance with applicable regulatory standards. Emmaus also actively engages in global patient registries and collaborates with academic institutions to advance its understanding of targeted genetic disorders.
The leadership team at Emmaus Life Sciences brings together seasoned professionals from the pharmaceutical and rare disease communities, with expertise spanning clinical development, regulatory affairs, commercial strategy and patient advocacy. Guided by a board of directors experienced in orphan drug development, the management group is committed to executing on a dual strategy of maximizing Endari’s market opportunity while progressing ELX‐02 and other pipeline assets through clinical milestones. Through this approach, Emmaus Life Sciences aims to deliver transformative therapies and improve quality of life for patients affected by life‐threatening genetic conditions.
