On October 8, 2021, the FDA approved Rethymic, marking a life-changing day of hope for children with congenital athymia and their families. Rethymic is not only the first, it is also the only treatment option that rebuilds the immune system for pediatric congenital athymia approved by the FDA. The scientists at Enzyvant have been researching this treatment option for 25 years. Their dedication was inspired by the world of possibilities in store for children with this rare disease, if only they could live past their 3rd birthday.
Pediatric congenital athymia is a rare disease found in 17-24 live births in the United States every year. Children with this disease are born severely immunodeficient because they lack a thymus. The thymus is where thymus cells or T cells are chosen to combat infections or dismantled if they pose a threat to the body. Patients with congenital athymia also have dysregulated immunity and are extremely susceptible to infections. Without the ability to properly attack these infections, children with this rare disease lose their lives by the age of 3 after receiving only supportive care. That is until now.
Rethymic is a regenerative therapy used in children with congenital athymia to rebuild their immunity. Rethymic bypasses the need for a donor because it is engineered thymus tissue. The 25 long years of research have paid off with this breakthrough treatment option called Rethymic. As Rachelle Jacques, CEO of Enzyvant explains, “For too long, families have faced a reality that the brutal journey for pediatric congenital athymia patients receiving supportive care only would end tragically. The FDA approval of Rethymic will help patients access this desperately needed therapy beyond clinical study. We are deeply grateful to the 105 patients who participated in clinical trials, their families, and all of the stakeholders who contributed to this pioneering regenerative medicine research program.”
Jacques has been a pioneer for leading the development and delivery of novel treatments for patients with rare diseases for years now. She has served in many leadership capacities in the U.S. and abroad from finance to manufacturing. As the Global Complement Franchise Head of Alexion she developed and executed the strategies for global franchises for several therapeutic areas of interest including hematology and nephrology. While at Alexion, she also served as the Senior Vice President. At the U.S. Hematology Marketing at Shire, she served as the Vice President. In 2016, when they acquired Baxalta, she was the Vice President of Business Operations. Presently, not only does she serve as the CEO of Enzyvant, she is also a member of the Board of Corbus Pharmaceuticals and uniQure. The Alliance of for Regenerative Medicine (ARM) Tissue Engineering & Biomaterials Committee are honored to have her as their co-chair and one of the founders of their Action for Equality Task Force.With this award winning FDA approval, there is more in store for Enzyvant and Jacques, who has spent many years committed to arming rare disease patients with a fighting chance at a future of possibilities