CAMBRIDGE, Mass. and BASEL, Switzerland, June 27, 2022 (GLOBE NEWSWIRE) -- Enzyvant, a commercial-stage biotechnology company with a focus on regenerative medicines for rare diseases, announced today it is among the National Organization for Rare Disorders (NORD) 2022 Industry Innovation Awards recipients for the development and U.S. Food and Drug Administration (FDA) approval of RETHYMIC® (allogeneic processed thymus tissue-agdc), a one-time regenerative tissue-based therapy for pediatric congenital athymia. Enzyvant received the award during NORD’s 2022 Rare Impact Award ceremony that took place during its Living Rare, Living Stronger Patient and Family Forum on June 26, 2022, in Cleveland, Ohio.
RETHYMIC is the first and only FDA-approved treatment indicated for immune reconstitution in pediatric patients with congenital athymia. Unlike a transplant, RETHYMIC is developed through a 12- to 21-day process in which donor thymus tissue is engineered for implantation via a single surgical procedure. Enzyvant developed this complex manufacturing process to make this treatment. During an in-patient procedure, RETHYMIC is surgically implanted in the thigh muscle of a child with congenital athymia.
“Enzyvant is honored to be a 2022 NORD Industry Innovation Award recipient, which emphasizes the importance of the Company’s work to pioneer the development of RETHYMIC. This achievement would not have been possible without our dedicated team and the supportive community of patients, caregivers, clinicians, and researchers,” said Enzyvant CEO William Symonds. “We would like to thank NORD for this honor as we build on our efforts to ensure all patients who may benefit from RETHYMIC have access to this important treatment option.”
Congenital athymia is an ultra-rare primary immunodeficiency that occurs when a child is born without a thymus, a critical component of the immune system, affecting about 17-24 infants per year in the U.S. Without treatment, these infants are extremely vulnerable to life-threatening infections and immune dysregulation – often forcing families into strict isolation to protect their child. Despite these measures, with only supportive care children with congenital athymia won’t survive beyond 2 to 3 years of age.
The NORD Rare Impact Awards celebrate the individuals, groups, and companies making extraordinary contributions to the lives of rare disease patients and caregivers. NORD is the leading independent advocacy organization committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.
About Enzyvant
Enzyvant is a commercial-stage biotechnology company with a focus on regenerative medicines for rare diseases. The company’s first commercial product is U.S. Food and Drug Administration (FDA)-approved RETHYMIC (allogeneic processed thymus tissue-agdc), a tissue-based regenerative therapy for an ultra-rare and life-threatening pediatric immunodeficiency. Enzyvant has distinctive capabilities in expedited development of regenerative therapies for rare diseases. The company has obtained and leveraged multiple regulatory designations including Regenerative Medicine Advanced Therapy, Breakthrough, Fast Track, Rare Pediatric Disease, Orphan Drug, and Advanced Therapies Medicinal Product. Enzyvant is wholly owned by Sumitovant Biopharma Ltd. (wholly owned by Sumitomo Pharma). For more information about Enzyvant, visit Enzyvant.com.
About Sumitovant Biopharma Ltd.
Sumitovant is a technology-driven biopharmaceutical company accelerating development of new potential therapies for patients with high unmet medical need. Through our company portfolio and use of embedded computational technology platforms to generate business and scientific insights, Sumitovant has supported development of FDA-approved products and advanced a promising pipeline of early-through late-stage investigational assets for other serious conditions. Sumitovant’s subsidiary portfolio includes wholly-owned Enzyvant, Urovant, Spirovant and Altavant, and majority-owned Myovant (NYSE: MYOV). Sumitomo Pharma is Sumitovant’s parent company. For more information, please visit sumitovant.com
About RETHYMIC®
RETHYMIC® (allogeneic processed thymus tissue-agdc) is a novel one-time tissue-based regenerative therapy used for immune reconstitution in pediatric patients with congenital athymia. RETHYMIC is engineered human thymus tissue designed to regenerate the thymic function children with congenital athymia are missing and does not require donor-recipient matching. RETHYMIC has been studied across 10 clinical trials for more than 25 years and was granted multiple U.S. Food and Drug Administration (FDA) designations including Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Rare Pediatric Disease, and Orphan Drug. It also has been granted the Orphan Drug designation and the Advanced Therapy Medicinal Product designation by the European Medicines Agency. RETHYMIC is the first and only treatment approved by the FDA for immune reconstitution in pediatric patients with congenital athymia.
Please see Full Prescribing Information
Indication and Important Safety Information
Indication
RETHYMIC® (allogeneic processed thymus tissue–agdc) is indicated for immune reconstitution in pediatric patients with congenital athymia.
Limitations of Use:
RETHYMIC is not indicated for the treatment of patients with severe combined immunodeficiency (SCID).
Important Safety Information
Immune reconstitution sufficient to protect from infection is unlikely to develop prior to 6-12 months after treatment with RETHYMIC. Given the immunocompromised condition of athymic patients, follow infection control measures until the development of thymic function is established as measured through flow cytometry. Monitor patients closely for signs of infection including fever. If a fever develops, assess the patient by blood and other cultures and treat with antimicrobials as clinically indicated. Patients should be maintained on immunoglobulin replacement therapy until specified criteria are met, and two months after stopping, IgG trough level should be checked. Prior to and after treatment with RETHYMIC, patients should be maintained on Pneumocystis jiroveci pneumonia prophylaxis until specified criteria are met.
RETHYMIC may cause or exacerbate pre-existing graft versus host disease (GVHD). Monitor and treat patients at risk for the development of GVHD. Risk factors for GVHD include atypical complete DiGeorge anomaly phenotype, prior hematopoietic cell transplantation (HCT) and maternal engraftment. GVHD may manifest as fever, rash, lymphadenopathy, elevated bilirubin and liver enzymes, enteritis, and/or diarrhea.
Autoimmune-related adverse events occurred in patients treated with RETHYMIC. These events included: thrombocytopenia, neutropenia, proteinuria, hemolytic anemia, alopecia, hypothyroidism, autoimmune hepatitis, autoimmune arthritis, transverse myelitis, albinism, hyperthyroidism, and ovarian failure. Monitor for the development of autoimmune disorders, including complete blood counts with differential, liver enzymes, serum creatinine, urinalysis, and thyroid function.
Pre-existing renal impairment is a risk factor for death.
In the clinical studies of RETHYMIC, 3 out of 4 patients with pre-existing cytomegalovirus infection died. The benefits/risks of treatment should be considered prior to treating patients with pre-existing CMV infection.
Because of the underlying immune deficiency, patients who receive RETHYMIC may be at risk of developing post-treatment lymphoproliferative disorder. Patients should be monitored for the development of lymphoproliferative disorder.
Transmission of infectious disease may occur because RETHYMIC is derived from human tissue and because product manufacturing includes porcine- and bovine-derived reagents.
Immunizations should not be administered in patients who have received RETHYMIC until immune-function criteria have been met.
All patients should be screened for anti-HLA antibodies prior to receiving RETHYMIC. Patients testing positive for anti-HLA antibodies should receive RETHYMIC from a donor who does not express those HLA alleles. HLA matching is required in patients who have received a prior HCT or a solid organ transplant. Patients who have received a prior HCT are at increased risk of developing GVHD after RETHYMIC if the HCT donor did not fully match the recipient.
Of the 105 patients in clinical studies, 29 patients died, including 23 deaths in the first year (< 365 days) after implantation.
The most common (>10%) adverse events related to RETHYMIC included: hypertension, cytokine release syndrome, rash, hypomagnesemia, renal impairment/failure, thrombocytopenia, and graft versus host disease.
To report suspected adverse reactions, please contact the FDA at 1-800-FDA-1088 or www.fda.gov/safety/medwatch
Media Contacts:
Enzyvant
Eliza Schleifstein
6 Degrees
(917) 763-8106
Eliza@schleifsteinpr.com
Sumitovant
Maya Frutiger
Head of Corporate Communications
media@sumitovant.com
