Enzyvant today announced the U.S. Food and Drug Administration
(FDA) approval of RETHYMIC® (allogeneic processed thymus
tissue-agdc), a one-time regenerative tissue-based therapy for
immune reconstitution in pediatric patients with congenital
athymia.
“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,” said Rachelle Jacques, CEO of Enzyvant. “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.”
Pediatric congenital athymia is ultra-rare with an estimated
incidence of about 17 to 24 live births each year in the United
States. Children who have this condition are born without a thymus
and therefore have profound immunodeficiency, life-threatening
immune dysregulation, and high susceptibility to potentially fatal
infections. With only supportive care, children with congenital
athymia typically die by age two or three.
“This therapy is the result of more than 25 years of research
aimed at increasing survival for patients who previously had very
little hope,” said Louise Markert, M.D, Ph.D., principal
investigator for RETHYMIC clinical trials and Professor of
Pediatrics and Immunology at the Duke University School of
Medicine. “Our research program was inspired each and every day by
the possibilities that exist for children who have congenital
athymia with an FDA-approved treatment for this devastating
condition.”
With this FDA approval, Enzyvant has obtained a Priority Review
Voucher (PRV) under the Rare Pediatric Disease Program.
RETHYMIC Clinical Trial DataTen prospective
single-arm, open-label studies with patient enrollment from 1993 to
2020 form the basis of the RETHYMIC data set. One hundred and five
patients were surgically implanted with RETHYMIC under one of 10
Institutional Review Board (IRB)-approved protocols. Ninety-five
patients were included in the Efficacy Analysis Set (EAS) and 105
patients were included in the Safety Analysis Set.
Survival rates were analyzed with the longest follow-up period
of 25.5 years. In the EAS, Kaplan-Meier estimated survival rates
(95% CI) were 77% (0.670–0.841) at one year and 76%
(0.658–0.832) at two years. For patients who were alive at one year
post implantation, the Kaplan-Meier estimated long-term survival
rate was 94% at a median follow-up time of 10.7 years. For the
patients in the clinical trials, naïve T-cell levels were measured
using flow cytometry at six, 12, and 24 months after implantation
with RETHYMIC. Patients in the clinical trials started out with
very few naïve T cells but naïve CD4+ and CD8+ T cells began to
reconstitute over the first year, with a durable increase through
year two. Reductions in the number of infections over time during
the first two years after treatment were statistically significant
(p<0.001).
The most common adverse reactions (incidence in at least 10% of
patients) reported following administration of RETHYMIC were
hypertension (high blood pressure), cytokine release syndrome,
rash, hypomagnesemia (low magnesium), renal impairment / failure
(decrease of kidney function), thrombocytopenia (low platelets),
and graft versus host disease. Of the 105 patients in the clinical
trials, 29 patients died after receiving RETHYMIC, including 23
deaths in the first year (< 365 days) after
implantation. Causes of death in the first year included 13
deaths due to infection or complications due to infection, five
deaths due to respiratory failure/hypoxia, three deaths due to
hemorrhage-related events, and two deaths due to cardiorespiratory
arrest. Of the six patients who died more than one year
post-implantation, the deaths were considered unrelated to study
treatment: two died due to respiratory failure and one died
due to each of the following: cardiopulmonary arrest, intracranial
hemorrhage, infection, and unknown cause.
About the Thymus and Congenital AthymiaThe “T”
in T cell stands for thymus because it is where T cells are
selected to fight infections or are destroyed if they have the
potential to attack the body instead of invaders. Congenital
athymia is an ultra-rare condition in which children are born
without a thymus, causing profound immunodeficiency, vulnerability
to potentially fatal infections, and life-threatening immune
dysregulation. With only supportive care, children with congenital
athymia typically die by age two or three. Congenital athymia is
initially detected by T-cell deficiency observed in newborn
screening for SCID (severe combined immune deficiency), which is
now required in all 50 U.S. states. SCID and congenital athymia are
both primary immunodeficiency disorders but they are distinct
conditions. The estimated incidence of pediatric congenital athymia
in the United States is 17 to 24 live births each year.
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
(RMAT), Breakthrough Therapy, Rare Pediatric Disease, and Orphan
Drug. It also has been granted the Orphan Drug designation and the
Advanced Therapy Medicinal Product (ATMP) designation by the
European Medicines Agency (EMA). 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.
Important Safety Information (ISI)
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 HCT and maternal
engraftment. GVHD may manifest as fever, rash, lymphadenopathy,
elevated bilirubin and liver enzymes, enteritis, and/or
diarrhea.
Treatment with RETHYMIC may increase the risk of
autoimmune-mediated conditions. 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 hematopoietic cell transplantation (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 http://www.fda.gov/safety/medwatch.
About EnzyvantEnzyvant, a wholly-owned
subsidiary of Sumitovant Biopharma Ltd. (wholly-owned by Sumitomo
Dainippon Pharma Co., Ltd.), is a biotechnology company dedicated
to developing novel, transformative regenerative therapies for
people with devastating rare diseases. Enzyvant’s RETHYMIC®
(previously RVT-802) is a one-time tissue-based regenerative
therapy approved by the U.S. Food and Drug Administration for
pediatric congenital athymia, an ultra-rare and life-threatening
pediatric immunodeficiency. For more information about Enzyvant,
visit Enzyvant.com. Follow @Enzyvant on Twitter, Facebook, and
LinkedIn.
About Sumitovant Biopharma Ltd.Sumitovant is a
global biopharmaceutical company leveraging data-driven insights to
rapidly accelerate development of new potential therapies for unmet
patient conditions. Through our unique portfolio of wholly-owned
“Vant” subsidiaries—Urovant, Enzyvant, Spirovant, Altavant—and use
of embedded computational technology platforms to generate business
and scientific insights, Sumitovant has supported development of
FDA-approved products including GEMTESA® for overactive bladder and
RETHYMIC® for pediatric congenital athymia, and has advanced a
promising pipeline of early-through late-stage investigational
assets for other serious conditions. Sumitovant is the
majority-shareholder of Myovant (NYSE: MYOV) whose marketed
products include ORGOVYX® for advanced prostate cancer and
MYFEMBREE® for uterine fibroids. Sumitovant is a wholly-owned
subsidiary of Sumitomo Dainippon Pharma. For more information,
please visit https://www.sumitovant.com.
About Sumitomo Dainippon Pharma Co.,
Ltd.Sumitomo Dainippon Pharma is among the top-ten listed
pharmaceutical companies in Japan, operating globally in major
pharmaceutical markets, including Japan, the U.S., China, and other
Asian countries. Sumitomo Dainippon Pharma is based on the merger
in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo
Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more
than 7,000 employees worldwide. Additional information about
Sumitomo Dainippon Pharma is available through its corporate
website at https://www.ds-pharma.com/.
Media Contacts:EnzyvantEliza
Schleifstein 6 Degrees (917)
763-8106Eliza@schleifsteinpr.com
Sumitovant BiopharmaMaya FrutigerVP, Corporate
Communicationsmedia@sumitovant.com
A photo accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/4b053993-b3fb-431e-981c-ddbbd721a29e
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