STOCKHOLM, Dec. 15, 2021 /PRNewswire/ -- Calliditas
Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX)
("Calliditas") today announced that the US Food and Drug
Administration (FDA) has approved TARPEYO (budesonide) delayed
release capsules to reduce proteinuria in adults with primary
immunoglobulin A nephropathy (IgAN) at risk of rapid disease
progression, generally a urine protein-to-creatinine ratio (UPCR)
≥1.5g/g. This indication is approved under accelerated approval. It
has not been established whether TARPEYO slows kidney function
decline in patients with IgAN. Continued approval may be contingent
upon verification and description of clinical benefit in a
confirmatory clinical trial.1
This approval marks the successful transition for Calliditas to
a commercial-stage biopharmaceutical company.
"We are very excited to bring the first and only FDA-approved
treatment to reduce proteinuria in IgAN to market," said
Renée Aguiar-Lucander, Chief Executive Officer of Calliditas.
"TARPEYO represents an FDA approved product to help these patients
who are at risk of rapid disease progression."
TARPEYO is approved under accelerated approval based on
achieving its primary endpoint of reduction in proteinuria in Part
A of the NeflgArd pivotal Phase 3 study, an ongoing, randomized,
double-blind, placebo-controlled, multicenter study conducted to
evaluate the efficacy and safety of TARPEYO 16 mg once daily vs
placebo in adult patients with primary IgAN.1 The effect
of TARPEYO was assessed in patients with biopsy-proven IgAN, eGFR
≥35 mL/min/1.73 m2, and
proteinuria (defined as either ≥1 g/day or UPCR ≥0.8 g/g) who were
on a stable dose of maximally-tolerated RAS inhibitor therapy.
Patients taking TARPEYO (n=97) showed a statistically
significant 34% reduction in proteinuria from baseline vs 5% with
RASi alone (n=102) at 9 months. The treatment effects for the
primary endpoint of UPCR at 9 months were consistent across key
subgroups, including key demographic and baseline disease
The most common adverse reactions (≥5%) in this study were
hypertension, peripheral edema, muscle spasms, acne, dermatitis,
weight increase, dyspnea, face edema, dyspepsia, fatigue, and
hirsutism. Please see additional Important Safety Information
Richard Lafayette M.D., Professor
of Medicine at Stanford University and
the Director of the Stanford Glomerular Disease Center commented,
"IgAN is a tough diagnosis for many patients, and it can
progressively lead to the need for dialysis and/or kidney
transplantation. The FDA approval of TARPEYO now offers
disease-specific treatment for patients with this complicated
Richard Philipson, Calliditas
Chief Medical Officer added, "TARPEYO was developed to target a
root cause of IgAN. The FDA's approval of TARPEYO demonstrates our
unwavering dedication to patients suffering from IgAN. We
would like to thank the patients, researchers and clinical staff
who participated in the studies of TARPEYO."
Bonnie Schneider, Director and
Co-Founder of the IGA Nephropathy Foundation of America commented,"
It has been a difficult journey not only for our family but for all
the IgA nephropathy patients we serve. Having this disease specific
option has our community very excited."
It is expected that TARPEYO will be available in the U.S. early
in the first quarter of 2022. To assist patients and their
healthcare providers who would prescribe TARPEYO, Calliditas is
launching a comprehensive patient support program, TARPEYO
Touchpoints™. This program offers services, assistance, and
resources designed to help patients access treatment as easily as
possible. To learn more visit TARPEYOTouchpoints.com or call
For access to additional materials related to this announcement,
click here to locate the media kit.
Investor presentation December
16, 8:00 ET / 14:00 CET
Calliditas will host an audio cast with teleconference, with
presentations from CEO Renée Aguiar-Lucander and Andrew Udell, President of North America, December
16, 8:00 ET / 14:00 CET.
Teleconference: SE: +46 856642651 PIN: 44920298# | UK: +44
3333000804 PIN: 44920298# | US: +1 6319131422 PIN: 44920298#
INDICATION and IMPORTANT SAFETY INFORMATION
TARPEYO™ (budesonide) delayed release capsules is a
corticosteroid indicated to reduce proteinuria in adults with
primary immunoglobulin A nephropathy (IgAN) at risk of rapid
disease progression, generally a urine protein-to-creatinine ratio
(UPCR) ≥1.5 g/g.
This indication is approved under accelerated approval based on
a reduction in proteinuria. It has not been established whether
TARPEYO slows kidney function decline in patients with IgAN.
Continued approval for this indication may be contingent upon
verification and description of clinical benefit in a confirmatory
Important Safety Information
Contraindications: TARPEYO is contraindicated in patients
with hypersensitivity to budesonide or any of the ingredients of
TARPEYO. Serious hypersensitivity reactions, including anaphylaxis,
have occurred with other budesonide formulations.
Warnings and Precautions
Hypercorticism and adrenal axis suppression: When
corticosteroids are used chronically, systemic effects such as
hypercorticism and adrenal suppression may occur. Corticosteroids
can reduce the response of the hypothalamus-pituitary-adrenal (HPA)
axis to stress. In situations where patients are subject to surgery
or other stress situations, supplementation with a systemic
corticosteroid is recommended. When discontinuing therapy [see
Dosing and Administration] or switching between
corticosteroids, monitor for signs of adrenal axis suppression.
Patients with moderate to severe hepatic impairment (Child-Pugh
Class B and C, respectively) could be at an increased risk of
hypercorticism and adrenal axis suppression due to an increased
systemic exposure to oral budesonide. Avoid use in patients with
severe hepatic impairment (Child-Pugh Class C). Monitor for
increased signs and/or symptoms of hypercorticism in patients with
moderate hepatic impairment (Child-Pugh Class B).
Risks of Immunosuppression: Patients who are on drugs
that suppress the immune system are more susceptible to infection
than healthy individuals. Chicken pox and measles, for example, can
have a more serious or even fatal course in susceptible patients or
patients on immunosuppressive doses of corticosteroids. Avoid
corticosteroid therapy in patients with active or quiescent
tuberculosis infection; untreated fungal, bacterial, systemic
viral, or parasitic infections; or ocular herpes simplex. Avoid
exposure to active, easily transmitted infections (eg, chicken pox,
measles). Corticosteroid therapy may decrease the immune response
to some vaccines.
Other corticosteroid effects: TARPEYO is a systemically
available corticosteroid and is expected to cause related adverse
reactions. Monitor patients with hypertension, prediabetes,
diabetes mellitus, osteoporosis, peptic ulcer, glaucoma, cataracts,
a family history of diabetes or glaucoma, or with any other
condition in which corticosteroids may have unwanted effects.
Adverse reactions: In clinical studies, the most common
adverse reactions with TARPEYO (occurring in ≥5% of TARPEYO
patients and ≥2% higher than placebo) were hypertension (16%),
peripheral edema (14%), muscle spasms (13%), acne (11%), dermatitis
increase (7%), dyspnea (6%), face edema (6%), dyspepsia (5%),
fatigue (5%), and hirsutism (5%).
Drug interactions: Budesonide is a substrate for CYP3A4.
Avoid use with potent CYP3A4 inhibitors, such as ketoconazole,
itraconazole, ritonavir, indinavir, saquinavir, erythromycin, and
cyclosporine. Avoid ingestion of grapefruit juice with TARPEYO.
Intake of grapefruit juice, which inhibits CYP3A4 activity, can
increase the systemic exposure to budesonide.
Use in specific populations
Pregnancy: The available data from published case series,
epidemiological studies, and reviews with oral budesonide use in
pregnant women have not identified a drug-associated risk of major
birth defects, miscarriage, or other adverse maternal or fetal
outcomes. There are risks to the mother and fetus associated with
IgAN. Infants exposed to in utero corticosteroids, including
budesonide, are at risk for hypoadrenalism.
Please see Full Prescribing Information for TARPEYO
Calliditas has introduced TARPEYO, to reduce proteinuria in
adults with primary IgAN at risk of rapid disease progression,
generally a UPCR≥1.5g/g. This indication is approved under
accelerated approval based on a reduction in proteinuria. It has
not been established whether TARPEYO slows kidney function decline
in patients with IgAN. Continued approval for this indication may
be contingent upon verification and description of clinical benefit
in a confirmatory clinical trial.1
TARPEYO is an oral, delayed release formulation of budesonide, a
corticosteroid with potent glucocorticoid activity and weak
mineralocorticoid activity that undergoes substantial first pass
metabolism. TARPEYO was designed as a 4 mg delayed release capsule
and is enteric coated so that it would remain intact until it
reaches the ileum. Each capsule contains coated beads of budesonide
that target mucosal B-cells present in the ileum, including the
Peyer's patches, which are responsible for the production of
galactose-deficient IgA1 antibodies (Gd-Ag1) causing IgA
nephropathy. It is unclear to what extent TARPEYO's efficacy is
mediated via local effects in the ileum vs systemic
About the NeflgArd Study
The global clinical trial NeflgArd is an ongoing Phase 3,
randomized, double-blind, placebo- controlled, multicenter study to
evaluate the efficacy and safety of TARPEYO 16 mg once daily vs
placebo in adult patients with primary IgAN (N=360) as an addition
to optimized RASi therapy.
The effect of TARPEYO was assessed in patients with
biopsy-proven IgAN, eGFR ≥35 mL/min/1.73 m2, and proteinuria
(defined as either ≥1 g/day or UPCR ≥0.8 g/g) who were on a stable
dose of maximally-tolerated RAS inhibitor therapy.
Part A of the study included a 9-month blinded treatment period
and a 3-month follow-up period. The primary endpoint was UPCR, and
eGFR was a secondary endpoint. Part B, a confirmatory validation
study in which no TARPEYO treatment will be administered, will
assess eGFR at two years.
The trial met its primary objective in Part A of demonstrating a
statistically significant reduction in urine protein creatinine
ratio, UPCR or proteinuria, after 9 months of treatment with 16 mg
once daily of TARPEYO compared to placebo. Patients taking TARPEYO
plus RASi (n=97) showed a statistically significant 34% reduction
from baseline vs 5% with RASi alone (n=102) at 9 months, resulting
in UPCR reduction of 31% (16% to 42%) p=0.0001
About Primary Immunoglobulin A Nephropathy
Primary immunoglobulin A nephropathy (IgA nephropathy or IgAN or
Berger's Disease) is a rare, progressive, chronic autoimmune
disease that attacks the kidneys and occurs when
galactose-deficient IgA1 are recognized by autoantibodies, creating
IgA1 immune complexes that become deposited in the glomerular
mesangium of the kidney.4,5 This deposition in the
kidney can lead to progressive kidney damage and potentially a
clinical course resulting in end-stage renal disease. IgAN most
often develops between late teens and late 30s.5,6
Calliditas Therapeutics is a biopharma company headquartered in
Stockholm, Sweden, focused on
identifying, developing, and commercializing novel treatments in
orphan indications, with an initial focus on renal and hepatic
diseases with significant unmet medical needs.
Calliditas is listed on Nasdaq Stockholm (ticker: CALTX) and the
Nasdaq Global Select Market (ticker: CALT).
Visit www.calliditas.com for further information.
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995, as amended, including, without limitation, statements
regarding Calliditas' strategy, business plans, regulatory
submissions, and focus. The words "may," "will," "could," "would,"
"should," "expect," "plan," "anticipate," "intend," "believe,"
"estimate," "predict," "project," "potential," "continue,"
"target," and similar expressions are intended to identify
forward-looking statements, although not all forward-looking
statements contain these identifying words. Any forward-looking
statements in this press release are based on management's current
expectations and beliefs and are subject to a number of risks,
uncertainties, and important factors that may cause actual events
or results to differ materially from those expressed or implied by
any forward-looking statements contained in this press release,
including, without limitation, any related to Calliditas' business,
operations, continued FDA approval for TARPEYO, market acceptance
of TARPEYO, clinical trials, supply chain, strategy, goals and
anticipated timelines, competition from other biopharmaceutical
companies, and other risks identified in the section entitled "Risk
Factors" in Calliditas' reports filed with the Securities and
Exchange Commission. Calliditas cautions you not to place undue
reliance on any forward-looking statements, which speak only as of
the date they are made. Calliditas disclaims any obligation to
publicly update or revise any such statements to reflect any change
in expectations or in events, conditions, or circumstances on which
any such statements may be based, or that may affect the likelihood
that actual results will differ from those set forth in the
forward-looking statements. Any forward-looking statements
contained in this press release represent Calliditas' views only as
of the date hereof and should not be relied upon as representing
its views as of any subsequent date.
The information in the press release is inside information
that Calliditas is obliged to make public pursuant to the EU Market
Abuse Regulation. The information was submitted for publication,
through the agency of the contact persons below, on December 15, 2021 at 3:45
For further information, please contact:
Marie Galay, Investor Relations
Tel: +44 79 55 12 98 45; email: email@example.com
Sky Striar, Calliditas Media
Contact, LifeSci Communications Tel: +1.617.797.6672; email:
1. TARPEYOTM (budesonide) [prescribing
information]. Stockholm, SE:
Calliditas Therapeutics AB; 2021
2. Fellstrom BC, Barratt J, Cook H, et
al. Targeted-release budesonide versus placebo in patients with IgA
nephropathy (NEFIGAN): a double-blind, randomised,
placebo-controlled phase 2b trial.
Lancet. May 27,
3. Hastings, M. C., Bursac, Z., Julian,
B. A., Villa Baca, E., Featherston, J., Woodford, S. Y., Bailey,
L., & Wyatt, R. J. (2018). Life Expectancy for Patients From
the Southeastern United States With IgA Nephropathy. Kidney Int
Rep, 3(1), 99-104. https://doi.org/10.1016/j. ekir.2017.08.008
4. Barratt, J., & Feehally, J.
(2005). IgA nephropathy. J Am Soc Nephrol, 16(7), 2088-2097.
5. Barratt, J., Rovin, B. H., Cattran,
D., et al. (2020). Why Target the Gut to Treat IgA Nephropathy?
Kidney Int Rep, 5(10), 1620-1624.
6. Jarrick, S., Lundberg, S., Welander,
A., et al. (2019). Mortality in IgA Nephropathy: A Nationwide
Population-Based Cohort Study. J Am Soc Nephrol, 30(5), 866-876.
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