Eton Pharmaceuticals Announces Availability of Orphan Drug ALKINDI® SPRINKLE (hydrocortisone) in the United States
November 24 2020 - 9:00AM
Eton Pharmaceuticals, Inc (Nasdaq: ETON), a specialty
pharmaceutical company focused on developing and commercializing
innovative treatments for rare pediatric diseases, today announced
the full availability of ALKINDI
® SPRINKLE for
sale and distribution in the United States. The U.S. Food and Drug
Administration (FDA) has approved ALKINDI
®
SPRINKLE (hydrocortisone) oral granules as replacement therapy for
Adrenocortical Insufficiency (AI) in children under 17 years of
age. ALKINDI
® SPRINKLE is the first and only
FDA-approved granular hydrocortisone formulation for the treatment
of adrenocortical insufficiency specifically designed for use in
children.
ALKINDI® SPRINKLE will be available exclusively
through AnovoRx, a specialty pharmacy dedicated to serving patients
with rare and chronic conditions. AnovoRx will administer the Eton
Cares Program in partnership with Eton Pharmaceuticals. The program
will complete prescription fulfillment, insurance benefits
investigation, educational support, aid qualified patients to
obtain financial assistance along with other services designed to
help patients access treatment. To enroll patients in the program
and prescribe ALKINDI® SPRINKLE, clinicians will
need to complete a patient referral form available at
www.alkindisprinkle.com.
“ALKINDI® SPRINKLE is a very welcome treatment
option for children who, for over 60 years, were forced to rely on
adult-strength hydrocortisone to treat adrenocortical
insufficiency,” said Mitchell Geffner MD, co-director, Congenital
Adrenal Hyperplasia (CAH) Comprehensive Care Clinic and Professor
of Pediatrics, Keck School of Medicine of University of Southern
California. “Low-dose options, as low as 0.5 mg, allow for more
accurate and individualized dosing for patients.” The FDA approval
of ALKINDI® SPRINKLE was supported by six clinical
studies, including the first and only interventional Phase III
study of oral hydrocortisone for Pediatric AI in neonates to
children under eight years of age. ALKINDI SPRINKLE achieved
significant increases in cortisol levels from baseline
(P<0.0001) and was found to be well tolerated with no serious
adverse events. Prior to the approval of ALKINDI®
SPRINKLE, oral hydrocortisone was only FDA-approved in tablet
formulations of 5 mg and stronger. Many pediatric patients require
significantly lower doses and the flexibility of precision
titration. ALKINDI® SPRINKLE will be available in
0.5-mg, 1-mg, 2-mg, and 5-mg strengths, allowing clinicians greater
flexibility to individualize dosing based on each patient’s needs
in accordance with the instructions for dosage and administration.
About Pediatric Adrenocortical
InsufficiencyPediatric adrenocortical
Insufficiency (AI) is a relatively rare disease characterized by an
inability to synthesize and release cortisol, and sometimes
aldosterone. When due to congenital adrenal hyperplasia (CAH), the
most common form of pediatric AI, there will also be excessive
androgens (resulting in masculinized external genitalia in affected
females and, without proper treatment, the potential for precocious
puberty, premature growth termination, and short stature in both
sexes). Patients with primary or central (secondary and tertiary)
AI lack appropriate levels of cortisol in their system. Diminished
cortisol in the system may result in deadly consequences such as
adrenal crisis. To survive, patients with AI must replace the
missing cortisol daily. Eton estimates that pediatric AI affects
between 5,000 and 11,000 children in the United
States.About ALKINDI
SPRINKLEALKINDI® SPRINKLE is an
immediate-release oral hydrocortisone granule preparation that has
been specifically designed to meet the dosing needs of pediatric
patients with adrenocortical insufficiency. Prior to
ALKINDI® SPRINKLE’s approval, parent caregivers
have had to cut or split higher-strength hydrocortisone tablets to
achieve the lower doses required for small children, which could
result in inaccurate dosing. ALKINDI® SPRINKLE is
manufactured using commercially proven technology in four
strengths: 0.5 mg, 1 mg, 2 mg and 5 mg, to give greater dosing
flexibility to clinicians. Taste-masking excipients that are
acceptable for pediatric use eliminate the bitter taste of
hydrocortisone. ALKINDI® SPRINKLE has a shelf
-life of three years at ambient temperature and does not require
refrigeration.
Indications and UsageALKINDI®
SPRINKLE is a corticosteroid indicated as replacement therapy in
pediatric patients with adrenocortical
insufficiency.Important Safety
InformationContraindications
ALKINDI® SPRINKLE is contraindicated in
patients with hypersensitivity to hydrocortisone or to any of the
ingredients in ALKINDI® SPRINKLE. Anaphylactic
reactions have occurred in patients receiving
corticosteroids.Warning and Precautions
- Adrenal Crisis: Undertreatment or sudden
discontinuation of therapy may lead to adrenal insufficiency,
adrenal crisis, and death. Adrenal crisis may also be induced by
stress events such as infections or surgery. Increase the dose
during periods of stress. Switch patients who are vomiting,
severely ill, or unable to take oral medications to parenteral
corticosteroid formulations.
- Infections: Excessive doses may increase the
risks of new infections or exacerbation of latent infections with
any pathogen, including viral, bacterial, fungal, protozoan, or
helminthic. Monitor patients for signs and symptoms of infections.
Treat all infections seriously and initiate stress-dosing of
corticosteroids early.
- Growth Retardation: Long-term use in excessive
doses may cause growth retardation. Use the minimum dosage of
ALKINDI® SPRINKLE to achieve the desired clinical
response and monitor the patient’s growth.
- Cushing Syndrome Due to Use of Excessive Doses of
Corticosteroids: Prolonged use with supraphysiologic doses
may cause Cushing syndrome. Monitor patients for signs and symptoms
of Cushing syndrome every 6 months; pediatric patients under one
year of age may require more frequent monitoring.
- Decrease in Bone Mineral Density:
Corticosteroids decrease bone formation and increase bone
resorption which may lead to inhibition of bone growth and
development of osteoporosis. Use the minimum dosage of
ALKINDI® SPRINKLE to achieve desired clinical
response.
- Psychiatric Adverse Reactions: Use may be
associated with severe psychiatric adverse reactions such as
euphoria, mania, psychosis with hallucinations, and delirium or
depression. Symptoms typically emerge within a few days or weeks of
starting the treatment. Most reactions resolve after either dose
reduction or withdrawal, although specific treatment may be
necessary. Monitor patients for behavioral and mood disturbances
during treatment. Instruct caregivers and/or patients to seek
medical advice if psychiatric symptoms develop.
- Ophthalmic Adverse Reactions: Cataracts,
glaucoma, and central serous chorioretinopathy have been reported
with prolonged use of high doses. Monitor patients for blurred
vision or other visual disturbances and, if they occur, refer them
to an ophthalmologist.
- Gastrointestinal Adverse Reactions: Increased
risk in patients with certain gastrointestinal disorders. Signs and
symptoms may be masked.
Adverse ReactionsThe most common adverse
reactions from corticosteroids include fluid retention, alteration
in glucose tolerance, elevation in blood pressure, behavioral and
mood changes, and increased appetite and weight gain.About
Eton PharmaceuticalsEton Pharmaceuticals, Inc. is a
specialty pharmaceutical company focused on developing and
commercializing innovative treatments for rare pediatric diseases.
Eton is primarily focused on hospital injectable and pediatric rare
disease products. The company currently owns or receives royalties
from three FDA-approved approved products, including ALKINDI®
SPRINKLE, Biorphen®, and Alaway® Preservative Free, and has six
additional products that have been submitted to the FDA.
Forward-Looking StatementsStatements contained
in this press release regarding matters that are not historical
facts are "forward-looking statements" within the meaning of the
Private Securities Litigation Reform Act of 1995, including
statements associated with the expected ability of Eton to
undertake certain activities and accomplish certain goals and
objectives. These statements include but are not limited to
statements regarding Eton’s business strategy, Eton’s plans to
develop and commercialize its product candidates, the safety and
efficacy of Eton’s product candidates, Eton’s plans and expected
timing with respect to regulatory filings and approvals, and the
size and growth potential of the markets for Eton’s product
candidates. Because such statements are subject to risks and
uncertainties, actual results may differ materially from those
expressed or implied by such forward-looking statements. Words such
as "believes," "anticipates," "plans," "expects," "intends,"
"will," "goal," "potential," and similar expressions are intended
to identify forward-looking statements. These forward-looking
statements are based upon Eton’s current expectations and involve
assumptions that may never materialize or may prove to be
incorrect. Actual results and the timing of events could differ
materially from those anticipated in such forward-looking
statements as a result of various risks and uncertainties, which
include, without limitation, risks associated with the process of
discovering, developing, and commercializing drugs that are safe
and effective for use as human therapeutics, and in the endeavor of
building a business around such drugs. These and other risks
concerning Eton’s development programs and financial position are
described in additional detail in Eton’s filings with the
Securities and Exchange Commission. All forward-looking statements
contained in this press release speak only as of the date on which
they were made. Eton undertakes no obligation to update such
statements to reflect events that occur or circumstances that exist
after the date on which they were made.
Company Contact:David
Krempadkrempa@etonpharma.com612-387-3740
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