Phathom Pharmaceuticals, Inc. (Nasdaq: PHAT), a biopharmaceutical
company focused on developing and commercializing novel treatments
for gastrointestinal diseases, announced today the FDA has accepted
for review the company’s NDA for VOQUEZNA (vonoprazan) as a daily
treatment of heartburn associated with Non-Erosive gastroesophageal
reflux disease (GERD) in adults. The FDA has assigned the
application a standard 10-month review with a Prescription Drug
User Fee Act (PDUFA) target action date of July 19, 2024.
"Today marks an important step forward for Phathom
Pharmaceuticals as we announce the FDA's acceptance for filing of
our New Drug Application for VOQUEZNA as a daily treatment of
heartburn for patients with Non-Erosive GERD,” said Azmi Nabulsi,
M.D., Chief Operating Officer at Phathom. “Non-Erosive GERD
represents a substantial segment of the U.S. GERD population,
affecting millions of individuals and impacting their quality of
life. The supportive data in our regulatory application include the
positive Phase 3 results from the investigational PHALCON-NERD-301
trial, along with a safety profile consistent with prior studies.
We look forward to working closely with the FDA throughout the
review process and if approved, expect to launch VOQUEZNA for this
new indication in the third quarter of 2024.”
Non-Erosive GERD is the largest subcategory of GERD and is
characterized by reflux-related symptoms in the absence of
esophageal mucosal erosions. There are an estimated 38 million U.S.
adults living with Non-Erosive GERD, of these approximately 15
million are diagnosed and treated with a prescription medicine
annually. Symptoms impact overall quality of life and can include
episodic heartburn, especially at night, regurgitation, problems
swallowing, and chest pain.
Phathom is also finalizing its plans to initiate an additional
Phase 3 study in 2024 evaluating VOQUEZNA as an investigational As
Needed treatment for episodic heartburn relief in adults with
Non-Erosive GERD, a novel dosing treatment regimen for which proton
pump inhibitors (PPIs) are not approved in the U.S.
INDICATIONS AND USAGE
VOQUEZNA® (vonoprazan) is a potassium-competitive acid blocker
(PCAB) indicated:
- for the healing of all grades of
Erosive Esophagitis (Erosive Gastroesophageal Reflux Disease or
Erosive GERD) and relief of heartburn associated with Erosive GERD
in adults.
- for the maintenance of healing of
all grades of Erosive GERD and relief of heartburn associated with
Erosive GERD in adults.
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS
VOQUEZNA is contraindicated in patients with a known
hypersensitivity to vonoprazan or any component of VOQUEZNA, or in
patients receiving rilpivirine-containing products.
WARNINGS AND PRECAUTIONS
Presence of Gastric Malignancy: In adults,
symptomatic response to therapy with VOQUEZNA does not preclude the
presence of gastric malignancy. Consider additional follow-up and
diagnostic testing in patients who have a suboptimal response or an
early symptomatic relapse after completing treatment with VOQUEZNA.
In older patients, also consider endoscopy.
Acute Tubulointerstitial Nephritis: Acute
tubulointerstitial nephritis (TIN) has been reported with VOQUEZNA.
If suspected, discontinue VOQUEZNA and evaluate patients with
suspected acute TIN.
Clostridioides difficile-Associated
Diarrhea: Published observational studies suggest that
proton pump inhibitors (PPIs) may be associated with an increased
risk of Clostridioides difficile-associated diarrhea (CDAD),
especially in hospitalized patients. VOQUEZNA may also increase the
risk of CDAD. Consider CDAD in patients with diarrhea that does not
improve. Use the shortest duration of VOQUEZNA appropriate to the
condition being treated.
Bone Fracture: Several published observational
studies suggest that PPI therapy may be associated with an
increased risk for osteoporosis-related fractures of the hip,
wrist, or spine, especially in patients receiving high dose
(multiple daily doses) and long-term therapy (a year or longer).
Bone fracture, including osteoporosis-related fracture, has also
been reported with vonoprazan. Use the shortest duration of
VOQUEZNA appropriate to the condition being treated. Patients at
risk for osteoporosis-related fractures should be managed according
to the established treatment guidelines.
Severe Cutaneous Adverse Reactions (SCAR):
Severe cutaneous adverse reactions, including Stevens-Johnson
syndrome (SJS) and toxic epidermal necrolysis (TEN) have been
reported with VOQUEZNA. Discontinue VOQUEZNA at the first signs or
symptoms of SCAR or other signs of hypersensitivity and consider
further evaluation.
Vitamin B12 (Cobalamin) Deficiency: Long-term
use of acid-suppressing drugs can lead to malabsorption of Vitamin
B12 caused by hypo- or achlorhydria. Vitamin B12 deficiency has
been reported postmarketing with vonoprazan. If clinical symptoms
consistent with vitamin B12 deficiency are observed in patients
treated with VOQUEZNA, consider further workup.
Hypomagnesemia and Mineral Metabolism:
Hypomagnesemia has been reported postmarketing with vonoprazan.
Hypomagnesemia may lead to hypocalcemia and/or hypokalemia and may
exacerbate underlying hypocalcemia in at-risk patients.
Consider monitoring magnesium levels prior to initiation of
VOQUEZNA and periodically in patients expected to be on prolonged
treatment, in patients taking drugs that may have increased
toxicity in the presence of hypomagnesemia or drugs that may cause
hypomagnesemia. Treatment of hypomagnesemia may require magnesium
replacement and discontinuation of VOQUEZNA.
Consider monitoring magnesium and calcium levels prior to
initiation of VOQUEZNA and periodically while on treatment in
patients with a preexisting risk of hypocalcemia. Supplement with
magnesium and/or calcium, as necessary. If hypocalcemia is
refractory to treatment, consider discontinuing VOQUEZNA.
Interactions with Diagnostic Investigations for
Neuroendocrine Tumors: Serum chromogranin A (CgA) levels
increase secondary to drug-induced decreases in gastric acidity.
The increased CgA level may cause false positive results in
diagnostic investigations for neuroendocrine tumors. Temporarily
discontinue VOQUEZNA treatment at least 14 days before assessing
CgA levels and consider repeating the test if initial CgA levels
are high.
Fundic Gland Polyps: Use of VOQUEZNA is
associated with a risk of fundic gland polyps that increases with
long-term use, especially beyond one year. Fundic gland polyps have
been reported with vonoprazan in clinical trials and during
postmarketing use with PPIs. Most patients who developed fundic
gland polyps were asymptomatic and fundic gland polyps were
identified incidentally on endoscopy. Use the shortest duration of
VOQUEZNA appropriate to the condition being treated.
ADVERSE REACTIONS:
Healing of Erosive GERD: The most common
adverse reactions (≥2% of patients in the VOQUEZNA arm) include
gastritis (3%), diarrhea (2%), abdominal distention (2%), abdominal
pain (2%), and nausea (2%).
Maintenance of Healed Erosive GERD: The most
common adverse reactions (≥3% of patients in the VOQUEZNA arm)
include gastritis (6%), abdominal pain (4%), dyspepsia (4%),
hypertension (3%), and urinary tract infection (3%).
DRUG INTERACTIONS
VOQUEZNA has the potential for clinically important drug
interactions, including interactions with drugs dependent on
gastric pH for absorption, drugs that are substrates for certain
CYP enzymes, and some diagnostic tests. Avoid concomitant use of
VOQUEZNA with atazanavir or nelfinavir. See full Prescribing
Information for more details about important drug interactions.
Consult the labeling of concomitantly used drugs to obtain further
information about interactions with vonoprazan.
USE IN SPECIFIC POPULATIONS
Lactation: Breastfeeding is not recommended
during treatment. Because of the potential risk of adverse liver
effects shown in animal studies with vonoprazan, advise patients
not to breastfeed during treatment with VOQUEZNA.
Renal Impairment: For the healing of Erosive
GERD, dosage reduction is recommended in patients with severe renal
impairment (eGFR < 30 mL/min).
Hepatic Impairment: For the healing of Erosive
GERD, dosage reduction is recommended in patients with moderate to
severe hepatic impairment (Child-Pugh Class B and C).
You are encouraged to report suspected adverse reactions
by contacting Phathom Pharmaceuticals at 1-888-775-PHAT (7428) or
FDA at 1-800-FDA-1088 or
www.fda.gov/medwatch.
Please see full Prescribing
Information for VOQUEZNA.
About PHALCON-NERD-301PHALCON-NERD-301 was a
randomized, placebo-controlled, double-blind, multicenter Phase 3
study that enrolled 772 adult patients with symptomatic Non-Erosive
GERD in the U.S. The trial evaluated the efficacy of VOQUEZNA 10 mg
and 20 mg as a daily dosing (QD) treatment, as compared to placebo
(QD), in the relief of heartburn over four weeks in participants
with symptomatic Non-Erosive GERD. The trial also included a
20-week blinded long-term extension period to further evaluate the
safety and efficacy of both doses of VOQUEZNA after six months of
continuous use.
About VOQUEZNA®VOQUEZNA® (vonoprazan) tablets
contain vonoprazan, an oral small
molecule potassium-competitive acid blocker (PCAB), approved
in the U.S. for the treatment of adults with Erosive Esophagitis,
also known as Erosive GERD, and the relief of heartburn associated
with Erosive GERD. PCABs are a novel class of medicines that block
acid secretion in the stomach. Phathom in-licensed the U.S.,
European, and Canadian rights to vonoprazan from Takeda, which
markets the product in Japan and numerous other countries in Asia
and Latin America.
About Phathom Pharmaceuticals,
Inc. Phathom Pharmaceuticals is a biopharmaceutical
company focused on the development and commercialization of novel
treatments for gastrointestinal diseases. Phathom has in-licensed
the exclusive rights in the U.S., Europe, and Canada to vonoprazan,
a first-in-class potassium-competitive acid blocker (PCAB). For
more information about Phathom, visit the Company’s website at
www.phathompharma.com and follow the Company on LinkedIn and X
(formerly Twitter).
Forward-Looking StatementThis press release
contains forward-looking statements. Investors are cautioned not to
place undue reliance on these forward-looking statements, including
statements about the timing of regulatory approval and launch of
VOQUEZNA for the treatment of heartburn associated with Non-Erosive
GERD, initiation of a Phase 3 trial evaluating VOQUEZNA as an As
Needed treatment of heartburn associated with Non-Erosive GERD, and
the size of the Non-Erosive GERD patient population. The inclusion
of forward-looking statements should not be regarded as a
representation by Phathom that any of its plans will be achieved.
Actual results may differ from those set forth in this press
release due to the risks and uncertainties inherent in Phathom’s
business, including, without limitation: we may not be able to
successfully commercialize VOQUEZNA which will depend on a number
of factors including coverage and reimbursement levels from
governmental authorities and health insurers as well as market
acceptance by healthcare providers; future data generated from our
stability program may be different from the data submitted to the
FDA to date and may not demonstrate that our mitigation efforts
will continue to maintain the level of the nitrosamine impurity
below the acceptable intake (AI) level throughout the shelf life of
products containing vonoprazan, which could result in market action
or shelf life reduction; risks associated with product
manufacturing or formulation changes required to be made in
connection with achieving the AI; the inherent risks of clinical
development of vonoprazan; Phathom’s dependence on third parties in
connection with product manufacturing, research and preclinical and
clinical testing; regulatory developments in the United States and
foreign countries; unexpected adverse side effects or inadequate
efficacy of vonoprazan that may limit its development, regulatory
approval and/or commercialization, or may result in recalls or
product liability claims; Phathom’s ability to access additional
capital under its term loan facility and royalty interest finance
agreements is subject to certain conditions; Phathom’s ability to
obtain and maintain intellectual property protection for
vonoprazan; Phathom’s ability to comply with its license agreement
with Takeda; and other risks described in the Company’s prior press
releases and the Company’s filings with the Securities and Exchange
Commission (SEC), including under the heading “Risk Factors” in the
Company’s Annual Report on Form 10-K and any subsequent filings
with the SEC. You are cautioned not to place undue reliance on
these forward-looking statements, which speak only as of the date
hereof, and Phathom undertakes no obligation to update such
statements to reflect events that occur or circumstances that exist
after the date hereof. All forward-looking statements are qualified
in their entirety by this cautionary statement, which is made under
the safe harbor provisions of the Private Securities Litigation
Reform Act of 1995.
MEDIA CONTACTNick
Benedetto1-877-742-8466media@phathompharma.com
INVESTOR CONTACTEric
Sciorilli1-877-742-8466ir@phathompharma.com
© 2023 Phathom Pharmaceuticals. All rights reserved.
VOQUEZNA, Phathom Pharmaceuticals, and their respective logos
are registered trademarks or trademarks of Phathom Pharmaceuticals,
Inc.
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