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Phathom Pharmaceuticals
Announces FDA Acceptance for Filing of
VOQUEZNA
® (
vonoprazan
) Tablets New Drug Application for the Treatment of
Heartburn
Associated with
Non-Erosive GERD
2023-12-06
·
BioSpace
临床3期
临床结果
申请上市
上市批准
New drug application (NDA) seeks
U.S. Food and Drug Administration (FDA)
approval for
VOQUEZNA
® as a daily treatment for
heartburn
associated with
Non-Erosive GERD
, the largest subcategory of
GERD
July 19, 2024 PDUFA target action date assigned by the FDA FLORHAM PARK, N.J., Dec. 06, 2023 (GLOBE NEWSWIRE) --
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 pro 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 . Please see full Prescribing Information for VOQUEZNA. About PHALCON-NERD-301 PHALCON-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 and follow the Company on LinkedIn and X (formerly Twitter). Forward-Looking Statement This 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 CONTACT Nick Benedetto 1-877-742-8466 media@phathompharma.com INVESTOR CONTACT Eric Sciorilli 1-877-742-8466 ir@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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机构
Phathom Pharmaceuticals, Inc.
US Food & Drug Administration
Takeda Pharmaceutical Co., Ltd.
适应症
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胃食管反流
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