Full Press Release Details
Pharmaceuticals Announces European Commission Marketing Authorization for Pedmarqsi (sodium thiosulfate) to Reduce the Risk of
Hearing Loss in Pediatric Oncology Patients
is the First and Only Approved Therapy in the European Union for Reducing the Risk of Cisplatin-induced Hearing Loss (Ototoxicity) in
Pediatric Patients with Localized, Non-metastatic Solid Tumors ~
TRIANGLE PARK, N.C., June 6, 2023 - Fennec Pharmaceuticals Inc. (NASDAQ: FENC; TSX: FRX), a commercial stage specialty
pharmaceutical company, today announced Pedmarqsi - known as PEDMARK in the U.S. - was granted marketing
authorization by the European Commission. Pedmarqsi is the first and only approved therapy in the EU for the prevention of ototoxicity
(hearing loss) induced by cisplatin chemotherapy in patients 1 month to <18 years of age with localised, non-metastatic, solid tumors.
Further, Pedmarqsi was granted the marketing authorization under the paediatric-use marketing authorization (PUMA) which includes 8 years
plus 2 years of data and market protection.
"Today's approval by the European Commission
for Pedmarqsi to help reduce the risk of hearing loss is critical for the approximately four
thousand children treated with cisplatin for solid tumors that have not spread," said Rosty Raykov, chief executive officer of Fennec
Pharmaceuticals. "Fennec is proud to bring this safe and effective therapy to the marketplace to mitigate the risk of permanent
and irreversible bilateral hearing loss which occurs in approximately 60 percent of children treated with cisplatin and can be as high
as 90 percent.i We look forward to making this important treatment available to the pediatric oncology community in the EU
as soon as possible."
The European Commission
granted marketing authorization approval to Pedmarqsi following the adoption of a positive opinion by the Committee for Medicinal Products
for Human Use (CHMP) earlier this year. The CHMP's decision was based on safety and efficacy data from two pivotal open-label, randomized
Phase 3 trials (SIOPEL 6 and Clinical Oncology Group [COG] Protocol ACCL0431), which compared Pedmarqsi
plus cisplatin-based regimen to cisplatin-based regimens alone for the reduction of cisplatin-induced hearing loss in pediatric patients.
The marketing authorization
is applicable to all 27 European Union member states, as well as Iceland, Norway and Liechtenstein. PEDMARK was approved
by the U.S. Food and Drug Administration (FDA) in September 2022.
Cisplatin-Induced Ototoxicity
and other platinum compounds are essential chemotherapeutic agents for the treatment of many pediatric malignancies. Unfortunately, platinum-based
therapies can cause ototoxicity, or hearing loss, which is permanent, irreversible, and particularly harmful to the survivors of pediatric
of ototoxicity depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids or cochlear
implants, which can be helpful for some, but do not reverse the hearing loss and can be costly over time.iii Infants and
young children that are affected by ototoxicity at critical stages of development lack speech and language development and literacy, and
older children and adolescents often lack social-emotional development and educational achievement.iv
PEDMARK (sodium thiosulfate)
PEDMARK is the first and only
U.S. Food and Drug Administration (FDA) approved therapy indicated to reduce the risk of ototoxicity associated with cisplatin treatment
in pediatric patients with localized, non-metastatic, solid tumors. It is a unique formulation of sodium thiosulfate in single-dose,
ready-to-use vials for intravenous use in pediatric patients.7 PEDMARK is also the only therapeutic agent with proven efficacy
and safety data with an established dosing paradigm, across two open-label, randomized Phase 3 clinical studies, the Clinical Oncology
Group (COG) Protocol ACCL0431 and SIOPEL 6.
and Europe, it is estimated that, annually, more than 10,000 children may receive platinum-based chemotherapy. The incidence of ototoxicity
depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids. There is currently no established
preventive agent for this hearing loss and only expensive, technically difficult, and sub-optimal cochlear (inner ear) implants have been
shown to provide some benefit. Infants and young children that suffer ototoxicity at critical stages of development lack speech language
development and literacy, and older children and adolescents lack social-emotional development and educational achievement.
has been studied by co-operative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity, COG ACCL0431 and SIOPEL
6. Both studies have been completed. The COG ACCL0431 protocol enrolled childhood cancers typically treated with intensive cisplatin therapy
for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, medulloblastoma,
and other solid tumors. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors.
(sodium thiosulfate injection) is indicated to reduce the risk of ototoxicity associated with cisplatin in pediatric patients 1
month of age and older with localized, non-metastatic solid tumors.
The safety and efficacy of PEDMARK have not been
established when administered following cisplatin infusions longer than 6 hours. PEDMARK may not reduce the risk of ototoxicity when administered
following longer cisplatin infusions, because irreversible ototoxicity may have already occurred.
Important Safety Information
PEDMARK is contraindicated in patients with history
of a severe hypersensitivity to sodium thiosulfate or any of its components.
Hypersensitivity reactions occurred in 8% to 13%
of patients in clinical trials. Monitor patients for hypersensitivity reactions. Immediately discontinue PEDMARK and institute appropriate
care if a hypersensitivity reaction occurs. Administer antihistamines or glucocorticoids (if appropriate) before each subsequent administration
of PEDMARK. PEDMARK may contain sodium sulfite; patients with sulfite sensitivity may have hypersensitivity reactions, including anaphylactic
symptoms and life-threatening or severe asthma episodes. Sulfite sensitivity is seen more frequently in people with asthma.
PEDMARK is not indicated for use in pediatric
patients less than 1 month of age due to the increased risk of hypernatremia or in pediatric patients with metastatic cancers.
Hypernatremia occurred in 12% to 26% of patients
in clinical trials, including a single Grade 3 case. Hypokalemia occurred in 15% to 27% of patients in clinical trials, with Grade 3 or
4 occurring in 9% to 27% of patients. Monitor serum sodium and potassium levels at baseline and as clinically indicated. Withhold PEDMARK
in patients with baseline serum sodium greater than 145 mmol/L.
Monitor for signs and symptoms of hypernatremia
and hypokalemia more closely if the glomerular filtration rate (GFR) falls below 60 mL/min/1.73m2.
Administer antiemetics prior to each PEDMARK administration.
Provide additional antiemetics and supportive care as appropriate.
The most common adverse reactions ( 25% with
difference between arms of >5% compared to cisplatin alone) in SIOPEL 6 were vomiting, nausea, decreased hemoglobin, and hypernatremia.
The most common adverse reaction ( 25% with difference between arms of >5% compared to cisplatin alone) in COG ACCL0431 was hypokalemia.
Please see full Prescribing Information for PEDMARK
at: www.PEDMARK.com.
Fennec Pharmaceuticals Inc. is a specialty pharmaceutical
company focused on the development and commercialization of PEDMARK and Pedmarqsi to reduce the risk of platinum-induced
ototoxicity in pediatric patients. Further, PEDMARK received FDA approval in September 2022 and European Commission Marketing Authorization
in June 2023 for Pedmarqsi. PEDMARK has received Orphan Drug Exclusivity in the U.S. for seven years of market protection and Pedmarqsi
has received Pediatric Use Marketing Authorization in Europe which includes eight years plus two years of data and market protection.
Fennec has a license agreement with Oregon Health and Science University (OHSU) for exclusive worldwide license rights to intellectual
property directed to sodium thiosulfate and its use for chemoprotection, including the reduction of risk of ototoxicity induced by platinum
chemotherapy, in humans. For more information, please visit www.fennecpharma.com.
Forward Looking Statements
Except for historical information described
in this press release, all other statements are forward-looking. Words such as "believe," "anticipate," "plan,"
"expect," "estimate," "intend," "may," "will," or the negative of those terms,
and similar expressions, are intended to identify forward-looking statements. These forward-looking statements include statements about
our business strategy, timeline and other goals, plans and prospects, including our commercialization plans respecting PEDMARK
and Pedmarqsi, the market opportunity for and market impact of PEDMARK and Pedmarqsi, its potential impact on patients
and anticipated benefits associated with its use, and potential access to further funding after the date of this release. Forward-looking
statements are subject to certain risks and uncertainties inherent in the Company's business that could cause actual results to
vary, including the risks and uncertainties that regulatory and guideline developments may change, scientific data and/or manufacturing
capabilities may not be sufficient to meet regulatory standards or receipt of required regulatory clearances or approvals, clinical results
may not be replicated in actual patient settings, unforeseen global instability, including political instability, or instability from
an outbreak of pandemic or contagious disease, such as the novel coronavirus (COVID-19), or surrounding the duration and severity of
an outbreak, protection offered by the Company's patents and patent applications may be challenged, invalidated or circumvented
by its competitors, the available market for the Company's products will not be as large as expected, the Company's products
will not be able to penetrate one or more targeted markets, revenues will not be sufficient to fund further development and clinical