Prescription Drug User Fee Act

Takeda and HUTCHMED Announce Marketing Authorization Application of Fruquintinib for Previously Treated Metastatic Colorectal Cancer Validated by the European Medicines Agency

Retrieved on: 
Thursday, June 15, 2023

OSAKA, Japan and CAMBRIDGE, Mass. and HONG KONG and SHANGHAI, China and FLORHAM PARK, N.J., June 15, 2023 (GLOBE NEWSWIRE) -- Takeda (TSE:4502/​NYSE:TAK) and HUTCHMED (China) Limited (Nasdaq/AIM:​HCM, HKEX:​13) (“HUTCHMED”) today announced that the European Medicines Agency (“EMA”) has validated and accepted for regulatory review the marketing authorization application (“MAA”) for fruquintinib, a highly selective and potent inhibitor of vascular endothelial growth factor receptors (“VEGFR”) -1, -2 and -3 for the treatment of adult patients with previously treated metastatic colorectal cancer (“CRC”). If approved, fruquintinib will be the first and only highly selective inhibitor of all three VEGF receptors approved in the E.U. for previously treated metastatic CRC.1,2

Key Points: 
  • Placebo plus BSC for Adult Patients with Previously Treated Metastatic Colorectal Cancer —
    OSAKA, Japan and CAMBRIDGE, Mass.
  • If approved, fruquintinib will be the first and only highly selective inhibitor of all three VEGF receptors approved in the E.U.
  • for previously treated metastatic CRC.1,2
    “European patients with metastatic colorectal cancer have not benefitted from a treatment advancement in over a decade,” said Awny Farajallah, M.D., head of Global Medical Affairs Oncology at Takeda.
  • who find treatment options to be limited for previously treated metastatic colorectal cancer,” said Dr. Michael Shi, Head of R&D and Chief Medical Officer, HUTCHMED.

Iovance Biotherapeutics Announces First Patient Randomized in Phase 3 TILVANCE-301 Trial in Frontline Advanced Melanoma

Retrieved on: 
Thursday, June 15, 2023

Friedrich Graf Finckenstein, M.D., Chief Medical Officer of Iovance, stated, “Our strategy is to offer TIL therapy across all lines of treatment for patients with advanced melanoma.

Key Points: 
  • Friedrich Graf Finckenstein, M.D., Chief Medical Officer of Iovance, stated, “Our strategy is to offer TIL therapy across all lines of treatment for patients with advanced melanoma.
  • Randomizing the first patient in TILVANCE-301, our first Phase 3 trial at Iovance, is an important milestone.
  • The trial offers TIL therapy as part of an earlier treatment approach for frontline advanced melanoma, while serving as a confirmatory trial to convert an accelerated approval to full approval for lifileucel in post-anti-PD-1 melanoma.
  • TILVANCE-301 is expected to be well underway at the time of potential accelerated approval in this initial indication.

Takeda and HUTCHMED Announce Marketing Authorization Application of Fruquintinib for Previously Treated Metastatic Colorectal Cancer Validated by the European Medicines Agency

Retrieved on: 
Thursday, June 15, 2023

Takeda ( TSE:4502/NYSE:TAK ) and HUTCHMED (China) Limited (Nasdaq/AIM: HCM, HKEX: 13) (HUTCHMED) today announced that the European Medicines Agency (EMA) has validated and accepted for regulatory review the marketing authorization application (MAA) for fruquintinib, a highly selective and potent inhibitor of vascular endothelial growth factor receptors (VEGFR) -1, -2 and -3 for the treatment of adult patients with previously treated metastatic colorectal cancer (CRC).

Key Points: 
  • Takeda ( TSE:4502/NYSE:TAK ) and HUTCHMED (China) Limited (Nasdaq/AIM: HCM, HKEX: 13) (HUTCHMED) today announced that the European Medicines Agency (EMA) has validated and accepted for regulatory review the marketing authorization application (MAA) for fruquintinib, a highly selective and potent inhibitor of vascular endothelial growth factor receptors (VEGFR) -1, -2 and -3 for the treatment of adult patients with previously treated metastatic colorectal cancer (CRC).
  • “We are thrilled to have submitted the marketing authorization application to the EMA, bringing us one step closer to potentially offering this innovative therapy to patients with advanced disease.
  • FRESCO-2 is a global Phase 3 multi-regional clinical trial (MRCT) conducted in the U.S., Europe, Japan and Australia investigating fruquintinib plus best supportive care (BSC) vs placebo plus BSC in patients with previously treated metastatic CRC.
  • Submission of an NDA to the Japan Pharmaceuticals and Medical Devices Agency (PMDA) is also planned in 2023.

Phathom Pharmaceuticals Announces FDA Acceptance of NDA Resubmission for Erosive GERD

Retrieved on: 
Monday, June 12, 2023

The FDA has classified this as a Class 2 resubmission and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 17, 2023.

Key Points: 
  • The FDA has classified this as a Class 2 resubmission and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 17, 2023.
  • “We are thrilled to announce the FDA has set a PDUFA goal date of November 17, 2023, for our Erosive GERD NDA and has acknowledged that our resubmission responds to the nitrosamine related issues cited in the complete response letter we received earlier this year and allows the Agency to continue its review of our NDA,” said Terrie Curran, President and Chief Executive Officer of Phathom.
  • “This significant milestone brings us one step closer to the approval of a new class of treatment for Erosive GERD representing the first major innovation to the U.S. GERD market in over 30 years.
  • Phathom will continue to provide additional stability data during the regulatory review as previously agreed with FDA as part of this resubmission.

Capivasertib in combination with FASLODEX® (fulvestrant) granted Priority Review in the US for patients with advanced HR-positive breast cancer

Retrieved on: 
Monday, June 12, 2023

In January 2023, capivasertib was granted Fast Track Designation by the FDA in this setting for this patient population.

Key Points: 
  • In January 2023, capivasertib was granted Fast Track Designation by the FDA in this setting for this patient population.
  • FASLODEX is contraindicated in patients with known hypersensitivity to the drug or to any of its components.
  • PARP inhibitor olaparib is a targeted treatment option that has been studied in early and metastatic breast cancer patients with an inherited BRCA mutation.
  • AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.

Press Release: FDA Advisory Committee unanimously recommends nirsevimab as first immunization against RSV disease for all infants

Retrieved on: 
Thursday, June 8, 2023

The Committee also voted 19 to 2 in support of nirsevimab’s favorable benefit risk profile for children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season.

Key Points: 
  • The Committee also voted 19 to 2 in support of nirsevimab’s favorable benefit risk profile for children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season.
  • Nirsevimab builds on AstraZeneca’s strong science, leadership in RSV and commitment to addressing the needs of the most vulnerable.
  • Across all clinical endpoints, a single dose of nirsevimab demonstrated high and consistent efficacy against RSV LRTD sustained through the entire RSV season.
  • The AMDAC’s recommendation, while not binding, will be considered by the FDA during its review of the BLA for nirsevimab.

FDA Accepts Biologics License Applications for exagamglogene autotemcel (exa-cel) for Severe Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Friday, June 9, 2023

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Applications (BLAs) for the investigational treatment exagamglogene autotemcel (exa-cel) for severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Applications (BLAs) for the investigational treatment exagamglogene autotemcel (exa-cel) for severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
  • The FDA has granted Priority Review for SCD and Standard Review for TDT and assigned Prescription Drug User Fee Act (PDUFA) target action dates of December 8, 2023, and March 30, 2024, respectively.
  • Updated data from the pivotal trials supporting the regulatory submissions will be presented at the Annual European Hematology Association Congress on June 11, 2023.
  • In the U.K., exa-cel has also been granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA.

SpringWorks Therapeutics Announces PDUFA Date Extension for Nirogacestat NDA

Retrieved on: 
Monday, June 5, 2023

The previously disclosed August 27, 2023 PDUFA date has been extended by the standard extension period of three months.

Key Points: 
  • The previously disclosed August 27, 2023 PDUFA date has been extended by the standard extension period of three months.
  • The FDA notified SpringWorks on June 2, 2023 that it required more time to review additional analyses of previously submitted data that had been provided by SpringWorks in response to the FDA’s information requests.
  • The submission of this additional information was determined by the FDA to constitute a Major Amendment to the NDA, thereby resulting in an extension of the PDUFA action date.
  • In addition, SpringWorks expects to file a Marketing Authorization Application for nirogacestat with the European Medicines Agency in 2024.

U.S. Food and Drug Administration Accepts for Priority Review Bristol Myers Squibb’s Application for Repotrectinib for the Treatment of Patients with Locally Advanced or Metastatic ROS1-Positive Non-Small Cell Lung Cancer

Retrieved on: 
Tuesday, May 30, 2023

The FDA granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2023.

Key Points: 
  • The FDA granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2023.
  • “The FDA’s acceptance of this application marks an exciting milestone on our journey to bring this next-generation tyrosine kinase inhibitor to patients.
  • The study remains ongoing to assess long-term outcomes and additional endpoints across patient populations with ROS1-positive locally advanced or metastatic NSCLC and NTRK-positive advanced solid tumors.
  • As of August 2022, Bristol Myers Squibb acquired the leading clinical stage precision oncology company and its pipeline of investigational drugs across precision oncology and advanced solid tumors, including repotrectinib.

Iovance Biotherapeutics Announces U.S. Food and Drug Administration Acceptance of the Biologics License Application of Lifileucel for the Treatment of Advanced Melanoma

Retrieved on: 
Friday, May 26, 2023

SAN CARLOS, Calif., May 26, 2023 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, today announced that the U.S. Food and Drug Administration (FDA) accepted its Biologics License Application (BLA) for lifileucel for patients with advanced melanoma. The FDA granted lifileucel Priority Review and assigned November 25, 2023 as the target action date for a decision under the Prescription Drug User Fee Act (PDUFA). The FDA is not currently planning to hold an advisory committee meeting to discuss this application and, after a preliminary review, has not at this time identified any potential review issues.

Key Points: 
  • The FDA granted lifileucel Priority Review and assigned November 25, 2023 as the target action date for a decision under the Prescription Drug User Fee Act (PDUFA).
  • Lifileucel is a TIL therapy intended for patients with advanced melanoma who progressed on or after prior anti-PD-1/L1 therapy and targeted therapy, where applicable.
  • The FDA also previously granted a Regenerative Medicine Advanced Therapy ( RMAT ) designation for lifileucel in advanced melanoma.
  • If lifileucel receives accelerated approval, the randomized Phase 3 TILVANCE-301 trial in frontline advanced melanoma can serve as the confirmatory study to support full approval.