Priority review

Press Release: Dupixent® sBLA accepted for FDA Priority Review for treatment of COPD with type 2 inflammation

Retrieved on: 
Friday, February 23, 2024
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The U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Dupixent® (dupilumab) in a sixth potential indication as an add-on maintenance treatment in certain adult patients with uncontrolled chronic obstructive pulmonary disease (COPD).

Key Points: 
  • The U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Dupixent® (dupilumab) in a sixth potential indication as an add-on maintenance treatment in certain adult patients with uncontrolled chronic obstructive pulmonary disease (COPD).
  • The target action date for the FDA decision is June 27, 2024.
  • In both trials, Dupixent also rapidly and significantly improved lung function compared to placebo, with improvements sustained at 52 weeks.
  • Priority Review is granted to regulatory applications seeking approval for therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions.

AnHeart Therapeutics and Innovent Announce China’s NMPA has Accepted Second New Drug Application for Taletrectinib for First-Line Treatment of ROS1-Positive Lung Cancer

Retrieved on: 
Tuesday, March 5, 2024
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, NSCLC, Ophthalmology, Senior, New Drug Application, MD, ROS1, HKEX, Patient, CDE, Medicine, ELCC, NDA, NMPA, TKI, Cancer, Priority review, Pharmaceutical industry

This NDA is for taletrectinib as a first-line treatment for adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) who have not previously been treated with ROS1 TKIs.

Key Points: 
  • This NDA is for taletrectinib as a first-line treatment for adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) who have not previously been treated with ROS1 TKIs.
  • In 2023, China’s NMPA accepted taletrectinib’s first NDA and granted Priority Review Designation for adult patients with locally advanced or metastatic ROS1-positive NSCLC who have been previously treated with ROS1 TKIs.
  • Both NDAs in China are based on positive results from the Phase 2 TRUST-I (NCT04395677) trial.
  • “I am pleased this second NDA for taletrectinib has been accepted in China.

Indapta Therapeutics Receives U.S. FDA Fast Track Designation for Lead Clinical Drug Candidate IDP-023 for Non-Hodgkin’s Lymphoma and Myeloma

Retrieved on: 
Thursday, February 29, 2024
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, FDA, Clinical Trials, Fast Track, CMV, BLA, Cell, Biologics license application, Cytomegalovirus, Interleukin, Safety, Therapy, Cytokine, Accelerated approval (FDA), Patient, NK, Clinical trial, Multiple myeloma, Rituximab, Lymphoma, Cancer, Priority review, Pharmaceutical industry

Companies granted Fast Track designation may be eligible for more frequent interactions with the FDA, Accelerated Approval and Priority Review, and Rolling Review of a Biologic License Application (BLA).

Key Points: 
  • Companies granted Fast Track designation may be eligible for more frequent interactions with the FDA, Accelerated Approval and Priority Review, and Rolling Review of a Biologic License Application (BLA).
  • “This designation highlights the promise of Indapta’s highly potent NK cell platform and will further accelerate clinical development of our lead drug candidate, IDP-023, for two of the largest unmet needs in B-cell driven blood cancers, non-Hodgkin’s lymphoma and multiple myeloma,” said Dr. Mark Frohlich, CEO of Indapta.
  • Patients being enrolled now in Indapta’s Phase 1 clinical trial are receiving up to three planned doses of IDP-023 with or without interleukin-2.
  • To generate IDP-023, Indapta preferentially expands g-NK cells from healthy donors with increased numbers of g-NK cells, with low donor-to-donor variability.

Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2023 Financial Results and Recent Corporate Developments

Retrieved on: 
Wednesday, February 28, 2024
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Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the fourth quarter and full-year 2023.

Key Points: 
  • Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the fourth quarter and full-year 2023.
  • Beginning in the fourth quarter of 2023, amortization of in-licensed rights (formerly included within depreciation and amortization expense) and income tax (benefit) expense are no longer excluded from the non-GAAP results.
  • Non-GAAP financial results for the fourth quarter and full-year 2022 have been updated to reflect this change for comparability.
  • All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table “Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures.”

U.S. FDA Accepts for Priority Review the Supplemental Biologics License Application for Epcoritamab (EPKINLY®) for Difficult-to-Treat Relapsed or Refractory Follicular Lymphoma

Retrieved on: 
Tuesday, February 27, 2024
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, ABBV, BTD, ASH, Food, AbbVie, PDUFA, Exposition, Annual general meeting, Safety, Society, Patient, Diagnosis, Priority review, Pharmaceutical industry

The FDA grants Priority Review to investigational therapies that, if approved, may offer significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.

Key Points: 
  • The FDA grants Priority Review to investigational therapies that, if approved, may offer significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.
  • “The acceptance of the epcoritamab application for Priority Review marks an important milestone toward potentially providing a new treatment option to patients affected by R/R follicular lymphoma.
  • The FDA previously granted Breakthrough Therapy Designation (BTD) to epcoritamab for the treatment of adult patients with R/R FL after two or more lines of systemic therapy.
  • The companies will share commercial responsibilities in the U.S. and Japan, with AbbVie responsible for further global commercialization.

Incyte Announces U.S. Food and Drug Administration Grants Priority Review for Axatilimab for the Treatment of Chronic Graft-Versus-Host Disease

Retrieved on: 
Tuesday, February 27, 2024
FDA, Health, Stem Cells, Clinical Trials, Pharmaceutical, Biotechnology, Food, BLA, PDUFA, SNDX, GVHD, Safety, Society, Patient, Priority, Biologics license application, Priority review, Pharmaceutical industry

Incyte (Nasdaq:INCY) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for axatilimab, an anti-CSF-1R antibody, for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy.

Key Points: 
  • Incyte (Nasdaq:INCY) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for axatilimab, an anti-CSF-1R antibody, for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy.
  • The Prescription Drug User Fee Act (PDUFA) date for the FDA decision is August 28, 2024.
  • Axatilimab is being developed by Incyte and Syndax Pharmaceuticals (Nasdaq:SNDX) as part of an exclusive worldwide co-development and co-commercialization license agreement.
  • “Axatilimab’s novel mechanism offers a differentiated treatment approach which may help patients suffering from this devastating disease.

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Monday, February 26, 2024
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Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023.
  • “I am pleased with the strong results Rocket delivered in 2023, as we closed another successful year of progress across all six disclosed gene therapy programs spanning our AAV cardiovascular and LV hematology portfolios,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma.
  • Results from the study demonstrated the potential of RP-A601 as a gene therapy for patients with PKP2-ACM.
  • As of December 31, 2023, Rocket had cash, cash equivalents and investments of $407.5 million.

U.S. Food and Drug Administration Approves Opdivo® (nivolumab), in Combination with Cisplatin and Gemcitabine, for First-Line Treatment of Adult Patients with Unresectable or Metastatic Urothelial Carcinoma

Retrieved on: 
Thursday, March 7, 2024
Oncology, Health, FDA, General Health, Clinical Trials, Pharmaceutical, Biotechnology, Orbis, BMY, PFS, Progression-free survival, Confidence interval, Research, Recurrence, MD, Pneumonitis, Immunotherapy, Hematology, Kidney, Food, Cancer, Bladder cancer, HSCT, Hepatotoxicity, Checkmate, Risk, Bristol Myers Squibb, Hepatitis, Dexamethasone, Cisplatin, History, Death, Patient, Colitis, Multiple myeloma, Nephritis, Disease, Mortality, Priority review, Pharmaceutical industry

Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) approved Opdivo® (nivolumab), in combination with cisplatin and gemcitabine, for the first-line treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC), the most common type of bladder cancer.1,2 This approval is based on results from the Phase 3 CheckMate –901 trial which evaluated Opdivo in combination with cisplatin and gemcitabine followed by Opdivo monotherapy (n=304), compared to cisplatin-gemcitabine alone (n=304), for patients with previously untreated unresectable or metastatic UC.1,3 The primary efficacy endpoints were overall survival (OS) and progression-free survival (PFS) assessed by Blinded Independent Central Review (BICR).1

Key Points: 
  • Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) approved Opdivo® (nivolumab), in combination with cisplatin and gemcitabine, for the first-line treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC), the most common type of bladder cancer.1,2 This approval is based on results from the Phase 3 CheckMate –901 trial which evaluated Opdivo in combination with cisplatin and gemcitabine followed by Opdivo monotherapy (n=304), compared to cisplatin-gemcitabine alone (n=304), for patients with previously untreated unresectable or metastatic UC.1,3 The primary efficacy endpoints were overall survival (OS) and progression-free survival (PFS) assessed by Blinded Independent Central Review (BICR).1
    In the trial, with a median follow-up of approximately 33 months, treatment with Opdivo in combination with cisplatin and gemcitabine reduced the risk of death by 22%, demonstrating a median OS of 21.7 months versus 18.9 months with cisplatin-gemcitabine alone (Hazard Ratio [HR] 0.78; 95% Confidence Interval [CI]: 0.63, 0.96; p=0.0171).1,4 Patients receiving Opdivo in combination with cisplatin and gemcitabine had their risk of disease progression or death reduced by 28%, with a median PFS of 7.9 months compared to 7.6 months with cisplatin-gemcitabine alone (HR 0.72; 95% CI: 0.59, 0.88; p=0.0012).1
    Additionally, in exploratory analyses, treatment with Opdivo in combination with cisplatin and gemcitabine resulted in an objective response rate (ORR) of 57.6% (n=175) (95% CI: 51.8, 63.2) versus 43.1% (n=131) (95% CI: 37.5, 48.9) with cisplatin-gemcitabine alone.1,4 The complete response (CR) rate and partial response (PR) rate seen in patients treated with Opdivo in combination with cisplatin and gemcitabine was 22% (n=66) and 36% (n=109), respectively, versus 12% (n=36) and 31% (n=95) with cisplatin-gemcitabine alone.1
    “This approval marks an important advancement in a historically difficult-to-treat setting, where there has been a need for new and differentiated first-line approaches that may offer patients a chance to live longer,”5 said Guru P. Sonpavde, MD, Medical Director of Genitourinary Oncology and the Phase I Clinical Research Unit and Christopher K. Glanz Chair for Bladder Cancer Research at the AdventHealth Cancer Institute, Orlando, Florida.
  • “Based on outcomes and the safety profile seen in the CheckMate -901 clinical trial, the approval of Opdivo in combination with cisplatin and gemcitabine has the potential to change how metastatic or unresectable UC is treated for certain patients and offers them new hope.”1
    Opdivo is associated with the following Warnings & Precautions: severe and fatal immune-mediated adverse reactions, including pneumonitis, colitis, hepatitis and hepatotoxicity, endocrinopathies, dermatologic adverse reactions, nephritis with renal dysfunction, other immune-mediated adverse reactions; infusion-related reactions; complications of allogeneic hematopoietic stem cell transplantation (HSCT); embryo-fetal toxicity; and increased mortality in patients with multiple myeloma when Opdivo is added to a thalidomide analogue and dexamethasone, which is not recommended outside of controlled clinical trials.
  • Please see Important Safety Information below.1
    “Bringing Opdivo to the first-line setting in UC with chemotherapy is the latest realization of our history of research and progress in immunotherapy, which has helped transform the treatment landscape for many cancers, including bladder cancer,”1,6 said Wendy Short Bartie, senior vice president and general manager, U.S. Hematology and Oncology at Bristol Myers Squibb.
  • “This milestone adds a meaningful expansion to our portfolio of Opdivo-based treatments in genitourinary cancers, where we now have offerings in UC spanning three indications across stages of disease and treatment needs.”1
    The FDA previously approved Opdivo for the adjuvant treatment of adult patients with UC who are at high risk of recurrence after undergoing radical resection of UC; it also previously approved Opdivo for the treatment of adult patients with locally advanced or metastatic UC who have had disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.1
    Bristol Myers Squibb’s supplemental Biologics License Application (sBLA) leading to today’s approval was granted Priority Review status by the FDA, and was approved under the FDA’s Real-Time Oncology Review (RTOR) pilot program, which aims to ensure that safe and effective treatments are available to patients as early as possible.7 The review was also conducted under the FDA’s Project Orbis initiative, which enables concurrent review by the health authorities in several other countries where the application remains under review.

FDA Approves First Treatment to Reduce Risk of Serious Heart Problems Specifically in Adults with Obesity or Overweight

Retrieved on: 
Friday, March 8, 2024
Gastroesophageal reflux disease, Research, Heartburn, Blood, Diet, Cholesterol, Dietary supplement, Obesity, Dizziness, Pancreatitis, Vomiting, Health care, History, Thyroid cancer, Fatigue, Hypoglycemia, Acute kidney injury, Depression, Patient, Kidney disease, Abdominal distension, Burping, Cosmetics, Cardiovascular disease, Suicide, Weight, Stroke, Nausea, Diabetic retinopathy, Pancreas, Insulin, Blood pressure, Multimedia, Overweight, Diarrhea, Novo Nordisk, Vaccine, Safety, Heated tobacco product, Indigestion, Heart, Human services, Priority review, Division, Headache, Inflammation, FDA, Food, Weight loss, Thought, Heart rate, Gallstone, Risk, Animal, Allergy, Constipation, Death, Flatulence, Public

"This patient population has a higher risk of cardiovascular death, heart attack and stroke.

Key Points: 
  • "This patient population has a higher risk of cardiovascular death, heart attack and stroke.
  • Providing a treatment option that is proven to lower this cardiovascular risk is a major advance for public health."
  • Obesity or overweight affect approximately 70% of American adults.
  • Obesity and overweight are serious health issues that increase the risk for premature death and a variety of health problems, including heart attack and stroke.

U.S. Food and Drug Administration (FDA) Grants Priority Review of Epcoritamab (EPKINLY®) for Difficult-to-Treat Relapsed or Refractory Follicular Lymphoma (FL)

Retrieved on: 
Tuesday, February 27, 2024
ABBV, EMA, ASH, FDA, Food, Follicular lymphoma, AbbVie, Breakthrough therapy, Exposition, Cytokine release syndrome, Risk, Annual general meeting, Safety, Hematology, Society, European Medicines Agency, CRS, Patient, Diagnosis, Priority review, DLBCL

NORTH CHICAGO, Ill., Feb. 27, 2024 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review of the supplemental Biologics License Application (sBLA) for epcoritamab-bysp, a subcutaneously administered T-cell engaging bispecific antibody for the treatment of adult relapsed or refractory (R/R) follicular lymphoma (FL) after two or more lines of therapy. If approved, epcoritamab-bysp (EPKINLY®) will be the first and only subcutaneous bispecific antibody to treat adults with R/R FL after two lines of prior therapy, marking its second indication following FDA and European Medicines Agency (EMA) approval of R/R third-line diffuse large B-cell lymphoma (DLBCL) treatment.

Key Points: 
  • The FDA grants Priority Review to investigational therapies that, if approved, may offer significant improvements in the safety or effectiveness of the treatment, diagnosis or prevention of serious conditions when compared to standard applications.
  • This designation shortens the review period to six months compared to 10 months for Standard Review.
  • "Together with our partner Genmab, we are committed to further advancing epcoritamab as a core lymphoma therapy."
  • Both companies will pursue additional international regulatory approvals for the investigational R/R FL indication and additional approvals for the R/R DLBCL indication.