PDUFA

Autolus Therapeutics announces acceptance of Biologics License Application for obecabtagene autoleucel (obe-cel) as a potential treatment for relapsed/refractory Adult B-cell Acute Lymphoblastic Leukemia (ALL)

Retrieved on: 
Monday, January 22, 2024
Patient, Therapy, European Medicines Agency, ALL, ASCO, Orphan, Hematology, Pivotal, Priority, Society, EMA, Food, FDA, BLA, ASH, Annual general meeting, PDUFA, File, Pharmaceutical industry

Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of November 16, 2024, a standard review timeline consistent with recently approved CAR T therapies.

Key Points: 
  • Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of November 16, 2024, a standard review timeline consistent with recently approved CAR T therapies.
  • The FDA is not currently planning to hold an advisory committee meeting to discuss this application.
  • The BLA submission is based on data from the Pivotal Phase 2 FELIX study of obe-cel in adult r/r B-ALL.
  • “Acceptance of the BLA filing is an important milestone for Autolus and we look forward to continuing our collaboration with the FDA during the review cycle,” commented Dr. Christian Itin, Chief Executive Officer of Autolus.

Elevar Therapeutics To Present Two Posters From Phase 3 CARES-310 Study at 2024 ASCO Gastrointestinal Cancers Symposium

Retrieved on: 
Thursday, January 18, 2024
CP, Patient, Therapy, Sorafenib, PD-1, ASCO, Survival, Society, Camrelizumab, Quality of life, Hepatocellular carcinoma, Food, DIH, Hepatotoxicity, FDA, PDUFA, TKI, Moscone Center

ASCO GI will be held Jan. 18-20, 2024, at the Moscone Convention Center (West) in San Francisco, and online.

Key Points: 
  • ASCO GI will be held Jan. 18-20, 2024, at the Moscone Convention Center (West) in San Francisco, and online.
  • Elevar’s poster presentations are:
    Abstract Title: Impact of baseline liver function on survival outcomes in patients with unresectable hepatocellular carcinoma (uHCC) treated with camrelizumab + rivoceranib vs sorafenib: A post hoc analysis of study CARES-310.
  • The results of this post-hoc analysis may have implications for selection of initial and subsequent therapies for uHCC patients.
  • The FDA assigned Prescription Drug User Fee Act (PDUFA) target action dates in May 2024.

ENHERTU® Granted Priority Review in the U.S. for Patients with Metastatic HER2 Positive Solid Tumors

Retrieved on: 
Monday, January 29, 2024
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, Orbis, HER2, Immunohistochemistry, Neoplasm, Patient, IHC, MD, ADC, Daiichi Sankyo, Survival, Breakthrough therapy, Medicine, Cancer, Food, Safety, Priority review, Doctor of Philosophy, Breast, AstraZeneca, PDUFA, Pharmaceutical industry

ENHERTU is a specifically engineered HER2 directed antibody drug conjugate (ADC) being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca.

Key Points: 
  • ENHERTU is a specifically engineered HER2 directed antibody drug conjugate (ADC) being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca.
  • The Priority Review follows receipt of Breakthrough Therapy Designation granted by the FDA in August 2023 for ENHERTU in metastatic HER2 positive solid tumors.
  • Data from other supporting trials in patients with HER2 positive IHC 3+ tumors in the ENHERTU clinical development program, including DESTINY-Lung01 and DESTINY-CRC02 , also were included in the submission.
  • We will continue working closely with the FDA to bring this potential first tumor agnostic HER2 targeted medicine to patients as quickly as possible.”

Kyowa Kirin Successfully Completes Acquisition of Orchard Therapeutics, a Global Gene Therapy Leader for Rare Diseases

Retrieved on: 
Wednesday, January 24, 2024
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Patient, MHRA, Therapy, Health care, Civil service commission, European Commission, Kyowa Kirin, Sanfilippo syndrome, ADS, Food, MLD, Acquisition, Acceleration, CVR, FDA, Priority review, Sanfilippo, HSC, PDUFA, Pharmaceutical industry

Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151) a Japan-based global specialty pharmaceutical company (J-GSP) discovering and developing novel medicines utilizing the latest biotechnology, and Orchard Therapeutics plc (Orchard Therapeutics, Nasdaq: ORTX), a global gene therapy leader, today announced Kyowa Kirin has successfully completed the acquisition of Orchard Therapeutics.

Key Points: 
  • Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151) a Japan-based global specialty pharmaceutical company (J-GSP) discovering and developing novel medicines utilizing the latest biotechnology, and Orchard Therapeutics plc (Orchard Therapeutics, Nasdaq: ORTX), a global gene therapy leader, today announced Kyowa Kirin has successfully completed the acquisition of Orchard Therapeutics.
  • The acquisition of all outstanding shares of Orchard Therapeutics by way of a Scheme of Arrangement procedure under the UK Companies Act 2006 was completed on January 24, 2024, and Orchard Therapeutics has become a wholly-owned subsidiary of Kyowa Kirin.
  • “We are truly excited about the acquisition of Orchard Therapeutics, a leading provider of HSC gene therapy.
  • Kyowa Kirin anticipates significant synergies with Orchard Therapeutics and plans to announce its 2024 Financial Guidance in conjunction with its 2023 earnings on 7 February.

Formosa Pharmaceuticals Announces Licensing Agreement with Cristália, for the Commercialization of APP13007 for the Treatment of Inflammation and Pain Following Ocular Surgery

Retrieved on: 
Friday, January 26, 2024
Inflammation, LTDA, Food, PDUFA, Geography of Taiwan, Pain, FDA, Pharmaceutical industry

TAIPEI, Jan. 26, 2024 /PRNewswire/ -- Taiwan-based Formosa Pharmaceuticals ("Formosa", 6838.TWO) announced today that the company has entered into a licensing agreement with CRISTÁLIA PRODUTOS QUÍMICOS FARMACÊUTICOS LTDA ("Cristália"), for exclusive Brazilian rights to the commercialization of APP13007 (clobetasol propionate ophthalmic nanosuspension, 0.05%) for the treatment of inflammation and pain following ocular surgery.

Key Points: 
  • TAIPEI, Jan. 26, 2024 /PRNewswire/ -- Taiwan-based Formosa Pharmaceuticals ("Formosa", 6838.TWO) announced today that the company has entered into a licensing agreement with CRISTÁLIA PRODUTOS QUÍMICOS FARMACÊUTICOS LTDA ("Cristália"), for exclusive Brazilian rights to the commercialization of APP13007 (clobetasol propionate ophthalmic nanosuspension, 0.05%) for the treatment of inflammation and pain following ocular surgery.
  • The licensing deal includes upfront, development milestones, and sales royalties, with additional considerations throughout the term of the agreement.
  • APP13007's active ingredient is the superpotent corticosteroid, clobetasol propionate, and is derived from Formosa Pharma's proprietary APNT™ nanoparticle formulation platform.
  • Ricardo S. Pacheco, Chairman of Cristália stated: "We are pleased to have entered into this agreement with our development partner, Formosa Pharmaceuticals.

Beta-thalassemia Market to Witness Upsurge in Growth During the Study Period (2019-2032), Evaluates DelveInsight | Leading Companies - Vertex Pharmaceuticals, CRISPR Therapeutics, Agios Pharmaceuticals, Celgene, Forma Therapeutics

Retrieved on: 
Wednesday, January 17, 2024
Vertex Pharmaceuticals, Patient, Biologics license application, Therapy, Endocrine system, TGF, Heart, LAS, HBA, Spleen, Diagnosis, Anemia, Mouse, Thalassemia, Beta thalassemia, HB, CBC, RBC, Pharmacosmos, Disease, Iron overload, TDT, EU4, FDA, Blas, Prevalence, Liver, Novo Nordisk, PDUFA, Red blood cell, CRISPR Therapeutics, Hemoglobin A, Cell, Iron, Pharmaceutical industry

LAS VEGAS, Jan. 17, 2024 /PRNewswire/ -- DelveInsight's Beta-thalassemia Market Insights report includes a comprehensive understanding of current treatment practices, beta-thalassemia emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Key Points: 
  • According to DelveInsight's analysis, the market size for beta-thalassemia across the 7MM is expected to grow with a significant CAGR by 2032.
  • The promising beta-thalassemia therapies in the pipeline include CTX001, EDIT-301, Mitapivat, ACE-536, Panobinostat, Etavopivat tablets, and others.
  • In September 2023, Pharmacosmos has initiated a Phase II trial of SP-420 in patients with transfusion-dependent β-thalassemia.
  • In June 2023, FDA accepted the Biologics License Application (BLAs) of exagamglogene autotemcel (exa-cel) for transfusion-dependent beta thalassemia (TDT).

Beta-thalassemia Market to Witness Upsurge in Growth During the Study Period (2019-2032), Evaluates DelveInsight | Leading Companies - Vertex Pharmaceuticals, CRISPR Therapeutics, Agios Pharmaceuticals, Celgene, Forma Therapeutics

Retrieved on: 
Wednesday, January 17, 2024
Vertex Pharmaceuticals, Patient, Biologics license application, Therapy, Endocrine system, TGF, Heart, LAS, HBA, Spleen, Diagnosis, Anemia, Mouse, Thalassemia, Beta thalassemia, HB, CBC, RBC, Pharmacosmos, Disease, Iron overload, TDT, EU4, FDA, Blas, Prevalence, Liver, Novo Nordisk, PDUFA, Red blood cell, CRISPR Therapeutics, Hemoglobin A, Cell, Iron, Pharmaceutical industry

LAS VEGAS, Jan. 17, 2024 /PRNewswire/ -- DelveInsight's Beta-thalassemia Market Insights report includes a comprehensive understanding of current treatment practices, beta-thalassemia emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Key Points: 
  • According to DelveInsight's analysis, the market size for beta-thalassemia across the 7MM is expected to grow with a significant CAGR by 2032.
  • The promising beta-thalassemia therapies in the pipeline include CTX001, EDIT-301, Mitapivat, ACE-536, Panobinostat, Etavopivat tablets, and others.
  • In September 2023, Pharmacosmos has initiated a Phase II trial of SP-420 in patients with transfusion-dependent β-thalassemia.
  • In June 2023, FDA accepted the Biologics License Application (BLAs) of exagamglogene autotemcel (exa-cel) for transfusion-dependent beta thalassemia (TDT).

CRISPR Therapeutics Announces U.S. Food and Drug Administration (FDA) Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Tuesday, January 16, 2024
SCD, Vertex Pharmaceuticals, Patient, Therapy, Hospital, CRISPR, Haematopoietic system, Food, TDT, CRSP, FDA, PDUFA, CRISPR Therapeutics, Pharmaceutical industry

ZUG, Switzerland and BOSTON, Jan. 16, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.

Key Points: 
  • “We are pleased with the approval of CASGEVY in TDT well ahead of the PDUFA date,” said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics.
  • “The approval is a reflection of the power and versatility of the CRISPR platform to bring a potentially curative treatment option to patients suffering from this devastating disease.”
    The administration of CASGEVY requires experience in stem cell transplantation; therefore, our partner Vertex Pharmaceuticals Incorporated is engaging with experienced hospitals to establish a network of independently operated, authorized treatment centers (ATCs) throughout the U.S. to offer CASGEVY to patients.
  • All nine ATCs activated in the U.S. are able to offer CASGEVY to eligible patients with TDT and sickle cell disease (SCD).
  • Additional ATCs will be activated in the coming weeks and a complete list of ATCs can be accessed at CASGEVY.com.

Vertex Announces US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Tuesday, January 16, 2024
Health, FDA, Genetics, General Health, Clinical Trials, Pharmaceutical, Biotechnology, SCD, Vertex Pharmaceuticals, Patient, Hospital, CRISPR, Haematopoietic system, Food, Sickle cell disease, TDT, PDUFA, VRTX, Pharmaceutical industry

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.
  • “On the heels of the historic FDA approval of CASGEVY for sickle cell disease, it is exciting to now secure approval for TDT well ahead of the PDUFA date,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex.
  • All nine ATCs activated in the U.S. are able to offer CASGEVY to eligible patients with TDT and sickle cell disease (SCD).
  • Additional ATCs will be activated in the coming weeks and a complete list of ATCs can be accessed at CASGEVY.com .

TIVDAK® Supplemental Biologics License Application Accepted for Priority Review by FDA for Patients with Recurrent or Metastatic Cervical Cancer

Retrieved on: 
Tuesday, January 9, 2024
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, FDA, Clinical Trials, Woman, Patient, Progression-free survival, Genmab, Pneumonitis, Peripheral neuropathy, Survival, Pfizer, ESMO, Bleeding, PFE, Food, Cervical cancer, Disease, PFS, Safety, Congress, Priority review, Toxicity, PDUFA, Inc., Pharmaceutical industry

The application has been granted Priority Review with a Prescription Drug User Fee Act (PDUFA) goal date of May 9, 2024.

Key Points: 
  • The application has been granted Priority Review with a Prescription Drug User Fee Act (PDUFA) goal date of May 9, 2024.
  • The safety profile of TIVDAK in innovaTV 301 was consistent with its known safety profile as presented in the U.S. prescribing information.
  • In October 2023, results from the innovaTV 301 study were presented during a Presidential Symposium at the European Society of Medical Oncology (ESMO) Congress.
  • The accelerated approval is based on tumor response and durability of response from the innovaTV 204 pivotal Phase 2 single-arm clinical trial evaluating TIVDAK as monotherapy in patients with previously treated recurrent or metastatic cervical cancer.