PDUFA

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

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

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

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

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.

TIVDAK® (tisotumab vedotin-tftv) Supplemental Biologics License Application Accepted for Priority Review by U.S. Food and Drug Administration for Patients with Recurrent or Metastatic Cervical Cancer

Retrieved on: 
Tuesday, January 9, 2024

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

Key Points: 
  • The application has been granted Priority Review with a Prescription Drug User Fee Act (PDUFA) action date of May 9, 2024.
  • The safety profile of tisotumab vedotin-tftv 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 the Presidential Symposium at the European Society of Medical Oncology (ESMO) Congress.
  • “The FDA acceptance of our sBLA for review is important progress toward continuing to offer an option that can extend the lives of more adults with cervical cancer.”

Mirum Pharmaceuticals Announces Preliminary Unaudited 2023 Net Revenue and Provides Corporate Updates

Retrieved on: 
Monday, January 8, 2024

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today provided its preliminary and unaudited estimates for full-year 2023 revenue and net product sales, corporate updates, and full-year 2024 outlook.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today provided its preliminary and unaudited estimates for full-year 2023 revenue and net product sales, corporate updates, and full-year 2024 outlook.
  • “2023 was a transformative year for Mirum as we cemented our position as a leader in rare disease and dramatically advanced our operating and financial scale.
  • Additional information and disclosure would be required for a more complete understanding of the company’s financial position and results of operations as of December 31, 2023.
  • Mirum will present at the 42nd annual J.P. Morgan Healthcare Conference in San Francisco on Wednesday, January 10, 2024 at 10:30 a.m. PT.

Abeona Therapeutics Announces $50 Million Credit Facility

Retrieved on: 
Monday, January 8, 2024

CLEVELAND, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that it has entered into a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P.

Key Points: 
  • CLEVELAND, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that it has entered into a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P.
  • The credit agreement, which has a term of three and a half years, includes a first tranche of $20 million at closing, a second tranche of $10 million of committed capital, and an additional accordion option to upsize the credit facility by an additional $20 million upon satisfaction of certain terms and conditions.
  • The FDA has accepted and granted Priority Review with a PDUFA target action date of May 25, 2024 for the Biologics License Application for pz-cel.
  • “We are excited to enter into this relationship with Avenue Venture Fund and secure additional financial resources to further support our launch and commercialization efforts for pz-cel,” said Joe Vazzano, Chief Financial Officer of Abeona.

Zevra Therapeutics Receives FDA Acceptance of Resubmission of NDA for Arimoclomol as a Treatment for Niemann-Pick Disease Type C

Retrieved on: 
Monday, January 8, 2024

CELEBRATION, Fla., Jan. 08, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra or the Company), a rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has acknowledged receipt of the resubmission of the New Drug Application (NDA) for arimoclomol as an orally-delivered, first-in-class treatment for Niemann-Pick disease type C. Under the Prescription Drug User Fee Act (“PDUFA”), the FDA has deemed the arimoclomol NDA resubmission to be a Class II complete response which has a six-month review period from the date of resubmission. As a result, the FDA has assigned a PDUFA action date of June 21, 2024, and currently intends to present the resubmission for discussion in an advisory committee.

Key Points: 
  • As a result, the FDA has assigned a PDUFA action date of June 21, 2024, and currently intends to present the resubmission for discussion in an advisory committee.
  • “We are very pleased that the FDA has accepted the resubmission of the arimoclomol NDA following multiple collaborative and constructive meetings,” said Neil F. McFarlane, President and Chief Executive Officer of Zevra.
  • “This significant milestone brings us one step closer to the potential approval of arimoclomol for a community of patients with debilitating unmet medical needs.
  • The resubmission includes additional evidence supporting trial metrics, FDA-preferred analyses, and data from multiple additional studies that provide supporting evidence of arimoclomol’s efficacy in clinical and non-clinical settings.

CRISPR Therapeutics Highlights Strategic Priorities and 2024 Outlook

Retrieved on: 
Monday, January 8, 2024

ZUG, Switzerland and BOSTON, Jan. 08, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today highlighted its strategic priorities and 2024 outlook as the Company enters its next phase of growth.

Key Points: 
  • ZUG, Switzerland and BOSTON, Jan. 08, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today highlighted its strategic priorities and 2024 outlook as the Company enters its next phase of growth.
  • Exa-cel is the first therapy to emerge from a strategic partnership between CRISPR Therapeutics and Vertex Pharmaceuticals.
  • Vertex leads global development, manufacturing, regulatory and commercialization of CASGEVY with support from CRISPR Therapeutics.
  • CRISPR Therapeutics continues to focus on resource efficiency and return on invested capital as it advances multiple clinical programs across its pipeline.

Ascendis Pharma Introduces Vision 2030

Retrieved on: 
Sunday, January 7, 2024

COPENHAGEN, Denmark, Jan. 07, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today introduced selected 2024 corporate milestones and Vision 2030, its strategic roadmap through 2030. Ascendis President and CEO Jan Mikkelsen will present the update tomorrow, January 8, at the 42nd Annual J.P. Morgan Healthcare Conference.

Key Points: 
  • COPENHAGEN, Denmark, Jan. 07, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today introduced selected 2024 corporate milestones and Vision 2030, its strategic roadmap through 2030.
  • Ascendis President and CEO Jan Mikkelsen will present the update tomorrow, January 8, at the 42nd Annual J.P. Morgan Healthcare Conference.
  • Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.
  • The Company’s slides from the J.P. Morgan presentation will be available on the same Investor Relations website at https://investors.ascendispharma.com .