Prescription Drug User Fee Act

Optinose Reports Third Quarter 2023 Financial Results and Operational Updates

Retrieved on: 
Thursday, November 9, 2023

YARDLEY, Pa., Nov. 09, 2023 (GLOBE NEWSWIRE) --  Optinose (NASDAQ:OPTN), a pharmaceutical company focused on patients treated by ear, nose and throat (ENT) and allergy specialists, today reported financial results for the quarter ended September 30, 2023, and provided operational updates.

Key Points: 
  • We are pleased with the progress we made in the third quarter towards our 2023 operating objectives while sharply reducing expenses.
  • Today’s results show that we are outperforming our initial expectations for 2023 XHANCE net revenues while operating far more efficiently and preparing our organization for a successful launch in 2024.
  • Previously, the Company expected XHANCE net revenues for the full year of 2023 to be between $64.0 to $70.0 million.
  • Members of the Company’s leadership team will host a conference call and presentation to discuss financial results and corporate updates beginning at 8:00 a.m. Eastern Time today.

U.S. Food and Drug Administration Accepts for Priority Review Bristol Myers Squibb’s Application for Breyanzi (lisocabtagene maraleucel) for Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL)

Retrieved on: 
Thursday, November 9, 2023

The FDA has granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 14, 2024.

Key Points: 
  • The FDA has granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 14, 2024.
  • Priority Review designation underscores the high unmet need and the significant advancement Breyanzi may offer this patient population for which there is no standard of care and limited treatment options.
  • “This FDA acceptance brings us one step closer to offering these patients, for the first time, a personalized, T-cell based treatment option.
  • TRANSCEND CLL 004 is the first pivotal multicenter study to show clinical benefit with a CD19-directed CAR T cell therapy in patients with relapsed or refractory CLL after progression following treatment with a BTKi and BCL2i.

Melinta Therapeutics and Venatorx Pharmaceuticals Announce Licensing Agreement to Commercialize Cefepime-Taniborbactam in the U.S.

Retrieved on: 
Thursday, November 9, 2023

The partnership follows Venatorx’s submission of a New Drug Application (NDA) for cefepime-taniborbactam for the treatment of cUTI including pyelonephritis, in adults.

Key Points: 
  • The partnership follows Venatorx’s submission of a New Drug Application (NDA) for cefepime-taniborbactam for the treatment of cUTI including pyelonephritis, in adults.
  • Venatorx has been assigned a Prescription Drug User Fee Act (PDUFA) target action date for February 22, 2024.
  • The U.S. Food and Drug Administration (FDA) granted cefepime-taniborbactam Qualified Infectious Disease Product (QIDP) and Fast Track designations for both the cUTI and HABP/VABP indications.
  • Venatorx has granted GARDP exclusive rights to distribute and sub-distribute cefepime-taniborbactam, once it is approved for clinical use, in low- and lower middle-income countries.

Liquidia Corporation Reports Third Quarter 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Tuesday, November 7, 2023

Oral arguments in appeal of ‘793 PTAB decision set for December 4, 2023

Key Points: 
  • Oral arguments in appeal of ‘793 PTAB decision set for December 4, 2023
    PDUFA goal date to add PH-ILD indication to YUTREPIA label is January 24, 2024
    MORRISVILLE, N.C., Nov. 07, 2023 (GLOBE NEWSWIRE) -- Liquidia Corporation (NASDAQ: LQDA) (Liquidia or the Company) today reported financial results for the third quarter ended September 30, 2023.
  • ET to discuss the financial results and provide a corporate update.
  • Cash totaled $76.2 million as of September 30, 2023, compared to $93.3 million as of December 31, 2022.
  • Total other expense, net was $0.9 million for the three months ended September 30, 2023, compared with $0.3 million for the three months ended September 30, 2022.

Verona Pharma Reports Third Quarter 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, November 2, 2023

LONDON and RALEIGH, N.C., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Verona Pharma plc (Nasdaq: VRNA) (“Verona Pharma” or the “Company”), a clinical-stage biopharmaceutical company focused on respiratory diseases, announces its financial results for the third quarter ended September 30, 2023, and provides a corporate update.

Key Points: 
  • PDUFA Target Action Date of June 26, 2024
    Conference call today at 9:00 a.m. EDT / 1:00 p.m. GMT
    LONDON and RALEIGH, N.C., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Verona Pharma plc (Nasdaq: VRNA) (“Verona Pharma” or the “Company”), a clinical-stage biopharmaceutical company focused on respiratory diseases, announces its financial results for the third quarter ended September 30, 2023, and provides a corporate update.
  • Net loss: Net loss was $14.7 million for the third quarter ended September 30, 2023 (Q3 2022: $15.6 million).
  • Verona Pharma will host an investment community webcast and conference call at 9:00 a.m. EDT / 1:00 p.m. GMT on Thursday, November 2, 2023, to discuss the third quarter 2023 financial results and the corporate update.
  • An electronic copy of the third quarter 2023 results press release will also be made available today on the Company’s website.

SpringWorks Therapeutics Reports Third Quarter 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Thursday, November 2, 2023

STAMFORD, Conn., Nov. 02, 2023 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today reported third quarter financial results for the period ended September 30, 2023 and provided an update on recent business highlights.

Key Points: 
  • – PDUFA target action date for nirogacestat NDA in adults with desmoid tumors remains set for November 27, 2023 –
    STAMFORD, Conn., Nov. 02, 2023 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today reported third quarter financial results for the period ended September 30, 2023 and provided an update on recent business highlights.
  • SpringWorks expects to file a Marketing Authorisation Application for nirogacestat with the European Medicines Agency (EMA) in the first half of 2024.
  • SpringWorks plans to file an IND for SW-682, the Company's TEAD inhibitor development candidate, in the fourth quarter of 2023.
  • Net Loss Attributable to Common Stockholders: SpringWorks reported a net loss of $79.4 million, or $1.27 per share, for the third quarter of 2023.

CRISPR Therapeutics Announces Completion of FDA Advisory Committee Meeting for Exagamglogene Autotemcel (exa-cel) for Severe Sickle Cell Disease

Retrieved on: 
Tuesday, October 31, 2023

ZUG, Switzerland and BOSTON, Oct. 31, 2023 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) today announced the completion of the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee meeting for exagamglogene autotemcel (exa-cel) for the treatment of SCD in people ages 12 and older with recurrent vaso-occlusive crises (VOCs). Exa-cel is the first potential therapy to emerge from a strategic partnership between CRISPR Therapeutics and Vertex Pharmaceuticals.

Key Points: 
  • Exa-cel is the first potential therapy to emerge from a strategic partnership between CRISPR Therapeutics and Vertex Pharmaceuticals.
  • If approved, exa-cel could be the first genetic therapy available to approximately twenty thousand people with severe SCD in the U.S.
  • The FDA granted priority review for exa-cel in the treatment of people with SCD and assigned a Prescription Drug User Fee Act (PDUFA) action date of December 8, 2023.
  • Exa-cel’s Biologics License Application (BLA) for transfusion-dependent beta-thalassemia (TDT) was assigned a PDUFA date of March 30, 2024.

XOMA Earns $5 Million Milestone Upon FDA Acceptance of Day One’s Tovorafenib NDA for Relapsed or Progressive Pediatric Low-Grade Glioma (pLGG)

Retrieved on: 
Tuesday, October 31, 2023

EMERYVILLE, Calif., Oct. 31, 2023 (GLOBE NEWSWIRE) -- XOMA Corporation (NASDAQ: XOMA), the biotech royalty aggregator, announced today it has earned a $5 million milestone related to the U.S. Food and Drug Administration’s (FDA) acceptance of Day One Biopharmaceuticals’ New Drug Application (NDA) for tovorafenib as a monotherapy for relapsed or progressive pediatric low-grade glioma (pLGG).

Key Points: 
  • EMERYVILLE, Calif., Oct. 31, 2023 (GLOBE NEWSWIRE) -- XOMA Corporation (NASDAQ: XOMA), the biotech royalty aggregator, announced today it has earned a $5 million milestone related to the U.S. Food and Drug Administration’s (FDA) acceptance of Day One Biopharmaceuticals’ New Drug Application (NDA) for tovorafenib as a monotherapy for relapsed or progressive pediatric low-grade glioma (pLGG).
  • The FDA has granted tovorafenib Priority Review and established a Prescription Drug User Fee Act (PDUFA) date of April 30, 2024.
  • “Tovorafenib has the potential to address a key unmet need in children whose low-grade gliomas with BRAF alterations have relapsed or progressed,” stated Owen Hughes, Executive Chairman of XOMA.
  • “A novel, targeted, orally available option has the opportunity to set a new standard-of-care in this patient population.”
    In March 2021, XOMA paid $13.5 million upfront plus a share of a future event-based milestone, to acquire the $54 million in potential milestones and mid-single digit royalties associated with tovorafenib, in addition to the economics associated with vosaroxin, from Viracta Therapeutics.

CRISPR Therapeutics Trading Halted Today; FDA Advisory Committee to Review Biologics License Application (BLA) for Exagamglogene Autotemcel (exa-cel) in Sickle Cell Disease (SCD)

Retrieved on: 
Tuesday, October 31, 2023

The U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee is meeting today to review the Biologics License Application (BLA) for exagamglogene autotemcel (exa-cel) for the treatment of sickle cell disease (SCD) in people ages 12 and older with recurrent vaso-occlusive crises.

Key Points: 
  • The U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee is meeting today to review the Biologics License Application (BLA) for exagamglogene autotemcel (exa-cel) for the treatment of sickle cell disease (SCD) in people ages 12 and older with recurrent vaso-occlusive crises.
  • The Advisory Committee meeting is scheduled for 9:00 AM ET.
  • The briefing materials can be found on the FDA website HERE .
  • The Prescription Drug User Fee Act date for completion of the review of the BLA for exa-cel for SCD is December 8, 2023.

bluebird bio to Present New and Updated Data from Gene Therapy Programs in Sickle Cell Disease and Beta-Thalassemia at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition

Retrieved on: 
Thursday, November 2, 2023

The meeting will take place December 9-12, 2023 at the San Diego Convention Center and online.

Key Points: 
  • The meeting will take place December 9-12, 2023 at the San Diego Convention Center and online.
  • lovo-cel treatment regimen largely reflects known side effects of hematopoietic stem cell collection and busulfan conditioning regimen and underlying sickle cell disease.
  • Updated iron management outcomes demonstrating sustained improvements in iron burden, with the majority of patients able to stop iron chelation therapy, will also be presented.
  • Safety of beti-cel treatment largely reflects the known side effects of hematopoietic stem cell collection and busulfan conditioning regimen.