Priority review

Updated Linvoseltamab Data Showcase Continued Deepening of Responses in Patients with Heavily Pre-Treated Multiple Myeloma

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воскресенье, июня 16, 2024
Journal, Congress, Neutropenia, Plasmacytoma, CRS, Patient, Progression-free survival, EHA, Doctor of Philosophy, Priority review, Death, B-cell maturation antigen, Cytokine release syndrome, Fast track (FDA), European Hematology Association, Safety, SOC, Multiple myeloma, Odds ratio, Grade 3, Probability, Infection, Anemia, Dor, BCMA, Survival, MD, Food, Columbia University, PFS, OS, Ageing, Regeneron Pharmaceuticals, Cancer, Kidney, Pharmaceutical industry

TARRYTOWN, N.Y., June 16, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that 14-month median follow-up data from the pivotal Phase 1/2 LINKER-MM1 trial of linvoseltamab in patients with relapsed/refractory (R/R) multiple myeloma (MM) were shared during an oral presentation at the European Hematology Association (EHA) Congress 2024 and published in the Journal of Clinical Oncology. These longer-term results show a deepening of responses following the 11-month median follow-up data presented at the American Association for Cancer Research Annual Meeting in April. Linvoseltamab is an investigational bispecific antibody designed to bridge B-cell maturation antigen (BCMA) on multiple myeloma cells with CD3-expressing T cells to facilitate T-cell activation and cancer-cell killing.

Key Points: 
  • These longer-term results show a deepening of responses following the 11-month median follow-up data presented at the American Association for Cancer Research Annual Meeting in April.
  • Linvoseltamab is an investigational bispecific antibody designed to bridge B-cell maturation antigen (BCMA) on multiple myeloma cells with CD3-expressing T cells to facilitate T-cell activation and cancer-cell killing.
  • “Previous results from LINKER-MM1 have demonstrated that linvoseltamab has compelling efficacy characterized by deep and durable responses.
  • “Additionally, a study using US-based electronic health record data to indirectly compare linvoseltamab to real-world standard-of-care treatment also support the overall body of evidence for this investigational medicine in heavily pretreated multiple myeloma.

Capricor Therapeutics Announces Positive Type-B Meeting with FDA for CAP-1002 Program for Duchenne Muscular Dystrophy with an Aim to Expedite BLA Pathway

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вторник, июня 11, 2024
Outline, Orphan, Marketing, OLE, PPMD, INN, DMD, SAN, Therapy, Exosome, International, Safety, Biologics license application, Priority review, World Health Organization, Regenerative medicine, BLA, FDA, Food, Duchenne muscular dystrophy, PRV, Biotechnology, Pharmaceutical industry

SAN DIEGO, June 11, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the successful completion of a Type-B meeting with the U.S. Food and Drug Administration (FDA) on next steps for the Biologics License Application (BLA) submission with its lead asset, CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD). Additionally, Deramiocel has been selected as the proposed International Nonproprietary Name (INN) for CAP-1002 by the World Health Organization.

Key Points: 
  • Additionally, Deramiocel has been selected as the proposed International Nonproprietary Name (INN) for CAP-1002 by the World Health Organization.
  • “The last year has been transformative for Capricor as we have moved rapidly towards potential approval for CAP-1002 for the treatment of DMD,” said Linda Marbán, Ph.D., Capricor’s chief executive officer.
  • Capricor plans to further outline the submission schedule with the FDA in the coming months.
  • Capricor retains full rights to the PRV, if received.

Zymeworks Announces China NMPA Acceptance of Biologics License Application for Zanidatamab for Second-Line Treatment of Biliary Tract Cancer

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понедельник, июня 10, 2024
CDE, Patient, Progression-free survival, Standard of care, Priority review, Safety, Senior, National Medical Products Administration, Biologics license application, BTC, Business, The Lancet, ICR, BLA, Quality control, FDA, Food, Partnership, NMPA, Business development, PFS, Pharmaceutical industry

Under the terms of Zymeworks’ Asia Pacific license and collaboration agreement with BeiGene for the development and commercialization of zanidatamab, Zymeworks is entitled to receive an $8 million milestone payment.

Key Points: 
  • Under the terms of Zymeworks’ Asia Pacific license and collaboration agreement with BeiGene for the development and commercialization of zanidatamab, Zymeworks is entitled to receive an $8 million milestone payment.
  • Zymeworks also remains eligible to receive up to $164 million based on additional milestones plus royalties on product sales in the Asia Pacific region.
  • Zanidatamab demonstrated clinically meaningful anticancer activity and durable responses in subjects with previously treated HER2-positive BTC.
  • “We thank our partners at Zymeworks for their dedication and contributions toward achievement of this important milestone for zanidatamab.

TAGRISSO® (osimertinib) granted Priority Review in the US for patients with unresectable, Stage III EGFR-mutated lung cancer

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понедельник, июня 10, 2024
Research, FDA, Clinical Trials, Biotechnology, Health, Pharmaceutical, General Health, Science, Oncology, NSCLC, Heart, Patient, LAURA, Progression-free survival, AstraZeneca, New Drug Application, QT interval, Shanda, Priority review, CRT, Cardiomyopathy, Risk, Safety, Electrolyte, Chemoradiotherapy, LVEF, BTD, Death, Breakthrough therapy, Food, Lung cancer, Neoplasm, EGFR, Pemetrexed, Pharmaceutical industry

If approved, TAGRISSO will be indicated for EGFRm patients whose tumors have exon 19 deletions or exon 21 (L858R) mutations.

Key Points: 
  • If approved, TAGRISSO will be indicated for EGFRm patients whose tumors have exon 19 deletions or exon 21 (L858R) mutations.
  • Median progression-free survival (PFS) was 39.1 months in patients treated with TAGRISSO versus 5.6 months for placebo.
  • Interstitial lung disease (ILD)/pneumonitis occurred in 4% of the 1813 TAGRISSO-treated patients; 0.4% of cases were fatal.
  • AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.

Press Release: ASCO: Sarclisa is first anti-CD38 to significantly improve progression-free survival in combination with VRd for newly diagnosed transplant-ineligible multiple myeloma in phase 3

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понедельник, июня 3, 2024
TAE, Académie Nationale de Médecine, ASCO, EHA, Dexamethasone, Cancer, Priority, Lenalidomide, Multiple myeloma, Patient, Safety, Society, Research, Abstract, Congress, MRD, Priority review, PFS, Risk, Disease, European, Food, Death, RD, FDA, European Hematology Association, Progression-free survival, Recurrence, Pharmaceutical industry

The use of Sarclisa in combination with VRd in transplant-ineligible NDMM is investigational and has not been fully evaluated by any regulatory authority.

Key Points: 
  • The use of Sarclisa in combination with VRd in transplant-ineligible NDMM is investigational and has not been fully evaluated by any regulatory authority.
  • At the median follow-up of 59.7 months, the median PFS with the Sarclisa-VRd combination was not reached versus 54.3 months with VRd.
  • Grade ≥3 treatment-emergent adverse events (TEAE) occurred in 91.6% of patients taking Sarclisa-VRd and 84% of patients taking VRd.
  • If approved, Sarclisa would be the first anti-CD38 therapy in combination with standard-of-care VRd in newly diagnosed patients not eligible for transplant, which would be the third indication for Sarclisa in multiple myeloma.

Telix Completes TLX250-CDx (Zircaix™) BLA Submission for Kidney Cancer Imaging

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воскресенье, июня 2, 2024
Food and Drug Administration, Medicine, Biologics license application, Network, Patient, TLX, Kidney, EAP, PPV, Kidney cancer, Breakthrough, SAS, Priority review, Clinical trial, ASX, Food, FDA, Physician, BLA, Pharmaceutical industry

The rolling BLA submission, initiated in December 20233 with timelines pre-agreed with the FDA, was based on Telix’s successful global Phase III ZIRCON4 study in ccRCC.

Key Points: 
  • The rolling BLA submission, initiated in December 20233 with timelines pre-agreed with the FDA, was based on Telix’s successful global Phase III ZIRCON4 study in ccRCC.
  • The clear cell variant of renal cancer is the most common and aggressive sub-type of kidney cancer.
  • As part of the BLA submission process, Telix has requested a Priority Review under the eligibility criteria of the Breakthrough Therapy designation6.
  • James Stonecypher, Chief Development Officer at Telix, stated, “Completing the BLA submission for TLX250-CDx represents a significant milestone for Telix as we bring our Breakthrough investigational kidney cancer imaging agent closer to market as a non-invasive diagnostic for patients.

Dupixent® (dupilumab) Recommended for EU Approval by the CHMP to Treat Patients with COPD

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пятница, мая 31, 2024
Patient, Headache, Diarrhea, Risk, Cough, COVID-19, Priority, Respiratory disease, Smoking, Food, Depression, FDA, Anxiety, Disease, Safety, Health, Mucus, Common, Hospital, Death, European Commission, COPD, Back pain, Chronic obstructive pulmonary disease, Regeneron Pharmaceuticals, Priority review, European, CHMP, Pharmaceutical industry

TARRYTOWN, N.Y. and PARIS, May 31, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the approval of Dupixent® (dupilumab) in the European Union (EU) as an add-on maintenance treatment in adults with uncontrolled chronic obstructive pulmonary disease (COPD) characterized by raised blood eosinophils. The European Commission is expected to announce a final decision on the Dupixent application in the coming months.

Key Points: 
  • The European Commission is expected to announce a final decision on the Dupixent application in the coming months.
  • COPD is a respiratory disease that damages the lungs and causes progressive lung function decline, and is the fourth leading cause of death worldwide.
  • COPD is also associated with a significant health and economic burden due to recurrent acute exacerbations that require systemic corticosteroid treatment and/or lead to hospitalization.
  • The use of Dupixent in COPD is investigational and is not yet approved by global regulatory authorities.

Press Release: Dupixent recommended for EU approval by the CHMP to treat patients with COPD

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пятница, мая 31, 2024
Patient, Headache, Diarrhea, Risk, Cough, COVID-19, Priority, Respiratory disease, Smoking, Food, Depression, FDA, Anxiety, Disease, Safety, Health, Mucus, Common, Hospital, Death, European Commission, COPD, Back pain, Chronic obstructive pulmonary disease, Priority review, European, CHMP

The European Commission is expected to announce a final decision on the Dupixent application in the coming months.

Key Points: 
  • The European Commission is expected to announce a final decision on the Dupixent application in the coming months.
  • COPD is a respiratory disease that damages the lungs and causes progressive lung function decline and is the fourth leading cause of death worldwide.
  • Safety results in both studies were generally consistent with the known safety profile of Dupixent in its approved indications.
  • The use of Dupixent in COPD is investigational and is not yet approved by global regulatory authorities.

Cyclo Therapeutics Achieves Landmark Milestone with Completion of Enrollment of Last Patient in Phase 3 Pivotal TransportNPC™ Trial of Niemann-Pick Type C1

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четверг, мая 30, 2024
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, Cyclo, Lung, Patient, RFL, SOC, NPC, Liver, FDA, Therapy, Overalls, Shoulder, Disease, Safety, PIP, CYTH, MD, Trial of the century, EMA, Feedback, Cell, Brain, Priority review, Spleen, Standard of care, DSM-IV codes, Pharmaceutical industry

The TransportNPC™ study is the most comprehensive controlled pivotal study regarding patient size, global footprint, duration and clinical outcomes of an investigational therapy for NPC1.

Key Points: 
  • The TransportNPC™ study is the most comprehensive controlled pivotal study regarding patient size, global footprint, duration and clinical outcomes of an investigational therapy for NPC1.
  • Additionally, the Company has enrolled ten (10) patients in its substudy per their adopted Paediatric Investigational Plan (PIP) treating newborn to 3 years of age.
  • “The completion of enrollment represents by far the most significant milestone for Cyclo Therapeutics to date.
  • For more information about the Company’s TransportNPC™ pivotal Phase 3 study, visit www.ClinicalTrials.gov and reference identifier NCT04860960 .

FDA Grants Priority Review to Genentech’s Inavolisib for Advanced Hormone Receptor-Positive, HER2-Negative Breast Cancer With a PIK3CA Mutation

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среда, мая 29, 2024
Oncology, Health, FDA, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, New Drug Application, Patient, Fulvestrant, Progression-free survival, Risk, Food, HER2, Recurrence, ROG, Death, Priority review, Roche, Pharmaceutical industry

“We welcome the FDA’s Priority Review designation for inavolisib, which underscores the urgency to bring this potential best-in-class treatment option to patients as quickly as possible.”

Key Points: 
  • “We welcome the FDA’s Priority Review designation for inavolisib, which underscores the urgency to bring this potential best-in-class treatment option to patients as quickly as possible.”
    The PIK3CA mutation is found in approximately 40% of HR-positive metastatic breast cancers.
  • Based on the Priority Review designation, the FDA has set a Prescription Drug User Fee Act date of November 27, 2024.
  • Inavolisib is currently being investigated in three company-sponsored Phase III clinical studies (INAVO120, INAVO121, INAVO122) in PIK3CA-mutated locally advanced or metastatic breast cancer in various combinations.
  • We continue to evaluate potential clinical development program expansion opportunities to address patient unmet needs in various tumor types across oncology.