Priority review

Applied Therapeutics Reports Fourth Quarter and Year-end 2023 Financial Results

Retrieved on: 
Wednesday, March 6, 2024
Janus, Sorbitol, Marketing, EMA, Doctor of Philosophy, New Drug Application, MAA, Interim analysis, P-value, FDA, Food, Growth, Treatment, INSPIRE, Priority review, PDUFA, Chairperson, Therapy, CHMP, European Medicines Agency, Patient, Private placement, PRV, NDA, Ageing, Pharmaceutical industry

The FDA also noted that it is planning to hold an advisory committee meeting to discuss the application.

Key Points: 
  • The FDA also noted that it is planning to hold an advisory committee meeting to discuss the application.
  • Govorestat was previously granted Pediatric Rare Disease designation and will qualify for a Priority Review Voucher (PRV) upon approval.
  • The Company expects a decision by the EMA in the fourth quarter of 2024.
  • Full study results will be presented at an upcoming medical conference, along with results of the Diabetic Peripheral Neuropathy sub-study, which are still being analyzed.

Madrigal Pharmaceuticals Announces EMA Validation of its Marketing Authorization Application for Resmetirom for the Treatment of NASH/MASH with Liver Fibrosis

Retrieved on: 
Tuesday, March 5, 2024
Therapy, EMA, Committee, MAA, FDA, PDUFA, Liver cancer, Cirrhosis, Liver failure, Data, THR, MASH, Chronic, CHMP, Death, Patient, Fibrosis, NASH, Disease, Breakthrough therapy, Fatty liver disease, Development, Priority review, Pharmaceutical industry

NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally.

Key Points: 
  • NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally.
  • Resmetirom is a once-daily, oral, liver-directed THR-β agonist designed to target key underlying causes of NASH.
  • The clinical development program for resmetirom is comprised of 18 clinical studies supporting the MAA: twelve Phase 1 studies, two Phase 2 studies, and four Phase 3 studies.
  • Without treatment, the disease can lead to cirrhosis, liver failure, liver cancer and premature death,” said Becky Taub, M.D., Chief Medical Officer and President of Research & Development of Madrigal.

Capricor Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, February 29, 2024
Vaccine, Project, Webcast, Exosome, Protein, Poster, Mouse, Infection, Liver, Bangladesh Technical Education Board, Biotechnology, NIAID, Marketing, File, BLA, Therapy, Congress, PPMD, PUL, WMS, CMC, Distribution, International, Regenerative medicine, ID, Duchenne muscular dystrophy, Safety, Dicarboxylic acid, Biologics license application, IND, Commercialization, SARS-CoV-2, Lipid, Messenger, MicroRNA, Inflammation, FDA, Food, DMD, Priority review, Cell, COVID-19, Report, Patient, Oligonucleotide, PRV, Kidney, Tissue, Pharmaceutical industry

Further, Capricor will receive a meaningful mid-range double-digit revenue share of product revenue under the terms of this Agreement.

Key Points: 
  • Further, Capricor will receive a meaningful mid-range double-digit revenue share of product revenue under the terms of this Agreement.
  • Next steps for Cohort A: plan to report top-line data in the fourth quarter of 2024.
  • Revenues for the fourth quarter of 2023 were approximately $12.1 million compared with approximately $1.0 million for the fourth quarter of 2022.
  • Operating expenses: Total operating expenses for the fourth quarter of 2023 were approximately $13.2 million compared with approximately $9.0 million for the fourth quarter of 2022.

PRISM MarketView Highlights Work of Emerging Companies on Rare Disease Day

Retrieved on: 
Thursday, February 29, 2024
Rare disease, Therapy, Regenerative Medicine Advanced Therapy, FAP, Concha bullosa, Sulindac, Chemistry, Fast track (FDA), Muscular dystrophy, FDA, Sensation, News, Eflornithine, DMD, Prader–Willi syndrome, BLA, Chromosome, RDEB, PDUFA, Skin, Voyager, PRISM, Duchenne muscular dystrophy, Safety, Neurocrine Biosciences, Rare Disease Day, Genetics, Fatigue, Walking, PWS, Patient, DSM-IV codes, Medicine, Disease, Exosome, FA, CMC, Priority review, Pharmaceutical industry

All areas of medicine, and all organs and body systems, are impacted by rare diseases, including rare neurological and neuromuscular diseases, metabolic, skin and bone diseases, and chromosomal disorders.

Key Points: 
  • All areas of medicine, and all organs and body systems, are impacted by rare diseases, including rare neurological and neuromuscular diseases, metabolic, skin and bone diseases, and chromosomal disorders.
  • Today on Rare Disease Day, PRISM MarketView highlights emerging companies working to deliver life changing treatments for those living with rare diseases.
  • RDEB is a severe rare disease characterized by extremely fragile, and extensive blistering and wounds.
  • Capricor is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases.

GENFIT Announces Revenues and Cash Position as of December 31, 2023

Retrieved on: 
Thursday, February 29, 2024
Marketing, EMA, New Drug Application, MAA, Annual report, FDA, Food, Part, PDUFA, NTZ, PBC, Primary biliary cholangitis, Collaboration, European Medicines Agency, Patient, Elafibranor, NDA, Research, Priority review, Pharmaceutical industry

As of December 31, 2023, the Company’s cash and cash equivalents amounted to €77.8 million compared with €140.2 million as of December 31, 2022.

Key Points: 
  • As of December 31, 2023, the Company’s cash and cash equivalents amounted to €77.8 million compared with €140.2 million as of December 31, 2022.
  • As of September 30, 2023, cash and cash equivalents totaled €93.9 million.
  • Revenues for 2023 amounted to €28.6 million compared to €20.2 million for the same period in 2022.
  • The 2023 Universal Registration Document, the 2023 Annual Financial Report (included in the 2023 Universal Registration Document), and the Annual Report on Form 20-F will be published by the end of April 2024.

Mesoblast Reports Financial Results and Operational Update for Half-Year Ended December 31, 2023

Retrieved on: 
Wednesday, February 28, 2024
DREAM, RPD, Growth, PLI, SIX, BMT, FIRST, Diagnosis, Mortality, Bangladesh Technical Education Board, Blood, HLHS, MSB, Degenerative disc disease, NIH, Marketing, Pain, MACE, BLA, Survival, Heart failure, Hypoplastic left heart syndrome, LVAD, Randomized controlled trial, FINANCIAL, Inflammation, Biopharmaceutical, ODD, Heart, Mesoblast, Regenerative Medicine Advanced Therapy, FDA, Priority review, MESO, Risk, Administration, Death, Patient, PRV, DDD, Pharmaceutical industry

NEW YORK, Feb. 28, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an operational update and reported financial results for the period ended December 31, 2023.

Key Points: 
  • NEW YORK, Feb. 28, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an operational update and reported financial results for the period ended December 31, 2023.
  • Reduction in net cash usage for operating activities:
    For the three months ended December 31, 2023, net cash usage was US$12.3 million, a 25% reduction versus the comparative quarter in FY2023.
  • For the six months ended December 31, 2023, net cash usage was US$26.6 million, a 14% reduction versus the comparative period in FY2023.
  • Manufacturing reduced by 47% for the six months ended December 31, 2023, from US$12.8 million to US$6.7 million.

Applied Therapeutics Announces FDA Acceptance and Priority Review of New Drug Application for Govorestat for the Treatment of Classic Galactosemia

Retrieved on: 
Wednesday, February 28, 2024
ARI, Marketing, EMA, Central nervous system, Doctor of Philosophy, New Drug Application, MAA, Physician, FDA, Food, Priority review, Hope, Therapy, Galactitol, CHMP, European Medicines Agency, CNS, Patient, PRV, Diagnosis, NDA, Ageing, Pharmaceutical industry

NEW YORK, Feb. 28, 2024 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) has accepted the filing of the New Drug Application (NDA) for govorestat (AT-007) for the treatment of Classic Galactosemia. The NDA was granted Priority Review status, and the FDA assigned a Prescription Drug User Free Act (PDUFA) target action date of August 28, 2024. The FDA also noted that it is planning to hold an advisory committee meeting to discuss the application. Govorestat was previously granted Pediatric Rare Disease designation, and will qualify for a Priority Review Voucher (PRV) upon approval.

Key Points: 
  • The NDA was granted Priority Review status, and the FDA assigned a Prescription Drug User Free Act (PDUFA) target action date of August 28, 2024.
  • Govorestat was previously granted Pediatric Rare Disease designation, and will qualify for a Priority Review Voucher (PRV) upon approval.
  • “The FDA’s acceptance of the NDA for govorestat for the treatment of Galactosemia represents a critical milestone for Applied Therapeutics and more importantly, for patients with Galactosemia and their families.
  • If approved, govorestat would be the first medication indicated for the treatment of Galactosemia and would be Applied Therapeutics’ first commercial product.

Day One Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Progress

Retrieved on: 
Monday, February 26, 2024
BRAF, Pfizer, Research, New Drug Application, Doctor of Philosophy, MD, Investment, FDA, Food, Priority review, Day One, Event, Patient, Medicine, Clinical trial, Nature Medicine, NDA, Head, Ageing, MEK, Pharmaceutical industry

BRISBANE, Calif., Feb. 26, 2024 (GLOBE NEWSWIRE) -- Day One Biopharmaceuticals (Nasdaq: DAWN) (“Day One” or the “Company”), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, today announced its fourth quarter and full year 2023 financial results and highlighted recent corporate achievements.

Key Points: 
  • “We have a monumental year ahead of us at Day One with the upcoming PDUFA date for tovorafenib,” said Jeremy Bender, Ph.D., chief executive officer of Day One.
  • The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2024.
  • In November 2023, Nature Medicine published the registrational Phase 2 FIREFLY-1 trial results investigating tovorafenib in patients with BRAF-altered, relapsed or progressive pediatric low-grade glioma (pLGG).
  • In the fourth quarter of 2023, Day One continued its commercial preparedness for the approval and launch of tovorafenib with the hiring of 18 sales representatives in the U.S.

Krystal Biotech Announces Fourth Quarter and Full Year 2023 Financial Results and Provides Business Updates

Retrieved on: 
Monday, February 26, 2024
Research, Investment, Form 10-K, Sale, Form, HSV, Society, KRYS, Immunotherapy, Trial of the century, Genotype, Eye, PI, Krystal, Transgene, European Medicines Agency, Security (finance), Pharmacology, NCT, DEB, Jeune, ID, ODD, AATD, Safety, Survival, Medicaid, IND, Bronchoscopy, IL-12, Gene, MAA, FDA, Patent, Priority review, Cell, Lung, Patient, Medicine, Pharmaceutical industry

(the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today reported financial results and key business updates for the fourth quarter and year ending December 31, 2023.

Key Points: 
  • (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today reported financial results and key business updates for the fourth quarter and year ending December 31, 2023.
  • Financial results for the quarter ended December 31, 2023:
    Cash, cash equivalents, and investments totaled $594.1 million on December 31, 2023.
  • The Company recorded net product revenue of $42.1 million from sales of VYJUVEK during the quarter ended December 31, 2023.
  • For additional information on the Company’s financial results for the year ended December 31, 2023, please refer to the Form 10-K filed with the Securities and Exchange Commission.

Dupixent® (dupilumab) sBLA Accepted for FDA Priority Review for Treatment of COPD with Type 2 Inflammation

Retrieved on: 
Friday, February 23, 2024
Regeneron Pharmaceuticals, Headache, Diarrhea, FDA, Food, Smoker, Back pain, Chronic obstructive pulmonary disease, Common, Safety, COVID-19, Patient, Priority, Disease, Diagnosis, Priority review, COPD, Pharmaceutical industry

TARRYTOWN, N.Y. and PARIS, Feb. 23, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that 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. Regulatory submissions are under review in China and Europe.

Key Points: 
  • The target action date for the FDA decision is June 27, 2024.
  • Regulatory submissions are under review in China and Europe.
  • In both trials, Dupixent 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.