Prescription Drug User Fee Act

U.S. Food and Drug Administration Accepts for Priority Review Bristol Myers Squibb’s Application for Repotrectinib for the Treatment of Patients with Locally Advanced or Metastatic ROS1-Positive Non-Small Cell Lung Cancer

Retrieved on: 
Tuesday, May 30, 2023
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, ENA, Bristol Myers Squibb, Priority review, File, New Drug Application, NDA, TKI, NSCLC, BMY, Prescription Drug User Fee Act, Patient, NTRK, Medicine, ROS1, PDUFA, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, NYSE, Food, Pharmaceutical industry

The FDA granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2023.

Key Points: 
  • The FDA granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2023.
  • “The FDA’s acceptance of this application marks an exciting milestone on our journey to bring this next-generation tyrosine kinase inhibitor to patients.
  • The study remains ongoing to assess long-term outcomes and additional endpoints across patient populations with ROS1-positive locally advanced or metastatic NSCLC and NTRK-positive advanced solid tumors.
  • As of August 2022, Bristol Myers Squibb acquired the leading clinical stage precision oncology company and its pipeline of investigational drugs across precision oncology and advanced solid tumors, including repotrectinib.

Iovance Biotherapeutics Announces U.S. Food and Drug Administration Acceptance of the Biologics License Application of Lifileucel for the Treatment of Advanced Melanoma

Retrieved on: 
Friday, May 26, 2023
Priority review, Interim, Prescription Drug User Fee Act, Regenerative Medicine Advanced Therapy, File, TIL, Clinical trial, Cancer, Iovance Biotherapeutics, Patient, Physician, Melanoma, PDUFA, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Food, Pharmaceutical industry

SAN CARLOS, Calif., May 26, 2023 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, today announced that the U.S. Food and Drug Administration (FDA) accepted its Biologics License Application (BLA) for lifileucel for patients with advanced melanoma. The FDA granted lifileucel Priority Review and assigned November 25, 2023 as the target action date for a decision under the Prescription Drug User Fee Act (PDUFA). The FDA is not currently planning to hold an advisory committee meeting to discuss this application and, after a preliminary review, has not at this time identified any potential review issues.

Key Points: 
  • The FDA granted lifileucel Priority Review and assigned November 25, 2023 as the target action date for a decision under the Prescription Drug User Fee Act (PDUFA).
  • Lifileucel is a TIL therapy intended for patients with advanced melanoma who progressed on or after prior anti-PD-1/L1 therapy and targeted therapy, where applicable.
  • The FDA also previously granted a Regenerative Medicine Advanced Therapy ( RMAT ) designation for lifileucel in advanced melanoma.
  • If lifileucel receives accelerated approval, the randomized Phase 3 TILVANCE-301 trial in frontline advanced melanoma can serve as the confirmatory study to support full approval.

FDA Advisory Committee Votes in Support of Approval for Pfizer’s Vaccine Candidate to Help Prevent RSV in Infants Through Maternal Immunization

Retrieved on: 
Thursday, May 18, 2023
Parenting, Infectious Diseases, Children, FDA, Baby, Maternity, Clinical Trials, Biotechnology, Pharmaceutical, Consumer, Health, Infection, Prescription Drug User Fee Act, Inc., Senior, Life, Safety, The New England Journal of Medicine, PFE, VRBPAC, Committee, Development, RSV, PDUFA, Vaccine, NYSE, Food, Pharmaceutical industry, Pfizer

The Committee voted 14 to 0 on effectiveness and 10 to 4 on safety.

Key Points: 
  • The Committee voted 14 to 0 on effectiveness and 10 to 4 on safety.
  • The vaccine candidate is currently under FDA review for the prevention of medically attended lower respiratory tract disease (MA-LRTD) and severe MA-LRTD caused by RSV in infants from birth up to six months of age by active immunization of pregnant individuals.
  • These results were also recently published in The New England Journal of Medicine.
  • The role of the VRBPAC is to provide recommendations to the FDA; however, these recommendations are not binding.

Health Canada Accepts New Drug Submission for Lecanemab as Treatment for Early Alzheimer's Disease

Retrieved on: 
Tuesday, May 16, 2023
Priority review, The New England Journal of Medicine, Plaque, NDS, Alzheimer's disease, MHLW, Marketing, Neurotoxicity, Food, Advisory Committee, European Medicines Agency, Medicines and Healthcare products Regulatory Agency, Lecanemab, Ministry of Health, Labour and Welfare, Brain, Medication, Pharmaceuticals and Medical Devices Agency, Oncology Drug Advisory Committee, EMA, Clarity, Ministry, Eisai, The Different Forms of Flowers on Plants of the Same Species, Prescription Drug User Fee Act, PMDA, National Medical Products Administration, Co-promotion, PDUFA, Ministry of Health (Kuwait), MAA, AD, FDA, Phase, Health Canada, BLA, NMPA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Disease, Medical imaging, Pharmaceutical industry, Biogen, Health

Lecanemab selectively binds and eliminates soluble, toxic Aβ aggregates (protofibrils) that are thought to contribute to the neurotoxicity in AD.

Key Points: 
  • Lecanemab selectively binds and eliminates soluble, toxic Aβ aggregates (protofibrils) that are thought to contribute to the neurotoxicity in AD.
  • As such, lecanemab may have the potential to have an effect on disease pathology and to slow down the progression of the disease.
  • The Clarity AD study of lecanemab met its primary endpoint and all key secondary endpoints with highly statistically significant results.
  • Lecanemab was approved under the accelerated approval pathway in the U.S. and was launched in the U.S. on January 18, 2023.

Health Canada Accepts New Drug Submission for Lecanemab as Treatment for Early Alzheimer’s Disease

Retrieved on: 
Tuesday, May 16, 2023
Priority review, The New England Journal of Medicine, Plaque, NDS, MHLW, Marketing, Neurotoxicity, Food, Advisory Committee, European Medicines Agency, Medicines and Healthcare products Regulatory Agency, Lecanemab, BIIB, Ministry of Health, Labour and Welfare, Brain, Pharmaceuticals and Medical Devices Agency, Medication, Oncology Drug Advisory Committee, EMA, Clarity, Ministry, The Different Forms of Flowers on Plants of the Same Species, Eisai, Prescription Drug User Fee Act, Headquarters, PMDA, National Medical Products Administration, Corporation, Co-promotion, PDUFA, Ministry of Health (Kuwait), MAA, AD, FDA, Phase, Health Canada, BLA, NMPA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Disease, Pharmaceutical industry, Biogen, Health

Lecanemab selectively binds and eliminates soluble, toxic Aβ aggregates (protofibrils) that are thought to contribute to the neurotoxicity in AD.

Key Points: 
  • Lecanemab selectively binds and eliminates soluble, toxic Aβ aggregates (protofibrils) that are thought to contribute to the neurotoxicity in AD.
  • As such, lecanemab may have the potential to have an effect on disease pathology and to slow down the progression of the disease.
  • The Clarity AD study of lecanemab met its primary endpoint and all key secondary endpoints with highly statistically significant results.
  • Lecanemab was approved under the accelerated approval pathway in the U.S. and was launched in the U.S. on January 18, 2023.

Fortress Biotech Reports First Quarter 2023 Financial Results and Recent Corporate Highlights

Retrieved on: 
Monday, May 15, 2023
CLL, Fortress Biotech, Checkpoint, Mustang Bio, Gout, Waldenström macroglobulinemia, Dotinurad, Radiation, Arm, Triplex, Fortification, Cytomegalovirus, Chronic lymphocytic leukemia, Follicular lymphoma, Biologics license application, Fred Hutchinson Cancer Research Center, ICML, Menkes disease, CKPT, New Drug Application, Clinical trial, Lymphoma, NDA, Prescription Drug User Fee Act, Prescription, Urica, WM, CAR, Waldenström, International Conference on Afghanistan, London (2010), Patient, Risk, Hyperuricemia, PD-1, FDA, Death, BLA, Conference, Rosacea, Safety, Disease, Pharmaceutical industry, Mastic (plant resin), PDUFA

MIAMI, May 15, 2023 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”), an innovative biopharmaceutical company focused on efficiently acquiring, developing and commercializing or monetizing promising therapeutic products and product candidates, today announced financial results and recent corporate highlights for the first quarter ended March 31, 2023.

Key Points: 
  • In March 2023, the FDA accepted the BLA filing for cosibelimab and set a PDUFA goal date of January 3, 2024.
  • Journey Medical’s total product net revenues were $12.2 million for the first quarter of 2023, compared to first quarter 2022 total product net revenues of $20.8 million.
  • On a non-GAAP basis, Fortress research and development expenses were $2.3 million for the first quarter of 2023, compared to $2.8 million for first quarter of 2022.
  • On a non-GAAP basis, Fortress selling, general and administrative expenses were $7.0 million, for the first quarter of 2023, compared to $6.2 million for the first quarter of 2022.

New 'Voice of the Patient' Report Highlights Unmet Medical Needs of Six Subtypes of Limb-Girdle Muscular Dystrophy

Retrieved on: 
Monday, May 15, 2023
Prescription Drug User Fee Act, Partnership, Muscular dystrophy, University, ML, Research, Patient, Common, Human voice, Sarepta Therapeutics, LGMD, PDUFA, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Caregiver, Pharmaceutical industry

This meeting focused on LGMD subtypes LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2i/R9.

Key Points: 
  • This meeting focused on LGMD subtypes LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2i/R9.
  • An additional 136 patients and family members responded to a live survey and over 250 written comments were submitted.
  • The LGMD Coalition is appreciative for support from our sponsors, Sarepta Therapeutics, AskBio, Edgewise Therapeutics, ML BioSolutions, and Vita Therapeutics.
  • We believe that their engagement in this process provided insights into these subtypes of LGMD which will help the research community develop effective therapies."

Heron Therapeutics Announces Financial Results for the Three Months Ended March 31, 2023 and Highlights Recent Corporate Updates

Retrieved on: 
Thursday, May 11, 2023
Prescription Drug User Fee Act, Centers for Medicare & Medicaid Services, Marketing, New Drug Application, Meloxicam, Center for Medicare and Medicaid Innovation, Vomiting, Medicare, Net, Aprepitant, Shanda, Comparables, Bupivacaine, Patient, PONV, Investment, SAN, Heron, PDUFA, Webcast, Growth, Research, Therapy, Video game, Management, Pharmaceutical industry, Internet service provider, Cryptocurrency

SAN DIEGO, May 11, 2023 /PRNewswire/ -- Heron Therapeutics, Inc. (Nasdaq: HRTX) (the "Company"), a commercial-stage biotechnology company focused on improving the lives of patients by developing and commercializing therapeutic innovations that improve medical care, today announced financial results for the three months ended March 31, 2023 and highlighted recent corporate updates.

Key Points: 
  • SAN DIEGO, May 11, 2023 /PRNewswire/ -- Heron Therapeutics, Inc. (Nasdaq: HRTX) (the "Company"), a commercial-stage biotechnology company focused on improving the lives of patients by developing and commercializing therapeutic innovations that improve medical care, today announced financial results for the three months ended March 31, 2023 and highlighted recent corporate updates.
  • First quarter 2023 net product sales grew 26% to $29.6 million, compared to the first quarter of 2022.
  • Net product sales of ZYNRELEF (bupivacaine and meloxicam) extended-release solution for the three months ended March 31, 2023 was $3.5 million.
  • Net product sales of APONVIE for the three months ended March 31, 2023 were $0.3 million.

Optinose Reports First Quarter 2023 Financial Results and Operational Updates

Retrieved on: 
Thursday, May 11, 2023
ENT, Patent, Exhalation delivery system, Ear, Conference call, OPTN, General Administration of Sport of China, Telephone, Prescription Drug User Fee Act, GAAP, Research, Nose, Orange Book, United States Patent and Trademark Office, Sinusitis, Patient, Exhalation, Approved Drug Products with Therapeutic Equivalence Evaluations, PDUFA, Webcast, FDA, MPH, Regulation of tobacco by the U.S. Food and Drug Administration, Food, Pharmaceutical industry, Cryptocurrency, MD, Conference

YARDLEY, Pa., May 11, 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 March 31, 2023, and provided operational updates.

Key Points: 
  • Conference call and webcast to be held today at 8:00 a.m. Eastern Time
    YARDLEY, Pa., May 11, 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 March 31, 2023, and provided operational updates.
  • “We are pleased with the progress we made in the first quarter of 2023 towards our strategic objectives," stated CEO Ramy Mahmoud, MD, MPH.
  • The Company expects XHANCE net revenues for the full year of 2023 to be between $62.0 to $68.0 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.

Krystal Biotech Announces First Quarter 2023 Financial Results and Operational Highlights

Retrieved on: 
Monday, May 8, 2023
NCT, Netherton syndrome, Cohort, European Medicines Agency, Ministry of Health, Labour and Welfare, Cystic fibrosis, Phase 1, Investment, Patient, Cystic Fibrosis Foundation, TGM1, Ministry of Health (Saudi Arabia), Association for Research in Vision and Ophthalmology, ARVO, Prescription Drug User Fee Act, Pannus, AATD, Rare, Jeune, FDA, IND, Skin, Safety, PIP, RDEB, Eye, Ministry, Financial result, Form, Research, Therapy, KRYS, DEB, MHLW, Clinical trial, Marketing, Annual general meeting, Pharmaceutical industry, Maple syrup, Ophthalmology, PDUFA, Health

Financial results for the quarter ended March 31, 2023:

Key Points: 
  • Financial results for the quarter ended March 31, 2023:
    Cash, cash equivalents, and investments totaled $355.5 million on March 31, 2023.
  • Research and development expenses for the quarter ended March 31, 2023 were $12.3 million, compared to $9.3 million for the quarter ended March 31, 2022.
  • General and administrative expenses for the quarter ended March 31, 2023 were $24.0 million, compared to $15.9 million for the quarter ended March 31, 2022.
  • For additional information on the Company’s financial results for the quarter ended March 31, 2023, please refer to the Form 10-Q filed with the SEC.