Prescription Drug User Fee Act

Apellis Pharmaceuticals Reports First Quarter 2023 Financial Results

Retrieved on: 
Thursday, May 4, 2023
Retina, Prescription Drug User Fee Act, ALS, Travel, Swedish Orphan Biovitrum, APLS, Stargardt disease, AMD, European Medicines Agency, Clinical trial, CAD, Geographic atrophy, Stargardt, Cold agglutinin disease, Research, GA, DSM-IV codes, C3, VALIANT, VEGF, Shanda, Patient, Physician, Data, Administration, PEGASUS, PNH, PDUFA, Webcast, United Kingdom, R, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Food, Safety, Mobile phone, Pharmaceutical industry, Nursing, Sewage treatment, Video game, Medical device, EU, Health, G&A, OAKS

EMPAVELI for the treatment of PNH:

Key Points: 
  • EMPAVELI for the treatment of PNH:
    Apellis recorded $20.4 million in EMPAVELI U.S. net product revenue for the first quarter 2023.
  • Cost of sales were $7.8 million for the first quarter 2023, compared to $1.2 million for same period in 2022.
  • Apellis reported a net loss of $177.8 million for the first quarter 2023, compared to a net loss of $138.9 million for the same period in 2022.
  • Apellis will host a conference call and webcast to discuss its first quarter 2023 financial results and business highlights today, May 4, 2023, at 4:30 p.m.

Pacira BioSciences Reports First Quarter 2023 Financial Results

Retrieved on: 
Wednesday, May 3, 2023
PGA Tour Champions, Osteoarthritis, Prescription Drug User Fee Act, Partnership, Table, SG, Conference call, Bank statement, Telephone, Femoral nerve block, Tax, Term loan, GAAP, 340B Drug Pricing Program, Sale, Interest, Shanda, Bupivacaine, PIN, Investment, Pain management, PDUFA, Webcast, Pain, Depreciation, PGA, R, Invitation system, Research, FDA, Conference, Official, Management, Pharmaceutical industry, Security (finance), Video game, Bank, EBITDA

TAMPA, Fla., May 03, 2023 (GLOBE NEWSWIRE) -- Pacira BioSciences, Inc. (Nasdaq: PCRX), the industry leader in its commitment to non-opioid pain management and regenerative health solutions, today reported financial results for the first quarter of 2023.

Key Points: 
  • ET --
    TAMPA, Fla., May 03, 2023 (GLOBE NEWSWIRE) -- Pacira BioSciences, Inc. (Nasdaq: PCRX), the industry leader in its commitment to non-opioid pain management and regenerative health solutions, today reported financial results for the first quarter of 2023.
  • First quarter 2023 iovera° net product sales were $4.0 million, versus $3.0 million reported for the first quarter of 2022.
  • First quarter royalty revenues were $0.9 million in the first quarter of 2023, versus $0.6 million reported for the first quarter of 2022.
  • The Pacira management team will host a conference call to discuss the company’s financial results and recent developments today, Wednesday, May 3, 2023, at 8:30 a.m.

Novaliq Announces Presentation of New Scientific Data on CyclASol® 0.1% (Cyclosporine Ophthalmic Solution) at the American Society of Cataract and Refractive Surgery Annual Meeting

Retrieved on: 
Tuesday, May 2, 2023
Biotechnology, Health, Pharmaceutical, Optical, Clinical Trials, DED, Refractive surgery, Therapy, Patient satisfaction, Safety, Efficacy, Cataract, ASCRS, American Society of Cataract and Refractive Surgery, Telecanthus, Prescription Drug User Fee Act, Oil, MD, Patient, PDUFA, Ciclosporin, FDA, Society, Treatment, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Pharmaceutical industry, LASIK

CyclASol® is the first water-free topical anti-inflammatory and immunomodulating ophthalmic solution, containing 0.1% cyclosporine.

Key Points: 
  • CyclASol® is the first water-free topical anti-inflammatory and immunomodulating ophthalmic solution, containing 0.1% cyclosporine.
  • CyclASol® is preservative-free and does not contain any oils or surfactants, which can be irritating and disturbing for the tear film.
  • CyclASol® is currently under regulatory review for the treatment of signs and symptoms of dry eye disease (DED).
  • “Dry eye is a very common ocular surface disease and has also an influence on patient satisfaction after LASIK or cataract surgery.

Hyloris announces potential registration date for Maxigesic® IV in the US

Retrieved on: 
Tuesday, May 2, 2023
US Foods, CET, IV, Food and Drug Administration, Prescription Drug User Fee Act, Brussels Stock Exchange, FDA, Hikma Pharmaceuticals, Glass, Pharmaceutical industry, AFT, PDUFA

Hyloris’ partner AFT Pharmaceuticals (“AFT”) submitted the additional data to the FDA on 17 April 2023.

Key Points: 
  • Hyloris’ partner AFT Pharmaceuticals (“AFT”) submitted the additional data to the FDA on 17 April 2023.
  • The Prescription Drug User Fee Act (PDUFA) goal date of 17 October 2023 set by the FDA confirms the anticipated review period of 6 months, and a potential registration of Maxigesic® IV for the US market before the end of 2023.
  • Sales could follow soon after, with an exclusive license and distribution agreement already signed between AFT and Hikma Pharmaceuticals (“Hikma”).
  • Under the terms of the development collaboration agreement between Hyloris and AFT, Hyloris is eligible to receive a share on any product-related revenues, such as license fees, royalties, milestone payments, received by AFT.

U.S. FDA Accepts for Priority Review Supplemental Biologics License Application and EMA Validates Application for Reblozyl® (luspatercept-aamt) as First-Line Treatment of Anemia in Adults with Lower-Risk Myelodysplastic Syndromes (MDS)

Retrieved on: 
Monday, May 1, 2023
FDA, Health, Clinical Trials, Pharmaceutical, Cardiology, Biotechnology, Epoetin alfa, Bristol Myers Squibb, RBC, Priority review, Failure, ESA, Safety, European Medicines Agency, Transfusion-dependent anemia, Type, BMY, EMA, MDS, HB, Prescription Drug User Fee Act, Acquisition, Patient, Syndrome, Anemia, PDUFA, Merck, Regulation of tobacco by the U.S. Food and Drug Administration, NYSE, Food, Pharmaceutical industry, Luspatercept

In the U.S., the FDA has granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of August 28, 2023.

Key Points: 
  • In the U.S., the FDA has granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of August 28, 2023.
  • Priority Review designation underscores the high unmet need and value that Reblozyl could bring to this patient population.
  • In Europe, the EMA’s validation of the application confirms the submission is complete and begins the EMA’s centralized review process.
  • Reblozyl is being developed and commercialized through a global collaboration with Merck following Merck’s acquisition of Acceleron Pharma, Inc. in November 2021.

Appendix 4C Quarterly Activity Report

Retrieved on: 
Friday, April 28, 2023
DREAM, MESO, Executive, Neural correlates of consciousness, Myocardial infarction, MI, Symantec Endpoint Protection, LVEF, Stroke, Heart, Hyaluronic acid, Mesoblast, MPC 75, Pain, Journal, Prescription Drug User Fee Act, Patient, Survival, PDUFA, CRL, MSB, American College, FDA, CHF, Appendix, Complete Response Letter, BLA, JACC, PLI, Inflammation, Video game, Pharmaceutical industry

NEW YORK, April 27, 2023 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an activity report for the third quarter ended March 31, 2023.

Key Points: 
  • NEW YORK, April 27, 2023 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an activity report for the third quarter ended March 31, 2023.
  • Successful completion of a global private placement primarily to Mesoblast’s existing major US, UK, and Australian shareholders raising approximately US$40 million, net of transaction costs.
  • Revenue from royalties on sales of TEMCELL® HS Inj.1 sold in Japan by our licensee for the quarter were US$1.8 million.
  • The data from both studies formed key components of Mesoblast’s recent resubmission of its remestemcel-L BLA to FDA for children with SR-aGVHD.

Intercept Pharmaceuticals Reports First Quarter 2023 Financial Results and Provides Update on Commercial Launch Strategy for NASH

Retrieved on: 
Thursday, April 27, 2023
Intercept, Abstract, Note, FXR, EASL, Bezafibrate, European Association for the Study of the Liver, Therapy, NASH, Serum, PBC, Advisory Committee, Total, NDA, Pharmacokinetics, PPAR, Biomarker, Sale, Trial of the century, Wind, Congress, SAH, Prescription Drug User Fee Act, Patient, Intercept Pharmaceuticals, OCA, PDUFA, Depreciation, Growth, ANDA, FDA, Research, Regulation of tobacco by the U.S. Food and Drug Administration, Conference, Food, Safety, Pharmaceutical industry, Video game, Liver, FRESH

MORRISTOWN, N.J., April 27, 2023 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq: ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced its financial results for the quarter ended on March 31, 2023.

Key Points: 
  • The Company is actively progressing its launch readiness activities for OCA for the treatment of patients with pre-cirrhotic fibrosis due to NASH.
  • Further information on the Company’s commercial launch strategy for NASH will be shared during a conference call and webcast presentation 8:00 a.m. to 9:30 a.m.
  • Intercept recognized $68.0 million in net sales in the first quarter 2023 compared to $59.2 million in net sales in the prior year quarter.
  • ET
    The conference call and webcast discussing the Company’s first quarter 2023 financial results and commercial launch strategy for NASH will take place on April 27, 2023, at 8:00 a.m.

Santhera Announces Preliminary Unaudited 2022 Annual Results Ahead of Full Report Publication by End of May and Provides Corporate Update

Retrieved on: 
Thursday, April 27, 2023
Vamorolone, Prescription Drug User Fee Act, Duchenne muscular dystrophy, Civil service commission, Education, Vitamin D deficiency, Health care, Glucocorticoid, European Medicines Agency, NDA, LR, European Directive on Traditional Herbal Medicinal Products, Biomarker, BMD, Committee, EMA, Vomiting, Depression, Prednisone, Publication, DMD, Vertebral compression fracture, PAMS, Patient, Annual report, Leber's hereditary optic neuropathy, European Commission, CEST, Syndrome, PDUFA, Committee for Medicinal Products for Human Use, MAA, FDA, LHON, CHF, EC, Safety, News, Bone fracture, MHRA, CHMP, BST, Cryptocurrency, Pharmaceutical industry, Medical device, Dietary supplement, Dentistry, EU, Idebenone

As permitted by SIX Exchange Regulation, the Company will publish the full 2022 Annual Report by the end of May.

Key Points: 
  • As permitted by SIX Exchange Regulation, the Company will publish the full 2022 Annual Report by the end of May.
  • The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, vomiting and vitamin D deficiency.
  • SIX Exchange Regulation has permitted Santhera to publish its 2022 Annual Report by May 31, 2023, at the latest.
  • The Company plans to publish its audited 2022 Annual Report during May 2023.

Ironwood to Present New Data at Digestive Disease Week® 2023 Demonstrating Potential of Linaclotide for Functional Constipation in Children and Adolescents Ages 6-17 Years-Old

Retrieved on: 
Monday, April 24, 2023
Research, FDA, Clinical Trials, Biotechnology, Health, Pharmaceutical, General Health, Other Science, Science, Functional, Priority review, University, Ironwood Pharmaceuticals, Safety, OH, New Drug Application, Functional constipation, IBS-C, Constipation, Ageing, Irritable bowel syndrome, Ironwood, University of Miami, CIC, Medication, DDW, Defecation, Prescription Drug User Fee Act, Patient, AbbVie, Randomness, CST, PDUFA, Hospital, Nationwide, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Pharmaceutical industry, Birth control, Communications satellite, Linaclotide

Ironwood Pharmaceuticals, Inc. (Nasdaq: IRWD), a GI-focused healthcare company, announced today that the company will present findings during the 2023 Digestive Disease Week® (DDW) from studies on the potential of linaclotide for children and adolescents ages 6-17 years-old with functional constipation.

Key Points: 
  • Ironwood Pharmaceuticals, Inc. (Nasdaq: IRWD), a GI-focused healthcare company, announced today that the company will present findings during the 2023 Digestive Disease Week® (DDW) from studies on the potential of linaclotide for children and adolescents ages 6-17 years-old with functional constipation.
  • Ironwood had previously announced topline data from this trial in September 2022.
  • Poster presentations will highlight data on the efficacy of linaclotide in addressing bothersome functional constipation symptoms in pediatric patients ages 6-17 years old, as well as aggregate data on the safety of linaclotide in this patient population.
  • There are currently no FDA -approved prescription therapies for functional constipation.

Quizartinib NDA Review for Patients with Newly Diagnosed FLT3-ITD Positive AML Extended by FDA

Retrieved on: 
Friday, April 21, 2023
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, REMS, Prescription Drug User Fee Act, New Drug Application, NDA, ECG, Medicine, Risk, QT, Risk Evaluation and Mitigation Strategies, Acute myeloid leukemia, Incidence, AML, MD, Patient, Standard of care, Daiichi Sankyo, EHA, PDUFA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Safety, Food, TSE, Pharmaceutical industry

Daiichi Sankyo (TSE: 4568) today announced that the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) of quizartinib in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, and as continuation monotherapy following consolidation, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3-ITD positive.

Key Points: 
  • Daiichi Sankyo (TSE: 4568) today announced that the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) of quizartinib in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, and as continuation monotherapy following consolidation, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3-ITD positive.
  • The FDA has extended the Prescription Drug User Fee Act (PDUFA) action date by three months to July 24, 2023 to allow additional time to review requested updates to the proposed Risk Evaluation and Mitigation Strategies (REMS) included in this application.
  • The incidence of grade ≥3 QT prolongation events was low, with uncommon ventricular arrythmia events.
  • Overall, the risk of QT prolongation was manageable with ECG monitoring, quizartinib dose modification and correction/elimination of additional risk factors.