COMP

Mirum Pharmaceuticals Announces Positive CHMP Opinion for LIVMARLI® (maralixibat) Oral Solution for the Treatment of PFIC in Patients Three Months of Age and Older

Retrieved on: 
fredag, maj 31, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, Progressive familial intrahepatic cholestasis, Patient, ALGS, Shanda, Medication, Cholestatic pruritus, European, Orphan, Civil service commission, European Commission, Alagille syndrome, Cholestasis, Committee, COMP, CHMP, Pharmaceutical industry

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion of LIVMARLI® (maralixibat) oral solution for the treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) in patients three months of age and older.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion of LIVMARLI® (maralixibat) oral solution for the treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) in patients three months of age and older.
  • The CHMP positive opinion is the scientific recommendation to the European Commission for marketing authorization in Europe.
  • In addition, the Committee for Orphan Medicinal Products (COMP), has also recommended maintenance of Orphan Drug Designation for LIVMARLI in PFIC.
  • LIVMARLI is currently approved in Europe for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) two months of age and older.

Rocket Pharmaceuticals Receives Orphan Medicinal Product Designation from the European Commission for RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy

Retrieved on: 
onsdag, maj 29, 2024
Research, Genetics, Clinical Trials, Cardiology, Biotechnology, Health, Pharmaceutical, Science, Oncology, Patient, Disorder, PKP2, European Medicines Agency, Medication, Disease, Safety, Person, Marketing, Arrhythmogenic cardiomyopathy, Civil service commission, European Commission, EMA, Therapy, Committee, COMP, European, Arrest

PKP2-ACM is a devastating, inherited heart disease associated with life-threatening arrhythmias, cardiac structural abnormalities, and sudden cardiac death.

Key Points: 
  • PKP2-ACM is a devastating, inherited heart disease associated with life-threatening arrhythmias, cardiac structural abnormalities, and sudden cardiac death.
  • The current standard of care consists of medical therapy, implantable cardioverter defibrillators (ICDs), and ablation procedures.
  • Orphan medicinal product designation by the EC is available to novel therapeutics that prevent or treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 persons in the European Union (EU).
  • The study is assessing the impact of RP-A601 on PKP2 myocardial protein expression, cardiac biomarkers, clinical predictors of life-threatening ventricular arrhythmias, and sudden cardiac death.

Compass Strengthens Footprint in Southeastern U.S., Welcoming the Top Brokerage in Tennessee

Retrieved on: 
måndag, maj 13, 2024
Marketing, Real estate, Culture, COMP

The combination of Compass and Parks agents now represents one of four homes sold in Tennessee.

Key Points: 
  • The combination of Compass and Parks agents now represents one of four homes sold in Tennessee.
  • With this addition, Compass strengthens its position in Nashville and enhances its presence in the Southeastern U.S. Compass continues to attract top talent nationwide through deliberate acquisitions and organic growth.
  • "Today, we have significantly added to our presence in the Southeast, welcoming the top brokerage in Tennessee," noted Compass CEO & Founder Robert Reffkin.
  • In 2023, Compass introduced 103 new features to the Compass platform, including Compass AI and Performance Tracker.

Compass, Inc. Reports First Quarter 2024 Results

Retrieved on: 
onsdag, maj 8, 2024
Conference proceedings, Conversation, Net, AI, Growth, GTV, Privacy, GAAP, Disclosure, Internet, Learning, COMP, Machine learning, Software, Conference, National Association, Result, Form 10-Q, Annual report, NAR, Health, ...Continued, Insurance, Artificial intelligence, Federation, OPEX, Depreciation, Security (finance), Regulation Fair Disclosure, Risk, Analysis, Acquisition, Form 10-K, Regulation, Trust, Bank statement, Adjustment, Real estate

NEW YORK, May 8, 2024 /PRNewswire/ -- Compass, Inc. (NYSE: COMP) ("Compass" or "the Company"), the largest residential real estate brokerage in the United States by sales volume1 for the third year in a row, announced its financial results for the first quarter ended March 31, 2024.

Key Points: 
  • "This is the first time we have generated positive free cash flow in the first quarter, the industry's slowest quarter of the year, and in a historically challenging market.
  • OPEX for Q1 2024 was $211.2 million, an improvement of $32.1 million from Q1 2023 OPEX of $243.3 million."
  • Management will conduct a conference call to discuss the first quarter 2024 results as well as outlook at 5:00 p.m.
  • Accordingly, actual results could differ materially from those predicted or implied or such uncertainties could cause adverse effects on our results.

Benitec Biopharma Reports Positive Interim Clinical Trial Data for First OPMD Subject Treated with BB-301 in Phase 1b/2a Study

Retrieved on: 
torsdag, april 18, 2024
FDA, Dysphagia, Mutation, EDT, Nature, COMP, Oculopharyngeal muscular dystrophy, Hope, Food, News, Death, Yogurt, Pharmacotherapy, Observation, PABPN1, Disease, OPMD, Subject, Ptosis, Patient, EMA, Caregiver, Committee, Clinical trial, Trial of the century, Orphan drug, European Medicines Agency, Pharmaceutical industry

HAYWARD, Calif., April 18, 2024 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today announces positive interim clinical data from the 90-day timepoint following the administration of BB-301 to the study’s first subject (Subject 1) treated in the BB-301 Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study (NCT06185673) in Oculopharyngeal Muscular Dystrophy (OPMD). BB-301 has been granted Orphan Drug designation by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP).

Key Points: 
  • “To date, no clinical studies have systematically demonstrated a clinical improvement in OPMD patients across both objective and subjective measures of swallowing.
  • We are, therefore, pleased to report positive interim clinical data from multiple radiographic measures as well as subject-reported outcome measures from the first subject treated with BB-301,” said Jerel A.
  • Videofluoroscopic swallowing studies represent the gold standard analytical method for the quantitative assessment of dysphagia (swallowing difficulty) in the clinical setting.
  • The event replay will be placed on the News & Events tab on the Investor page of the Benitec website.

Electronic Offender Monitoring Solutions Report 2024: Smartphone Apps and Wrist-worn Devices Expand the Electronic Monitoring Market - Forecasts to 2028 - ResearchAndMarkets.com

Retrieved on: 
onsdag, april 24, 2024
Technology, Hardware, Apps, Applications, Sentinel, Companding, COMP, Spacecom, GPS, Software, Securus Technologies, EM, Private sector, Electronic, GEO Group, Sentinel Offender Services, Video game

The report estimates that the number of daily users will grow to 94,000 in Europe, 680,000 in North America and 239,000 in Latin America by 2028.

Key Points: 
  • The report estimates that the number of daily users will grow to 94,000 in Europe, 680,000 in North America and 239,000 in Latin America by 2028.
  • The market value in 2023 reached US$ 226 million in Europe, US$ 1.3 billion in North America and US$ 76 million in Latin America.
  • What are the latest developments on the Electronic Offender Monitoring market?
  • Electronic monitoring (EM) programmes was first introduced in the US in the early 1980s.

Compass to Announce First Quarter 2024 Earnings on Wednesday, May 8

Retrieved on: 
torsdag, april 18, 2024
Compass, COMP, Conference

NEW YORK, April 18, 2024 /PRNewswire/ -- Compass, Inc. (NYSE: COMP), the largest residential real estate brokerage in the United States by sales volume1, announces its first quarter 2024 financial results will be released after market close on Wednesday, May 8, 2024.

Key Points: 
  • NEW YORK, April 18, 2024 /PRNewswire/ -- Compass, Inc. (NYSE: COMP), the largest residential real estate brokerage in the United States by sales volume1, announces its first quarter 2024 financial results will be released after market close on Wednesday, May 8, 2024.
  • The company will host a conference call and webcast to discuss its results that afternoon at 5:00 p.m.
  • You can also register in advance to access the live conference call and shareholder presentation at: Compass Inc. 1Q24 Earnings Conference Call .
  • To access the replay and shareholder presentation, visit the Events and Presentations section of the Compass Investor Relations website.

Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- Treatment of cystic fibrosis, 14/10/2016 Withdrawn

Retrieved on: 
torsdag, april 18, 2024
Eurostat, Diagnosis, Civil service commission, Absorption, Lower respiratory tract infection, Exercise, Cell, Infection, DSM-IV codes, Inflammation, Patient, Committee, Cystic fibrosis, Knowledge, Digestion, Regulation, Bacteria, Food, EMA, IRIS, Anti-inflammatory, Mucus, CB2, European, COMP, Pancreas, Sponsor, Ivacaftor, European Commission, Safety, CFTR, Marketing, Medicine, Pharmaceutical industry

Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- Treatment of cystic fibrosis, 14/10/2016 Withdrawn

Key Points: 


Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- Treatment of cystic fibrosis, 14/10/2016 Withdrawn

Orphan designation: mRNA encoding the human CFTR gene Treatment of cystic fibrosis, 19/02/2024 Positive

Retrieved on: 
torsdag, april 18, 2024
Patient, Committee, Arcturus Therapeutics, Messenger, EMA, IRIS, European, Therapy, COMP, European Commission, CFTR, Marketing, Cystic fibrosis, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of cystic fibrosis in the European Union on 19 February 2024.
  • Orphan designation does not mean the medicine is available or authorised for use.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

Orphan designation: (4R)-3-(4-fluoro-2-hydroxyphenyl)-4-methyl-4,5-dihydro-1H-pyrazole-1-carboximidamide hydrochloride Treatment of systemic sclerosis, 12/01/2024 Positive

Retrieved on: 
torsdag, april 18, 2024
Patient, Committee, EMA, IRIS, COMP, European Commission, Marketing, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of systemic sclerosis on 12 January 2024.
  • Orphan designation does not mean the medicine is available or authorised for use.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: