Medicines and Healthcare products Regulatory Agency

Tasso Recommended for Certification from Medical Device Single Audit Program (MDSAP)

Retrieved on: 
Thursday, May 18, 2023
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Tasso, Inc., the leading provider of patient-centric, clinical-grade blood collection solutions, today announced that it has been recommended for certification from the Medical Device Single Audit Program (MDSAP), under which a single regulatory audit of a medical device manufacturer may satisfy the requirements of multiple regulatory jurisdictions participating in the program.

Key Points: 
  • Tasso, Inc., the leading provider of patient-centric, clinical-grade blood collection solutions, today announced that it has been recommended for certification from the Medical Device Single Audit Program (MDSAP), under which a single regulatory audit of a medical device manufacturer may satisfy the requirements of multiple regulatory jurisdictions participating in the program.
  • The certification confirms Tasso’s compliance with the standards and regulatory requirements of Australia, Canada and the United States, opening potential new global markets for Tasso.
  • Once received, the certification also can streamline the process of reaching additional markets covered under the MDSAP, including Brazil and Japan.
  • The MDSAP program is part of the International Medical Device Regulators Forum.

Kapruvia® (difelikefalin) recommended by England's NICE for the treatment of adults with moderate-to-severe CKD-associated pruritus

Retrieved on: 
Thursday, May 18, 2023
New England, National Health Service, Barts Health NHS Trust, Chronic kidney disease, Medicine, Health care, Quality of life, Medication, CSL Vifor, Hemodialysis, Medicines and Healthcare products Regulatory Agency, Patient, Itch, Therapy, CKD, NICE, MHRA, National Institute for Health and Care Excellence, Nursing, Pharmaceutical industry

ST. GALLEN, Switzerland and STAMFORD, Conn., May 18, 2023 /PRNewswire/ -- Vifor Fresenius Medical Care Renal Pharma (VFMCRP) and Cara Therapeutics, Inc. (Nasdaq: CARA) today announced that England's National Institute for Health and Care Excellence (NICE) has recommended Kapruvia® for the treatment of moderate-to-severe CKD-associated pruritus in adult patients on haemodialysis. The decision follows authorisation from the UK's Medicines and Healthcare products Regulatory Agency (MHRA) in April 2022.

Key Points: 
  • The decision follows authorisation from the UK's Medicines and Healthcare products Regulatory Agency (MHRA) in April 2022.
  • "The recommendation of Kapruvia® by NICE is a key step on our journey to bring this breakthrough treatment to in-centre haemodialysis patients living with moderate-to-severe CKD-associated pruritus in the UK," said Fabio Dorigotti, Head Global Medical Affairs of CSL Vifor.
  • "We look forward to continue working with the National Health Service to ensure access to this important medicine for patients as quickly as possible."
  • "Together with VFMCRP, we are committed to bringing our first-of-its kind therapy to providers and patients around the world to help change the way pruritus is managed."

Kapruvia® (difelikefalin) recommended by England's NICE for the treatment of adults with moderate-to-severe CKD-associated pruritus

Retrieved on: 
Thursday, May 18, 2023
New England, National Health Service, Barts Health NHS Trust, Chronic kidney disease, Medicine, Health care, Quality of life, Medication, CSL Vifor, Hemodialysis, Medicines and Healthcare products Regulatory Agency, Patient, Itch, Therapy, CKD, NICE, MHRA, National Institute for Health and Care Excellence, Nursing, Pharmaceutical industry

ST. GALLEN, Switzerland and STAMFORD, Conn., May 18, 2023 /PRNewswire/ -- Vifor Fresenius Medical Care Renal Pharma (VFMCRP) and Cara Therapeutics, Inc. (Nasdaq: CARA) today announced that England's National Institute for Health and Care Excellence (NICE) has recommended Kapruvia® for the treatment of moderate-to-severe CKD-associated pruritus in adult patients on haemodialysis. The decision follows authorisation from the UK's Medicines and Healthcare products Regulatory Agency (MHRA) in April 2022.

Key Points: 
  • The decision follows authorisation from the UK's Medicines and Healthcare products Regulatory Agency (MHRA) in April 2022.
  • "The recommendation of Kapruvia® by NICE is a key step on our journey to bring this breakthrough treatment to in-centre haemodialysis patients living with moderate-to-severe CKD-associated pruritus in the UK," said Fabio Dorigotti, Head Global Medical Affairs of CSL Vifor.
  • "We look forward to continue working with the National Health Service to ensure access to this important medicine for patients as quickly as possible."
  • "Together with VFMCRP, we are committed to bringing our first-of-its kind therapy to providers and patients around the world to help change the way pruritus is managed."

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.

Cara Therapeutics Reports First Quarter 2023 Financial Results

Retrieved on: 
Monday, May 15, 2023
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STAMFORD, Conn., May 15, 2023 (GLOBE NEWSWIRE) -- Cara Therapeutics, Inc. (Nasdaq: CARA), a commercial-stage biopharmaceutical company leading a new treatment paradigm to improve the lives of patients suffering from pruritus, today announced financial results and operational highlights for the first quarter ended March 31, 2023.

Key Points: 
  • – Conference call today at 4:30 p.m. EDT –
    STAMFORD, Conn., May 15, 2023 (GLOBE NEWSWIRE) -- Cara Therapeutics, Inc. (Nasdaq: CARA), a commercial-stage biopharmaceutical company leading a new treatment paradigm to improve the lives of patients suffering from pruritus, today announced financial results and operational highlights for the first quarter ended March 31, 2023.
  • “The U.S. and global launches of KORSUVA® (difelikefalin) injection / Kapruvia® gained momentum in the first quarter of 2023.
  • Wholesalers shipped 45,720 vials to dialysis centers, the majority of which were Fresenius clinics, during the first quarter of 2023.
  • Vial orders more than doubled quarter to quarter, signifying an acceleration in patient demand.

Cocrystal Pharma Reports First Quarter 2023 Financial Results and Provides Updates on its Antiviral Drug Development Programs

Retrieved on: 
Monday, May 15, 2023
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BOTHELL, Wash., May 15, 2023 (GLOBE NEWSWIRE) -- Cocrystal Pharma, Inc. (Nasdaq: COCP) (Cocrystal or the Company) reports financial results for the three months ended March 31, 2023 and provides updates on its antiviral pipeline, upcoming milestones and business activities.

Key Points: 
  • BOTHELL, Wash., May 15, 2023 (GLOBE NEWSWIRE) -- Cocrystal Pharma, Inc. (Nasdaq: COCP) (Cocrystal or the Company) reports financial results for the three months ended March 31, 2023 and provides updates on its antiviral pipeline, upcoming milestones and business activities.
  • “Recent progress in advancing our pipeline of antiviral drug candidates keeps us on pace to meet our 2023 milestones,” said Sam Lee, Ph.D., President and co-CEO of Cocrystal.
  • Research and development expenses for the first quarter of 2023 were $3.9 million, compared with $2.9 million for the first quarter of 2022.
  • General and administrative expenses for the first quarter of 2023 were $1.2 million, compared with $1.3 million for the first quarter of 2022.

Orchard Therapeutics Reports First Quarter 2023 Financial Results and Announces Initiation of Rolling Submission for Biologics License Application of OTL-200 for Metachromatic Leukodystrophy to U.S. FDA

Retrieved on: 
Monday, May 15, 2023
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BOSTON and LONDON, May 15, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced various business accomplishments along with its financial results for the quarter ended March 31, 2023.

Key Points: 
  • OTL-200 is approved as Libmeldy® (atidarsagene autotemcel) by the European Commission (EC) and Medicines and Healthcare products Regulatory Agency (MHRA).
  • The company has secured reimbursement agreements for Libmeldy in three new markets so far in 2023.
  • Orchard Therapeutics has outlined the following key milestones expected for 2023:
    Libmeldy: Establish qualified treatment centers in Sweden, Spain and Saudi Arabia.
  • The company had approximately 184.3 million ordinary shares, equivalent to 18.4 million American Depositary Shares, outstanding as of March 31, 2023.

Neurocrine Biosciences Presents Data on Treatment of Patients with Congenital Adrenal Hyperplasia at ECE 2023

Retrieved on: 
Friday, May 12, 2023
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Post hoc analyses of these data released today assessed whether baseline hormone levels and glucocorticoid (GC) doses correlated with treatment response.

Key Points: 
  • Post hoc analyses of these data released today assessed whether baseline hormone levels and glucocorticoid (GC) doses correlated with treatment response.
  • See the abstract (Poster #274; Response to Crinecerfont Treatment in Adults with Classic Congenital Adrenal Hyperplasia is Correlated with Elevated Baseline Hormone Levels, but Not Glucocorticoid Dose) for more information here .
  • "Androgen excess is a hallmark feature of classic congenital adrenal hyperplasia.
  • These data will be part of an oral presentation (Oral Presentation, May 15, 2:20–2:30pm: Switching Patients with Congenital Adrenal Hyperplasia to Modified-Release Hydrocortisone Capsules: Relative Bioavailability and Disease Control).

Decibel Therapeutics Announces Approval of Clinical Trial Application by the Spanish Agency of Medicines and Medical Devices (AEMPS) to Initiate Clinical Development of Lead Gene Therapy Candidate DB-OTO

Retrieved on: 
Friday, May 12, 2023
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The Phase 1/2 dose escalation clinical trial, known as CHORD™, is designed to evaluate the safety, tolerability and efficacy of DB-OTO in pediatric patients with congenital hearing loss due to an otoferlin deficiency. In addition to safety and tolerability endpoints, the auditory brainstem response (ABR) -- an objective, clinically accepted, physiologic measure of hearing sensitivity -- will be used as an efficacy endpoint in the clinical trial. The ABR, which was used to characterize dose-response of DB-OTO after intra-cochlear delivery in translational studies in animal models, provides an opportunity to rapidly assess hearing functionality and sensitivity.

Key Points: 
  • BOSTON, May 12, 2023 (GLOBE NEWSWIRE) -- Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, today announced approval of its Clinical Trial Application (CTA) by the Spanish Agency of Medicines and Medical Devices (AEMPS) to initiate a Phase 1/2 clinical trial in pediatric patients of DB-OTO, its lead gene therapy product candidate.
  • Medicines and Healthcare Products Regulatory Agency (MHRA) in January 2023.
  • Decibel expects that clinical trial participants in the U.K. and Spain will be infants two years of age and younger.
  • In addition to safety and tolerability endpoints, the auditory brainstem response (ABR) -- an objective, clinically accepted, physiologic measure of hearing sensitivity -- will be used as an efficacy endpoint in the clinical trial.